Neurology & Neurological Sciences Clinical Trials
How to Participate
Sign up here to be added to a registry for clinical trials. This survey will ask you questions about yourself and contact information that takes about 10 minutes.
Learn More
For more details on all of our clinical trials visit the SOM Clinical Trial Database or ClinicalTrials.gov
What is a clinical trial?
- Clinical trials are research studies that explore whether a medical strategy, treatment or device is safe and effective for people. Before people are given a new intervention (drug, device, or treatment), it is carefully studied in the laboratory or in animals. Studies with the most promising results are then moved into clinical trials with people. Clinical trials are used to evaluate new and better ways to treat, prevent, detect, diagnose, and manage symptoms of diseases.
Why Participate?
- By participating in clinical research, you help medical science by providing valuable information about treatments and prevention of disease. Most treatments we use today are the result of past clinical trials. One way to gain access to promising new interventions is to participate in a clinical trial while also helping science.
Eligibility
- Researchers follow clinical trials guidelines when deciding who can participate in a study. These guidelines are called Inclusion/Exclusion Criteria. These criteria are based on factors such as age, gender, the type and stage of a disease, treatment history, and other medical conditions. Trial coordinators can provide additional information about individual trials.
What are the benefits and risks of participating in a clinical trial?
- By being in a clinical trial you help advance medicine and help future patients. You may also have access to promising treatments by joining a clinical trial. However, all clinical trials have risks. There may be side effects or other risks, which may or may not be worse than those from regular care. The intervention (drug, device, treatment) may not work for you, even if it helps others and you may get a placebo. Joining a research trial may be more burdensome due to more frequent visits. However, more visits may give you more attention and monitoring which you would not normally get by your regular doctor.
Are the research staff vaccinated against Covid-19?
- Yes. All staff are compliant with mandates set forth by the state and Stanford University unless the staff have a valid exception
Are research staff wearing protective equipment?
- Yes. All research staff are required to wear masks and may have additional requirements based upon each building protocol. All buildings for clinical care such as our Stanford Neuroscience Health Center (SNHC) have requirements for Health Screening, physical distancing, and masks.
Are there safety protocols required due to the Covid-19 pandemic?
- Yes. Stanford University has safety requirement protocols and some buildings locations have additional protocols as mandated by Stanford Healthcare.
Are there safety protocols for visitors arriving to buildings?
- Yes. There are requirements for masks for all visitors. There may be additional requirements based upon Stanford University, county, and state mandates. All research participants are required be tested for covid-19 within 72 hours of arriving to a University building or are required to be vaccinated against covid-19. All visitors are required to answer health screenings before arriving. This may be subject to change according to local and state mandates.
The Experimental Subject's Bill of Rights include the basic required elements of informed consent and the Experimental Subject's Bill of Rights (California Law).
These documents do not contain study specific information, but state what will be explained to the participant about the specific study by the interpreter (e.g. key information, purpose of the research, expected duration, procedures, risks, benefits, alternatives [if any], confidentiality of information, compensation [if any] for research-related injuries, whom to contact for questions about the research and research subjects' rights, that participation is voluntary, future use of the participants' information or biological specimens, and whether information about the research will be submitted for inclusion in a clinical trial registry).
Clinical Trials
Central Neuroscience Trials Group
Our Central Neuroscience Trials Group provides professional clinical trial staff for Investigators from both Neurology and Neurosurgery Departments, including the divisions of NeuroImmunology, Headache, Autonomic Disorders, Epilepsy, Memory disorders, and Neuroradiology. Our team supports over 100 observational and interventional, industry sponsored, and investigator-initiated projects. Our goal is to facilitate the timely, safe, and successful implementation of Neuroscience clinical research.
Healthy Brain Aging Study
This is a longitudinal study enrolling individuals with Alzheimer’s disease, Mild Cognitive Impairment, Lewy Body Dementia, Parkinson’s disease, Parkinson’s disease with dementia and healthy volunteers. It is funded by the National Institute of Health and is part of the Alzheimer’s Disease Research Center (ADRC). This study will collect data including medical history, family history and medication. There will be questionnaires to be filled out, cognitive testing and PET/MRI of the brain. This study will follow participants over time.
Status: Active recruiting
PI: Victor Henderson, MD
Status: Open, enrollment ongoing
Contact: Veronica Ramirez
vramirez1@stanford.edu
(650) 721-2409
Natural History Study of Synucleinopathies
PI: Mitchell Miglis, MD
Study Status: Open to enrollment
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu
A novel noninvasive thermoregulatory device for postural tachycardia syndrome
PI: Mitchell Miglis, MD
Study Status: Closed to enrollment
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu
Autonomic Complications of Post-Acute COVID Syndrome
PI: Mitchell Miglis, MD
Study Status: Open to enrollment
Research Coordinator: Jordan Seliger or Adele Viviani
Contact: jseliger@stanford.edu or aviviani@stanford.edu
A phase 2 randomized, double-blinded, placebo-controlled study to evaluate the efficacy and safety of efgartigimod IV in adult patients with post–COVID-19 postural orthostatic tachycardia syndrome (POTS)
PI: Mitchell Miglis, MD
Study Status: Closed to enrollment
Research Coordinator: Jannika Machnik
Contact: jmachnik@stanford.edu
Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Efgartigimod in Adult Patients with Post-COVID-19 Postural Orthostatic Tachycardia Syndrome (PC-POTS) who Completed Study ARGX-113-2104
PI: Mitchell Miglis, MD
Study Status: Closed to enrollment
Research Coordinator: Jannika Machnik
Contact: jmachnik@stanford.edu
TTR Amyloidosis Prevalence And Phenotype (TTRAPP Study)
PI: Safwan Jaradeh, MD
Study Status: Open to enrollment
Research Coordinator: Jannika Machnik
Contact: jmachnik@stanford.edu
North American Prodromal Synucleinopathy Stage 2 (NAPS2) Consortium
PI: Mitchell Miglis, MD
Study Status: Open to enrollment
Research Coordinator: Adele Viviani
Contact: aviviani@stanford.edu
Autonomic Complications of PASC (RECOVER)
PI: Mitchell Miglis, MD
Study Status: Open to enrollment
Research Coordinator: Adele Viviani
Contact: aviviani@stanford.edu
Open-label prospective study for immune therapies in autoimmune gastrointestinal dysmotility (AGID)
PI: Dong-In Sinn, MD
Study Status: Open to enrollment
Research Coordinator: Jannika Machnik or Jordan Seliger
Contact: jmachnik@stanford.edu or jseliger@stanford.edu
A Phase 3, Multi-center, Randomized Withdrawal and Long-Term Extension Study of Ampreloxetine for the Treatment of Symptomatic Neurogenic Orthostatic Hypotension in Participants with Multiple System Atrophy
PI: Dong-In Sinn, MD
Study Status: Open to enrollment
Research Coordinator: Jordan Seliger or Adele Viviani
Contact: jseliger@stanford.edu or aviviani@stanford.edu
Syn-Sleep Study
PI: Mitchell Miglis, MD
Study Status: Open to enrollment
Research Coordinator: Adele Viviani
Contact: aviviani@stanford.edu
A Phase 2, double-blind, placebo-controlled, parallel-group study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and potential efficacy of multiple doses of ONO-2808 in patients with Multiple System Atrophy (MSA)
PI: Mitchell Miglis, MD
Study Status: Enrolling Soon
Research Coordinator: Jordan Seliger or Adele Viviani
Contact: jseliger@stanford.edu or aviviani@stanford.edu
A First-in-human Study of Inhibitory Interneurons (NRTX-1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)
PI: Kevin Graber, MD
NCT05135091
Study Status: Enrolling
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu
Medtronic Deep Brain Stimulation (DBS) Therapy for Epilepsy Post-Approval Study (EPAS)
PI: Kevin Graber, MD
NCT03900468
Study Status: Enrolling
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu
Maternal Outcomes and Neurodevelopmental Effects of Antiepileptic Drugs (MONEAD)
PI: Kimford Meador, MD
NCT01730170
Study Status: Closed to Enrollment
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu
Electrophysiological Biomarkers of Consciousness in Patients with Epilepsy and Patients with Psychogenic Non-Epileptic Spells
PI: Kimford Meador, MD
Study Status: Enrolling
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu
Electroencephalographic Biomarkers of Neuro-Cognitive Function
PI: Kimford Meador, MD
Study Status: Enrolling
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu
Using Artificial Intelligence to Choose the Optimal Antiepileptic Drug
Protocol ID : 46059
PI: Robert S. Fisher, MD, PhD
Status: Enrolling subjects
Transcranial Direct Current Stimulation to Treat Epilepsy
Protocol ID : 47711
PI: Robert S. Fisher, MD, PhD
Status: Enrolling subjects
Focused ultrasound to treat epilepsy (EP001)
Protocol ID : 55981
PI: Robert S. Fisher, MD, PhD
Status: Enrolling subjects
AI-Guided 3D Video Analysis for Seizure Detection
Protocol ID : 53035
PI: Robert S. Fisher, MD, PhD
Status: Pending loosening of COVID travel requirements
Cognitive Effects of Vinpocetine in Patients with Epilepsy
PI: Kimford Meador, MD
NCT02011971
Study Status: Closed to Enrollment
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu
Effects of Titration Rate on Cognitive and Behavioral Side Effects of Perampanel
PI: Kimford Meador, MD
NCT04417907
Study Status: Closed to Enrollment
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu
RNS® System Post-Approval Study in Epilepsy Clinical Investigational Plan (CIP)
PI: Babak Razavi
NCT02403843
Study Status: Closed to enrollment
Research Coordinator: Jordan Seliger, Adele Viviani
Contact: jseliger@stanford.edu or aviviani@stanford.edu
Personal Impact of Epilepsy Scale (PIES): Determining the Minimally Important Change
Protocol ID : 43107
PI: Robert S. Fisher, MD, PhD
Status: Closed, in data analysis phase
Evaluation of the 24/7 EEG SubQ System for Ultra Long-Term Recording of Patients with Temporal Lobe Epilepsy
PI: Babak Razavi
NCT04526418
Study Status: Enrolling Soon
Research Coordinator: Jordan Seliger, Adele Viviani
Contact: jseliger@stanford.edu or aviviani@stanford.edu
Open-label, Multi-center, Phase 1/2a Observational, First-in-human (FIH) Study Investigating the Safety and Efficacy of AMT-260 in Adult Subjects with Refractory Mesial Temporal Lobe Epilepsy (MTLE) in the Non-Dominant Hemisphere Administered via Magnetic Resonance Imaging (MRI) guided Convection-enhanced Delivery (CED) (CT-AMT-260-01)
PI: Yi Li
NCT0603850
Study Status: Enrolling Soon
Research Coordinator: Jordan Seliger, Adele Viviani
Contact: jseliger@stanford.edu or aviviani@stanford.edu
A Phase 2/3 Multicenter, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy, Safety and Tolerability of BHV-7000 in Subjects with Refractory Focal Onset Epilepsy
PI: Yi Li
NCT06132893
Study Status: Enrolling Soon
Research Coordinator: Jordan Seliger, Adele Viviani
Contact: jseliger@stanford.edu or aviviani@stanford.edu
Interventional, randomized, double-blind, parallel-group, placebo-controlled delayed-start study to evaluate the efficacy and safety of eptinezumab in patients with episodic Cluster Headache (Alleviate)
PI: Addie Peretz, MD
NCT04688775
Study Status: Enrolling
Contact: neurotrials@stanford.edu
Sunstar: The Stanford Neuroscience Headache Biorepository
PI: Robert Cowan, MD, FAAN
NCT03231241
Study Status: Enrolling
Contact: neurotrials@stanford.edu
Efficacy of ‘Green Glasses’ to Reduce Headache Symptoms in Individuals with Migraine Disorders
PI: Robert Cowan, MD, FAAN
Study Status: Not Yet Enrolling
A prospective, multi-centre, randomized, double-blind, sham-controlled, parallel-group, group-sequential study to investigate safety and effectiveness of the Rehaler partial rebreathing device, in adults suffering from migraine with aura: PAREMA1
PI: Robert Cowan, MD, FAAN
Study Status: Not Yet Enrolling
Stanford Memory Disorders Clinical Trials
For patient referrals, please reach out to:
Active, Open for Enrollment
Active, Open for Enrollment
Lewy Bodies Dementia Trials
Cognition Trial (SHIMMER)
Sponsor: Cognition Therapeutics Inc.
Intervention: CT1812
Indication: Mild or moderate Dementia with Lewy Bodies
Age: 50-85 years old
Brief Summary: A Randomized, Double-blind, Placebo-controlled, Phase 2, 6-month Study to Evaluate the Safety, Tolerability and Exploratory Efficacy of CT1812 in Subjects with Mild to Moderate Dementia with Lewy Bodies
Clinicaltrials.gov identifier: NCT05225415
PI: Sharon Sha, MD
Contact: Stephanie Tran
Who can participate
- Subjects must have a caregiver/ study partner
- Men or women 50-85 years of age (inclusive), meeting criteria for probable DLB .
- MMSE 18-27 inclusive.
- If receiving acetylcholinesterase inhibitors (AChEI), memantine or a combination of the two, must have been on a stable dose for at least 12 weeks before the screening visit.
EIP Pharma DLB Phase 2bTrial
Sponsor: EIP Pharma,Inc.
Intervention: Neflamapimod
Indication: Dementia with Lewy Bodies
Age: ≥55 years
Brief Summary: A Phase 2b Clinical Study of the P38 Alpha Kinase Inhibitor Neflamapimod in Patients with Dementia with Lewy Bodies (DLB)
Clinicaltrials.gov identifier: NCT04001517
PI: Sharon Sha, MD
Contact: Kaila Sevilla
Who can Participate:
- Men and women aged ≥55 years.
- Subject or subject’s legally authorized representative is willing and able to provide written informed consent.
- Probable DLB by consensus criteria (McKeith et al, 2017), including a positive DaTscan™. If the DaTscan is negative, but the subject has historical polysomnography (PSG)-verified REM sleep behavioral disorder (RBD), this will also qualify as probable DLB.
- CDR Global Score 0.5 or 1.0 during Screening.
- If the patient is currently receiving cholinesterase inhibitor therapy, the patient must have received such therapy for greater than 3 months and on a stable dose for at least 6 weeks at the time of randomization. Except for reducing the dose for tolerability reasons, the dose of cholinesterase inhibitor may not be modified during the study. If the patient is not currently receiving cholinesterase inhibitor therapy, but received such therapy previously, that therapy must have been discontinued at least 3 months prior to randomization. Memantine therapy is allowed, if it had been started at least 3 months prior to randomization and the patients is also receiving cholinesterase inhibitor therapy (memantine monotherapy, i.e., without concomitant cholinesterase inhibitor therapy, is excluded).
- Normal or corrected eyesight and auditory abilities, sufficient to perform all aspects of the cognitive and functional assessments.
- No history of learning difficulties that may interfere with their ability to complete the cognitive tests.
- Received vaccination for SARS-CoV-19, unless medical contraindications prevent being vaccinated. 9. Must have reliable informant or caregiver.
Alzheimer’s Dementia Trials
CELIA Trial
Sponsor: Biogen MA, Inc.
Intervention: BIIB080
Indication:MCI and Dementia
Age: 50-80 Years old
Brief Summary: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, and Tolerability of BIIB080 in Subjects with Mild Cognitive Impairment Due to Alzheimer’s Disease or Mild Alzheimer’s Disease Dementia
Clinicaltrials.gov identifier: NCT05399888
PI: Irina Skylar-Scott, MD
Contact: Olivia Lu
Who can participate:
- Subjects must have a caregiver/ study partner.
- Male and females aged 50 to 80 years, inclusive, at the time of informed consent.
- Must meet all of the clinical criteria for MCI due to AD or mild AD dementia and must have the following at Screening Visit 1:
- RBANS Delayed Memory Index score of ≤ 85, indicative of objective evidence of memory impairment.
- CDR global score of 0.5 for MCI due to AD or 0.5 or 1 for mild AD dementia.
- MMSE score of 22 to 30 (inclusive).
- CDR Memory Box score of ≥ 0.5.
- Evidence of amyloid pathology as measured by PET or CSF sampling.
- Must consent to APOE genotyping. The participant’s APOE status may be disclosed to him/her at the Investigator’s discretion.
ARI-Bio Trial (Polaris-AD)
Sponsor: AriBio Co., Ltd.
Intervention: AR1001
Indication: Early Alzheimer’s Disease
Age: ≥55 years
Brief Summary: A Phase 3 Double-blind, Randomized, Placebo-controlled Trial to Evaluate the Efficacy and Safety of AR1001 in Participants with Early Alzheimer’s Disease
Clinicaltrials.gov identifier: NCT05531526
PI: Sharon Sha, MD
Contact: Kaila Sevilla
Who can Participate:
- Male or female participants aged 55 to 90 years of age inclusive at the time of signing the informed consent form
- Mild cognitive impairment or mild dementia consistent with AD defined by stages 3 to 4 according to the National Institute on Aging and Alzheimer’s Association (NIA-AA) (Jack et al., 2018) at Screening
- Participants with a history of subjective cognitive and memory decline with onset within 5 years before Screening, confirmed by study partner.
- Participants who have a MMSE score greater than or equal to 20
- Participants with a CDR global rating of 0.5 or 1
- Participants with a RBANS score based on the Delayed Memory Index (DMI) score less than or equal to 85
- If an historic magnetic resonance imaging (MRI) is available, findings must exclude other causes of dementia
- ositive biomarker for brain amyloid pathology as indicated by assessment of at least one of the following: a. Current or historic CSF with Lumipulseâ beta-amyloid ratio [1-42/1-40] ≤ 0.072 or Elecsysâ p-tau 181/Aβ[1-42] ≤ 0.028 b. Historic amyloid positron emission tomography (PET) assessment of imaging agent uptake into brain is acceptable to determine eligibility (PET confirmed by central read).
- Participants (or participant's legally authorized representative) and caregiver(s) who can sign an informed consent to participate in the study
- Participants who have one (or more) identified adult study partner(s) who, in the opinion of the Investigator, has sufficient contact with and knowledge about the participant as to be able to report knowledgably about the participant’s cognition, function, behavior, and safety, and compliance with the protocol. The informant/care partner must be available by phone to provide information to the Investigator and study staff about the participant as well as agree to attend in-person clinic visits that require partner input for scale completion. The informant/care partner must be literate and provide informed consent and should be available for the duration of the study. The same informant/care partner is required to be consistent across all study visits except under rare, unavoidable circumstances (e.g., unexpected informant health crisis) that are approved by the Investigator and Sponsor.
LEADS Trial
Sponsor: Indiana University and NIA (LEADS)
Intervention: N/A - Observational
Indication: Early onset Alzheimer's Disease (EOAD), Early onset non-Alzheimer's Disease (EO-nonAD), and cognitively normal (CN)
Age: 40-64 years old
Brief Summary: A natural history, non-treatment study designed to look at disease progression in individuals with early onset cognitive impairment (less than 65 year old).
Clinicaltrials.gov identifier: NCT03507257
PI: Sharon Sha, MD
Contact: Stephanie Tran
Website: https://leads-study.medicine.iu.edu/about/leads-lectures-webinars/
Who can participate
Cognitively impaired (EOAD and EOnonAD) Cohorts Only:
- Meets NIA-AA criteria for MCI due to AD or probable AD dementia
- Have a global CDR score ≤ _1.0
- Have capacity to provide informed consent (IC) or has a legal authorized representative or guardian who provides IC
- Age between 40-64 years (inclusive) at the time of consent
- Must have a study partner (informant) who spends a minimum average of 10 hours per week with the participant
- Fluent in English or Spanish
Coming soon in March 2024 Alzheimer’s Dementia Trials
MAP Trial
Sponsor: NIA (Columbia University Primary)
Intervention: Metformin
Indication:MCI and Dementia
Age: 55-90 Years old
Brief Summary: Metformin in Alzheimer’s dementia Prevention
Clinicaltrials.gov identifier: NCT04098666
PI: Irina Skylar-Scott, MD
Contact: Olivia Lu
1. Diagnosis of aMCI:
- Participants must have subjective memory concern reported by participant, study partner, or clinician.
- A mini-mental state exam ≥ 22 for participants with more than 8 years of education. For participants with less than 8 years of education, a MMSE ≥ 20 will be allowed.
- Clinical Dementia Rating = 0.5. The memory box score must be at least 0.5.
- General cognition and functional performance sufficiently preserved such that a diagnosis of Alzheimer’s disease cannot be made by the site physician at the time of the screening visit.
- Abnormal memory function documented by scoring within the education adjusted ranges on the Logical Memory II subscale from the Wechsler Memory Scale-Revised.
2. For early MCI: 9-11 for 16 or more years of education
- 5-9 for 8-15 years of education
- 3-6 for 0-7 years of education
3. For late MCI
- ≤ 8 for 16 or more years of education
- ≤ 4 for 8-15 years of education
- ≤ 2 for 0-7 years of education
4. Age range: 55 years to 90 years.
5. Sex distribution: all eligible men and women will be included, and no one will be excluded because of gender.
6. Languages: fluent in English or Spanish. We have reliable, well-validated Spanish tests for all outcome measures.
7. Participants without a known history of diabetes. If diabetes is diagnosed during screening (hemoglobin A1c [HbA1c] of 6.5% or greater) participants will be excluded. The main justification for this exclusion is the potential for these participants to be placed on other diabetes medications that may confound our study.
8. General cognition and functional performance such that a diagnosis of dementia cannot be made at the time of screening based on DSM-V criteria.
9. Vision and hearing must be sufficient for compliance with testing procedures.
10. Must have a study partner to come to all appointments or be available by telephone at follow-up visits.
Study Partner Inclusion Criteria
- The study partner can provide an independent evaluation of functioning for a person enrolled in the MAP study as a participant.
- The study partner agrees to attend study visits with the MAP participant or be available by telephone.
ALZ-NET Trial
Sponsor: Alzheimer’s Association
Intervention: N/A observational trial. ALZ-NET will collect longitudinal clinical and safety data for enrolled patients treated with novel FDA approved AD therapies.
Indication: Alzheimer’s Dementia or other dementia
Age: ≥18 years
Brief Summary: Patient registry for Alzheimer's Network for Treatment and Diagnostics (ALZ-NET)
Clinicaltrials.gov identifier: N/A
PI:Kyan Younes, MD
Contact: Kaila Sevilla
Who can participate:
- Patient or patient’s legally authorized representative (LAR) or proxy (e.g., spouse or legal guardian) has the ability to understand the purpose and risks of ALZ-NET and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local patient privacy regulations.
- Patient is at least 18 years of age at the time of informed consent.
- Patient has memory concerns and/or may have diagnosis of Alzheimer’s disease (AD) and has been identified by an approved site investigator (as defined by protocol) to be appropriate for treatment with novel FDA-approved AD therapies in real world clinical practice.
- Patient meets appropriate label designations according to appropriate use recommendations for novel FDA-approved AD therapy/therapies.
- Patient’s treating clinician has made the decision to treat the patient with novel FDA-approved therapy for AD independent of the purpose of ALZ-NET and has already or will be initiating treatment.
Bumetanide AD Trial
Sponsor: Knight Initiative Brain Resilience Initiative Translational Grant, Stanford University
Intervention: Bumetanide
Indication: Mild Cognitive impairment or mild dementia due to Alzheimer’s
Brief Summary: A Phase IIa, Randomized, Double-Blind, Active Placebo-Controlled, Parallel Group study to Evaluate the Safety and Tolerability of Bumetanide in patients with Alzheimer’s Disease
Clinicaltrials.gov identifier: NCT06052163.
PI: Kyan Younes,MD
Contact: Mina Kmiecik
Who can participate:
1. Ages 50-85 years, inclusive.
2. Diagnosis of either mild cognitive impairment due to Alzheimer`s disease or mild dementia due to Alzheimer`s disease with evidence of the AD pathophysiological process according to National Institute on Aging and Alzheimer’s Association (NIA-AA) criteria. Global CDR score of 0.5 (for MCI due to AD) and 0.5 to 1 (for mild AD).
Operational Diagnostic Process Inclusion Criteria for AD Subjects:
- Gradual and progressive change in memory function for ≥ 6 months reported by subjects or study partner.
- Screening MMSE score 18-30 inclusive.
- Evidence of AD pathological process, as confirmed on amyloid PET, plasma ptau-181, or cerebrospinal fluid amyloid-beta/phosphorylated tau index.
- Willingness and ability to complete all aspects of the study including assessments, neuropsychological testing, and MRI.
- AD medication is planned to remain stable throughout.
3. Persons with female biological sex, must be of non-childbearing potential. Persons with male biological sex who are sexually active must agree to use acceptable contraceptives during the trial and for 3 months after their last dose unless their partner is using an acceptable means of birth control or is of non-childbearing potential.
4. Persons with male biological sex who are sexually active with a female of child-bearing potential must agree to use condoms during the trial and for 3 months after the last dose unless the female is using an acceptable means of birth control. Acceptable forms of birth control include abstinence, birth control pills, or any double combination of intrauterine device (IUD), male or female condoms, diaphragm, sponge, and cervical cap.
5. Ability to swallow bumetanide tablet.
6. Plasma amyloid or tau consistent with Alzheimer’s disease.
7. Neuroimaging (MRI) obtained during screening or historic within a year of enrolment consistent with the clinical diagnosis of Alzheimer’s disease.
8. Stable dose of permitted medications for 30 days prior to screening or 60 days prior to screening for medications that affect cognitive function.
9. Resides at home or in the community (assisted living acceptable).
10. In the opinion of the PI, has a study partner able and willing to provide accurate information about the participant, oversee the administration of study drug, and participate in study visits and informant-based assessments (usually requires at least 5 hours of contact per week).
11. As assessed by the PI, participant is likely to be able to comply with the protocol, including completion of all screening evaluations, and has adequate vision, hearing (hearing aid permitted), and literacy in English sufficient for compliance with required testing procedures.
Cognitively Normal patient population trials
AHEAD 3-45 Trial
Sponsor: Eisai and NIH (AHEAD 3-45 Study)
Intervention: BAN2401 (monoclonal antibody binding to amyloid)
Indication: Healthy individuals with normal cognition at risk for Alzheimer’s disease
Age: 55-80 years old
Brief Summary: A Placebo-Controlled, Double-Blind, Parallel-Treatment Arm, 216 Week Study to Evaluate Efficacy and Safety of Treatment With BAN2401 in Subjects With Preclinical Alzheimer’s Disease and Elevated Amyloid (A45 Trial) and in Subjects With Early Preclinical Alzheimer’s Disease and Intermediate Amyloid (A3 Trial)
Clinicaltrials.gov identifier: NCT04468659
PI: Sharon Sha, MD
Contact: Jade Perry
Study Link: https://studypages.com/s/the-ahead-study-440395/
Who can participate
1. Male or female, age 55 to 80 years inclusive at the time of informed consent.
a. Those 55 to 64 must have 1 of the following additional risk factors:
i. First degree relative diagnosed with dementia onset before age 80, or
ii. Known to possess at least 1 apolipoprotein є4 variant (APOE4) allele, or
iii. Known before Screening to have elevated brain amyloid according to previous PET or CSF testing. Individuals with historical amyloid PET scans with Aβi (eg, from preclinical AD studies such as A4 or EARLY) are eligible to be screened, provided the subject did not participate in any clinical studies involving anti-amyloid therapies subsequent to the PET assessment.
2. Global CDR score of 0 at Screening
3. Mini Mental State Examination (MMSE) score ≥27 (with educational adjustments) at Screening
4. Wechsler Memory Scale-Revised Logical Memory subscale II (WMS-R LM II) score at Screening of ≥6
5. A45 Trial: Elevated brain amyloid pathology by amyloid PET: defined as approximately >40 centiloids on Screening scan.
A3 Trial: Intermediate levels of brain amyloid pathology by amyloid PET: defined as approximately 20 to 40 centiloids on screening scan.
6. Has a study partner that is willing to participate as a source of information and has approximately weekly contact with the participant (contact can be in-person, via telephone or electronic communication).
Active, not recruiting
Sponsor: American College of Radiology (New IDEAS)
Intervention: N/A - Observational
Indication: MCI and dementia
Brief Summary: New IDEAS: Imaging Dementia—Evidence for Amyloid Scanning Study
A Study to Improve Precision in Amyloid PET Coverage and Patient Care
Clinicaltrials.gov identifier: NCT02420756
PI: Sharon Sha, MD
Contact: Anthony Velasquez
Sponsor: Janssen Research & Development (Autonomy Study)
Intervention: JNJ-63733657 (monoclonal anti-tau antibody)
Indication: mild cognitive impairment and mild AD
Age: 55-80 years old
Brief Summary: A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicenter Study to Assess the Efficacy and Safety of JNJ-63733657, an Anti-tau Monoclonal Antibody, in Participants with Early Alzheimer’s Disease
Clinicaltrials.gov identifier: NCT04619420
PI: Sharon Sha, MD
Contact: Kaila Sevilla
Sponsor: Eli Lilly (Trailblazer Study)
Intervention: Donanemab (monoclonal antibody binding to amyloid)
Indication: early symptomatic AD.
Protocol NCT#: NCT04437511
PI: Sharon Sha, MD
Contact:Jade Perry
Sponsor: Eisai (Clarity AD)
Intervention: BAN2401 (monoclonal antibody binding to amyloid)
Indication: MCI/Mild AD
Clinicaltrials.gov identifier: NCT03887455
PI: Sharon Sha, MD
Contact: Olivia Lu
PARKINSON'S DISEASE
The Role of the Gut Microbiome in Parkinson’s Disease
Studies show that the gut microbiome in Parkinson’s patients is different from the gut microbiome in healthy individuals. What is not clear, however, is how these differences impact one’s risk for developing Parkinson’s Disease or how the different microbiome in Parkinson’s patients impacts disease course. Additionally, in Parkinson’s patients there is chronic systemic inflammation due to excessive immune activity, and the gut microbiome is known to impact immune functioning. Therefore, in this study we examine whether the differences in the microbiome of Parkinson’s patients are associated with increased inflammatory markers in the blood, and whether the differences in microbiome impact immune cell activation. Through this study, we hope to better understand the mechanism by which the microbiome in Parkinson’s patients may be contributing to the disease. In the future, these findings could be used to develop disease treatments that target the microbiome. We are recruiting individuals who have been diagnosed by a neurologist with Parkinson’s Disease and healthy individuals from the same house. Participation entails answering an online questionnaire and giving small amounts of blood and stool. Each participant will receive a gift card for their involvement.
PI: Bianca Palushaj, MD
Co-PI: Ami S. Bhatt, MD, PhD
Study status: Open, enrollment ongoing
Research Coordinators: Meena Chakraborty, Erin Brooks, Gabriella Green
Contact: mchakra@stanford.edu, efbrooks@stanford.edu, gzmg1@stanford.edu
Blood biomarkers study in REM sleep behavior disorder (RBD)
Purpose: studying neuroinflammation and immune protective factors in patients with RBD
PI: Emmanuel H. During, MD
Co-PI: Emmanuel Mignot, MD, PhD
Study status: Open, enrollment ongoing
Research Coordinator: Ana Cahuas
acahuas@stanford.edu 650 721 5489
Home diagnosis of REM sleep behavior disorder (RBD) using wearable sleep trackers
Purpose: developing a machine learning method to diagnose RBD in the home environment with wrist-worn actigraphy
PI: Emmanuel H. During, MD
Study status: Open, enrollment ongoing
Research Coordinator: Ana Cahuas
acahuas@stanford.edu 650 721 5489
U19 North American Prodromal Synucleinopathy (NAPS) consortium
Purpose: Neuroprotection trial planning in REM sleep behavior disorder (RBD)
PI: Emmanuel H. During, MD
Study status: Open, enrollment ongoing
Research Coordinator: Vincent Nguyen
vnguyen9@stanford.edu
Sodium Oxybate in Refractory REM Sleep Behavior Disorder: A Randomized Controlled Study
Purpose: This study evaluates the efficacy of sodium oxybate in individuals with and without Parkinson’s disease who act out their dreams (REM Sleep Behavior Disorder, known as “RBD”) and do not respond to usual therapies.
PI: Mitchell Miglis, MD
Study status: Open, enrollment ongoing
Research Coordinator: Ana Cahuas
acahuas@stanford.edu 650 721 5489
Pacific Udall Center
Sponsor: NIH/NINDS Morris K. Udall Center of Excellence for Parkinson's Disease Research
PI: Tom Montine, MD, PhD
Study status: Open, Enrollment ongoing
Research coordinator: Hannah Schmitz
hschmitz@stanford.edu
Healthy Brain Aging Study
Sponsor: NIH/NIA
PI:Victor Henderson, MD and Tony Wyss-Coray, PhD
Study Status: Open, enrollment ongoing
Research coordinator: Christina Wyss-Coray
ADRCstanford@stanford.edu (650) 721-2409
Bilateral Closed Loop Deep Brain Stimulation for Freezing of Gait using Neural and Kinematic Feedback (NCT04043403)
Sponsor(s): NIH/NINDS, BRAIN Initiative
Purpose: This study aims to investigate the safety and feasibility of adaptive deep brain stimulation for impaired gait and freezing of gait in Parkinson’s disease, driven by subject-specific neural or behavioral control variables, and in response to medication.
PI: Helen Bronte-Stewart, MD, MS
Study status: Open, enrollment ongoing
Research coordinator: Sudeep Aditham
sudeepa@stanford.edu (650) 723-6709
The Natural History of Synucleinopathies
PI: Mitchell Miglis, MD
Study status: Open, enrollment by invitation only
Research coordinator: Jordan Seliger
jseliger@gmail.com
ATYPICAL PARKINSONISM (MSA, PSP, CBD, DLB)
M-STAR
Sponsor: Biohaven
Site PI: Mitchell Miglis, MD
Study status: Closed for enrollment
Research coordinator: Ruba Shaik
rubas@stanford.edu 650-546-1436
HUNTINGTON’S DISEASE
Generation HD
Clinical Trials Protocol#: NCT03761849
Sponsor: Roche
Site PI: Jacinda Sampson, MD, PhD
Study status: Enrollment Closed, trial ongoing
Research coordinator: Stephanie Tran
trans@stanford.edu
Upcoming Movement Clinical Trials
PARKINSON’S DISEASE
ADX48621 for the Treatment of Levodopa Induced Dyskinesia in Patients With Parkinson's Disease
Clinical Trials Protocol#: NCT01336088
Sponsor: Addex Pharmaceuticals
Study status: Pending, open to enrollment Fall/Winter 2020
Research coordinator: Lila Perrone
perronel@stanford.edu
Open-Label Study With Pimavanserin on Activities of Daily Living in Subjects With Parkinson's Disease Psychosis
Clinical Trials Protocol#: NCT04292223
Sponsor: Acadia
Site PI: Sharon Sha, MD, MS
Study status: Pending, open to enrollment Fall/Winter 2020
Research coordinator: Lila Perrone
perronel@stanford.edu
Project BIG: The Stanford Brain Immune Gut Research Initiative
PI: Jeffrey Dunn, MD
Research Coordinator: Julia Sumera, jsumera@stanford.edu
Status: Enrolling
A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell ( CAR-T) Therapy in Subjects With Non-relapsing and Progressive Forms of Multiple Sclerosis
NCT06138132
PI: Jeffrey Dunn, MD
Research Coordinator: Crystal Ton-Nu, ctonnu@stanford.edu
Status: Enrolling
A Multicenter Randomized Controlled Trial of Best Available Therapy versus Autologous Hematopoietic Stem Cell Transplant for Treatment-Resistant Relapsing Multiple Sclerosis
NCT04047628
PI: Jeffrey Dunn, MD
Research Coordinator: Crystal Ton-Nu, ctonnu@stanford.edu
Status: Enrolling
A Multiple-center, Non-Randomized, Open-Label, Adaptive, Single Ascending Dose Phase I Study to Investigate the Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of RO7121932 Following Intravenous Administration in Patients with Multiple Sclerosis
NCT05704361
PI: Christopher Lock, MD
Research Coordinator: Safiyyah Bachar, sbachar@stanford.edu
Status: Enrolling
North American Registry for Care and Research in Multiple Sclerosis (NARCRMS) Study
PI: McDonald Jamie, MD
Research Coordinator: Safiyyah Bachar, sbachar@stanford.edu
Status: Enrolling
Ozanimod (Zeposia) in Multiple Sclerosis and Ulcerative Colitis: High Dimensional Immunometabolomic Analysis of Immune Cell Subsets with CyToF, CITE-seq, and Multiparameter Flow Cytometry In Treated Individuals Compared with Baseline.
PI: Lawrence Steinman MD
Research Coordinator: Julia Sumera, jsumera@stanford.edu
Status: Enrolling
CorEvitas SPHERES (Synergy of Prospective Health & Experimental Research for Emerging Solutions) Registry for Neuromyelitis Optica Spectrum Disorder (NMOSD)
NCT04886492
PI: May Han, MD
Research Coordinator: Tara Sarkar, tarats@stanford.edu
Status: Enrolling
A Randomized, Double-blind, Placebo-Controlled, Multicenter, Phase 3, Pivotal Study with an Open-Label Extension Period to Evaluate the Efficacy and Safety of Rozanlixizumab in Adult Participants with Myelin Oligodentrocyte Glycoprotein (MOG) antibody-associated disease (MOG-AD)
NCT05063162
PI: May Han, MD
Research Coordinator: Tara Sarkar, tarats@stanford.edu
Status: Enrolling
Retinal Biomarkers of Inflammation and Degeneration in Multiple Sclerosis
PI: Heather Moss, MD
Research Coordinator: Tara Sarkar, tarats@stanford.edu
Status: Enrolling
Neuromuscular Clinical Research Group
The Neuromuscular Research Team includes 9 Neurologists, 3 Nurse Practitioners, 3 Clinical Evaluators (Physical Therapists), a Research Administrative Associate, a Research Administrator, a Clinical Research Manager, 14 Clinical Research Coordinators, 4 Assistant Clinical Research Coordinators, a Data Analyst, a Research Scientist, a Life Science Research Professional, and a Post-Doctoral Research Fellow. Our team is involved in both adult and pediatric research. We have about 60-70 research projects at any given time including industry-sponsored clinical trials, observational studies, investigator-initiated studies, and lab-based research projects. Our goal is the translation of research findings into new and more effective clinical interventions to control or cure neuromuscular diseases.
All Neuromuscular Conditions
Subject Database and Specimen Repository for Neuromuscular and Neurodegenerative Disorders
Protocol ID: 23888
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: This is a study that involves collecting clinical information of subjects (patients with any neurological condition or their close family member) and tissue samples to develop a recruitment database and tissue bank, which will be of great value in helping the investigators learn more about various related neurological conditions.
Status: Recruiting
Tissue Banking of Blood, Spinal Fluid or Skin Biopsy for the Research of Neurological Diseases
Protocol ID: 16472
NCT: N/A
PI: Dr. Yuen So
Study Coordinator: Shirley Paulose, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: To collect blood, spinal fluid, or skin biopsy specimens to create a tissue bank for current or future neuroscience research, which would help to learn more about various neurological conditions
Status: Recruiting
MOVR Registry
Protocol ID: 52620
NCT: N/A
PI: Dr. Jacinda Sampson
Study Coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Muscular Dystrophy Association
Purpose: Observational study of neuromuscular conditions including ALS, BMD/DMD, FSHD, LGMD, Pompe and SMA
Status: Recruiting soon in clinic
Determination of Standards for Maximal and Submaximal Exercise testing for Individuals with Neuromuscular Disease (CPET)
Protocol ID: 54078
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Sabrina Salvatore, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Understanding exercise testing which will assess how heart and lungs work together to get oxygen to muscles in children and adults with muscular dystrophies
Status: Recruiting
Recruitment Notes: All clinic patients ages 9+ with diagnosed neuromuscular disorders who are able to perform testing on cycle ergometer as well as controls
Amyotrophic Lateral Sclerosis (ALS)
A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB105 Administered Intrathecally to Adults With Amyotrophic Lateral Sclerosis With or Without Poly-CAG Expansion in the Ataxin-2 Gene
Protocol ID: 56187
NCT04494256
PI: Dr. John W. Day
Study Coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Assessing the safety and tolerability of BIIB105 in adults with ALS
Status: Active, not recruiting
Recruitment Notes: Sporadic ALS with or without ATX2 gene mutation
Clinical Procedures to Support Research in ALS (CAPTURE-ALS)
Protocol ID: 54467
NCT03489278
PI: Dr. Yuen So
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NINDS/NIH, MDA
Purpose: Natural History Study of ALS and PLS
Status: Recruiting in clinic
A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients With Fused in Sarcoma Mutations (FUSION)
Protocol ID: 59619
NCT04768972
PI: Dr. John W. Day
Study Coordinator: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: IONIS Pharmaceuticals
Purpose: Assessing the safety and efficacy of ION363 in children with ALS
Status: Recruiting
Recruitment Notes: Confirmed genetic mutation in FUS
Expanded Access Program to ION363 FUS ALS treatment
Protocol ID: N/A
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Expanded Access Program
Status: Recruiting on case-by-case basis
Recruitment Notes:
Becker and Duchenne muscular dystrophy (BMD/DMD)
A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents with Becker Muscular Dystrophy (GRAND CANYON)
Protocol ID: 72069
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Edgewise Therapeutics
Purpose: Assessing the safety and efficacy of EDG-5506-201 in adults with BMD
Status: Recruiting soon
Recruitment Notes: Ambulatory adults with BMD ages 18-50
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (ENVISION)
Protocol ID: 64248
NCT05881408
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Sarepta Therapeutics, Inc
Purpose: Assessing the safety and efficacy of SRP-9001 gene therapy in individuals with DMD
Status: Active, not recruiting
Long-term follow-up study for all SRP-9001 studies (EXPEDITION)
Protocol ID: 71344
NCT05967351
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Lesly Welsh, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Sarepta Therapeutics, Inc
Purpose: Long term follow-up study of safety and efficacy of SRP-9001
Status: Active, recruiting by invitation only
Recruitment Notes: Requires enrollment in previous Sarepta SRP-9001 trials
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping (EXPLORE44)
Protocol ID: 69829
NCT05670730
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the safety and efficacy of AOC1044 in DMD
Status: Recruiting
Recruitment Notes: Individuals with DMD mutations amenable to exon 44 skipping, ages 7-27
Charcot-Marie Tooth Disease (CMT)
Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Type (CMT1B), 2A (CMT2A), 4A (CMT4A), 4C (CMT4C), and Others
Protocol ID: 23094
NCT01193075
PI: Dr. Maxwell Greene
Study Coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NIH and MDA
Purpose: Natural history study of CMT
Status: Recruiting in clinic
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J (CMT4J)
Protocol ID: 72459
NCT06151600
PI: Dr. John W. Day
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Elpida Therapeutics
Purpose: Natural history study of CMT4J
Status: Recruiting soon
Cystinosis myopathy
The Effect of Exercise on Muscle Dysfunction in Cystinosis
Protocol ID: 46941
NCT04071548
PI: Dr. Richard Reimer and Dr. Tina Duong
Study Coordinator: NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Cystinosis Myopathy Foundation
Purpose: Assessing the effect of a short, high intensity interval training exercise program on individuals with cystinosis myopathy
Status: Recruiting
Recruitment Notes: Adults ≥18 years diagnosed with cystinosis myopathy
Myotonic dystrophy (DM)
Outcome validation with functional measures in myotonic dystrophy (MYOCAP/Actimyo)
Protocol ID: 62522
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Outcome validation with functional measures in myotonic dystrophy
Status: Recruiting
Stanford brain biomarkers study
Protocol ID: TBD
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: TBD, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Characterization of neurological symptoms and biomarkers of DM1
Status: Recruiting soon
Recruitment Notes: Adults with DM1 and their unaffected family members
Avidity HARBOR trial
Protocol ID: 74759
NCT: TBD
PI: Dr. John W. Day
Study Coordinator: Tia Lum, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the safety and efficacy of Delpacibart etedesiran in people with DM1
Status: Recruiting soon
Recruitment Notes: Ambulatory individuals with DM1 ages 16-65 with hand myotonia
Facioscapulohumeral Muscular Dystrophy (FSHD)
Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
Protocol ID: 57072
NCT04635891
PI: Dr. John W. Day
Study Coordinator: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NIH and MDA
Purpose: Motor Outcomes to Validate Evaluations in FSHD
Status: Recruiting
Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)
Protocol ID: 67690
NCT05747924
PI: Dr. John W. Day
Study Coordinator: Susan Thomas, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the safety and efficacy of AOC-1020 in adults with FSHD
Status: Active, not recruiting
Recruitment Notes: Adults with FSHD ages 18-65
GNE myopathy
Observational study of adults with GNE myopathy
Protocol ID: 23888
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Natural history study and development of improved measures of muscle involvement of GNE myopathy in adults
Status: Recruiting
Recruitment Notes: Adults with GNE ages 18-65
IBM
A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis
Protocol ID: 68761
NCT05721573
PI: Dr. Neelam Goyal
Study Coordinator: Emily Lien, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Abcuro, Inc.
Purpose: Assessing the safety and efficacy of ABC008 in adults with IBM
Status: Recruiting
Recruitment Notes: Ambulatory adults with IBM age 40+
Limb Girdle Muscular Dystrophy (LGMD)
An International Clinical Outcome Study of Dysferlinopathy (COS2)
Protocol ID: 51930
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Jain Foundation
Purpose: Validation of clinical outcome measures in participants with dysferlinopathy
Status: Active, not recruiting
Pompe disease (PD)
Pompe Disease Registry
Protocol ID: 12372
NCT00231400
PI: Dr. John W. Day
Study Coordinator: Lesly Welsh, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Genzyme/Sanofi
Purpose: To collect information on the subjects with rare diseases like Pompe Disease and other lysosomal storage disorders longitudinally
Status: All Stanford patients diagnosed with Pompe disease
Recruitment Notes: All Stanford patients diagnosed with Pompe disease
Spinal bulbar muscular atrophy (SBMA)
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients with Spinal and Bulbar Muscular Atrophy (SBMA)
Protocol ID: 67773
NCT05517603
PI: Dr. John W. Day
Study Coordinator: Emily Lien, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: AnnJi
Purpose: Assessing safety and efficacy of JM17 in individuals with SBMA
Status: Active, not recruiting
Spinal muscular atrophy (SMA)
A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen (ONWARD)
Protocol ID: 59527
NCT04729907
PI: Dr. John W. Day
Study Coordinator: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Assessing the long term safety and efficacy of higher doses of Spinraza in SMA
Status: Active, not recruiting
A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam (ASCEND)
Protocol ID: 62798
NCT05067790
PI: Dr. John W. Day
Study Coordinator: Emily Lien, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Assessing safety and efficacy of higher dose of Spinraza in SMA
Status: Recruiting
Recruiting Notes: Previous treatment with Evrysdi or treatment-naive, non-ambulatory ages 15 to 50
iSMAC/PNCR
Protocol ID: 31140
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: SMA Foundation, CureSMA, Biogen
Purpose: To study the natural history of Spinal Muscular Atrophy to help with clinical trials in future
Status: Recruiting in clinic
CureSMA registry
Protocol ID: 49895
NCT: N/A
PI: Dr. Jacinda Sampson
Study Coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: CureSMA
Purpose: Clinical data registry with CureSMA
Status: Recruiting in clinic
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab (ONYX)
Protocol ID: 68228
NCT05626855
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Ayesha Zaina, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Scholar Rock
Purpose: Long term safety and efficacy of Apitegromab
Status: Active, not recruiting
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
Protocol ID: 52085
NCT05626855
PI: Dr. John W Day
Study Coordinator: Ayesha Zaina, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avexis/Novartis
Purpose: Assessing the long term effects of AVXS-101
Status: Active, not recruiting
Adult SMA Exploratory study (ASE)
Protocol ID: 55518
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Sabrina Salvatore, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Observational study and MRI of adults with SMA clinically treated with nusinersen or risdiplam
Status: Recruiting
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy (SYNAPSE-SMA)
Protocol ID: 69319
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Lidia Choniawko, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NMD Pharma
Purpose: Assessing safety and efficacy of NMD670 in ambulatory adults with SMA
Status: Recruiting
Recruiting Notes: Ambulatory adults with SMA3 ages 18-75
Development and Validation for the Adult Test of Neuromuscular Disorders (ATEND), a Functional Motor Outcome Measure
Protocol ID: 31140
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Development and evaluation of psychometric properties of the ATEND for weaker individuals with neuromuscular disorders
Status: Recruiting
Recruiting Notes: Non-ambulatory individual in a wheelchair with diagnosis of SMA
A Randomized, Double-blind, Placebo-controlled, Multinational, Multicenter study with open-label treatment extension to assess the effect of Min-102 on the progression of adrenomyeloneuropathy in male patients with x-linked adrenoleukodystrophy
PI: Jacinda Sampson, MD
NCT03231878
Study Status: Closed to Enrollment
Research Coordinator: Lila Perrone
Contact: Perronel@stanford.edu
Stroke and Neurocritical Care Clinical Research Group
The Divisions of Vascular Neurology and Neurocritical Care conduct a wide range of patient-centered research trials investigating the complex mechanisms of ischemic and hemorrhagic stroke injury, treatment, diagnosis and recovery. The Clinical Research Team consists of a Clinical Research Manager, 9 clinical research coordinators, a regulatory coordinator, data scientist and a data analyst supporting a variety of grant funded as well as industry and donor-sponsored research. In addition, the Divisions have a dedicated Brain Imaging Core Lab with over 17 years of experience of image processing as well as organization and storage of thousands of large imaging files.
BOOST3 – Brain Oxygen Optimization in Severe TBI Phase 3
BOOST3 is a study to learn if either of two strategies for monitoring and treating patients with traumatic brain injury (TBI) in the intensive care unit (ICU) is more likely to help them get better. More information: https://med.stanford.edu/neurology/divisions/neurocriticalcare/clinical-trials/boost3.html
Protocol ID: 49130
PI: Karen G. Hirsch, MD
Study Coordinator: Elizabeth Osborn
Status: RECRUITING
CERIBELL Stroke Study
The primary objective of this study is to acquire EEG data in suspected stroke patients, immediately following diagnostic brain imaging to detect potential EEG signals that differ among emergent large vessel occlusion stroke (ELVO), intracranial hemorrhage (ICH), and non-stroke patients (stroke mimics).
Protocol ID: 47822
PI: Anna Finley Caulfield, MD
Status: RECRUITING
ICECAP - Influence of Cooling duration on Efficacy in Cardiac Arrest Patients
The purpose of this study is to determine if increasing durations of induced hypothermia are associated with an increasing rate of good neurological outcomes and to identify the optimal duration of induced hypothermia for neuroprotection in comatose survivors of cardiac arrest.
NCT04217551
Protocol ID: 56493
PI: Karen Hirsch, MD
Status: RECRUITING
PRECICECAP - PREcision Care In Cardiac ArrEst -ICECAP
In partnership with the ICECAP trial, this project will use novel data-driven approaches to collect high resolution monitoring data from patients in the critical care unit and will use that multi-parametric data to develop newly defined patient sub-groups with differing clinical trajectories and responses to therapies.
Protocol ID: 58318
PI: Karen Hirsch, MD
Status: RECRUITING
PRECISE - Perfusion imaging to identify posterior circulation candidates for thrombectomy
The purpose of this research is to find out if advanced brain imaging can help identify which patients who have suffered an ischemic stroke (a blockage of blood flow) to the back of their brain are most likely to benefit from a procedure to remove the blood clot to restore the blood flow to the brain.
Protocol ID: Pro00053806
PI: Gregory Albers, MD
Study Coordinator: Irina Eyngorn
Status: RECRUITING
CRISP 2 - CT Perfusion to Predict Response to Recanalization in Ischemic Stroke Project
The purpose of this research is to analyze brain imaging data and clinical data to understand how stroke progresses through the brain.
Protocol ID: 56227
PI: Maarten Lansberg, MD, PhD
Study coordinator: Leonel Lugo
Status: RECRUITING
SATURN -Statins Use in Intracerebral Hemorrhage Patients
This research is being done to find out if it is better to continue or discontinue statin drugs (a type of cholesterol-lowering medication) in people who had a brain hemorrhage (bleeding in the brain) while taking such a statin drug. It will also examine if having certain genes (apolipoproteine E) influences the likelihood of getting a brain hemorrhage while taking a statin drug.
Protocol ID: 55536
PI: Chitra Venkatasubramanian, MD
Study coordinator: Madelleine Garcia
Status: RECRUITING
ASPIRE: Anticoagulation in ICH (intracerebral hemorrhage) Survivors for Stroke Prevention and Recovery
The purpose of this study is to compare the effects of a drug called apixaban with aspirin in patients with atrial fibrillation (irregular heart beating) and a recent brain hemorrhage (bleeding in the brain) to see which is better in preventing strokes and death.
NCT03907046
IRB #: 52983
PI: Chitra Venkatasubramanian, MD
Study coordinator: Madelleine Garcia
Status: RECRUITING
BEACH: Biomarker and Edema Attenuation in IntraCerebral Hemorrhage (BEACH)
The purpose of this research study is to find out if a study drug called MW189 is safe and well tolerated in patients with bleeding in the brain (Intracranial Hemorrhage or ICH), and whether it has a beneficial effect on patients’ outcomes and recovery. MW189 is an anti-inflammatory drug candidate being studied as a possible treatment to reduce the excessive inflammation and brain swelling.
NCT05020535
IRB #: 295926
PI: Chitra Venkatasubramanian, MD
Study coordinator: Madelleine Garcia
Status: RECRUITING
Librexia Stroke Study: Efficacy and Safety of Milvexian, an Oral Factor XIa Inhibitor, for Stroke Prevention after an Acute Ischemic Stroke or High-Risk Transient Ischemic Attack
The purpose of this research study is to see if an experimental drug, called milvexian in addition to standard of care, is safe and useful in reducing the risk of future ischemic stroke in participants after ischemic stroke or transient ischemic attack compared to placebo (tablet with no active drug) in addition to standard of care.
IRB #: 70999
PI: Nirali Vora, MD
Study coordinator: Leonel Lugo
Status: RECRUITING
CIRCA Registry: The CIRCA Chronotype and Stroke Registry: A Multicenter Study of the Association of Chronotype with Presentation, Course, and Outcome of Acute Cerebral Ischemia
The goal of this study is to figure out how natural sleep patterns, known as "chronotype," affect the course of stroke and stroke treatment.
IRB #: 72264
PI: Gregory Albers, MD
Study Coordinator: Frances Lizbeth Palomata, MBA
Status: RECRUITING
Home BP Cuff Study: Improving Blood Pressure Control in Stroke Patients by Increasing Access to a Home Blood Pressure Monitor
The purpose of this study is to investigate if providing focused teaching and a free blood pressure cuff to patients at the time of discharge from the hospital helps to facilitate improved blood pressure control. Eligible patients are those who have high blood pressure and are deemed to be at risk for stroke.
Protocol ID: 68463
PI: Lironn Kraler, MD
Study coordinator: Madelleine Garcia
Status: RECRUITING
Sodium imaging Study: Ischemic Brain Damage & Triple/Single Quantum Sodium MRI
The objective of this study is to use a new method of Magnetic Resonance Imaging (MRI) to identify the sodium concentration in brain tissue in patients with a stroke. We hope that this new method may be helpful in assessing the outcomes of potential therapies for acute stroke.
IRB #: 64720
PI: Fernando Boada, PhD
Study coordinator: Irina Eyngorn
Status: RECRUITING
StrokeCog
The purpose of this research study is to understand the long-term effects of stroke on a person’s memory and thinking. To determine this, we will schedule stroke patients to come in on a yearly basis for memory testing and collection of a small amount of blood.
Protocol ID: 42089
PI: Maarten Lansberg, MD, PhD
STATUS: RECRUITING
Neuro-Oncology Clinical Research Group
The Neuro-Oncology Research Group supports 9 Investigators (4 neuro-oncologists, 3 neurosurgeons and 2 radiation therapists). It is integrated into the Stanford Cancer Center’s Clinical Trials Office and works primarily in the Cancer Center Clinics. There are 7 research coordinators, a regulatory specialist and an operations manager that support observational, interventional, industry sponsored, and investigator-initiated projects, currently numbering 26 total. The Group’s goals are to ensure, timely, safe, and successful implementation of neuro-oncology clinical research.
Newly Diagnosed Glioma
A Phase I Study of Tumor Treating Fields with 5-Day Hypofractionated Stereotactic Radiosurgery and Concurrent and Maintenance Temozolomide in Newly Diagnosed Glioblastoma
PI: Scott Soltys, MD
ClinicalTrials.gov#: NCT04474353
A Phase II Study of BPM31510 with Vitamin K1 in Subjects with Newly Diagnosed Glioblastoma (GB)
PI: Seema Nagpal, MD
ClinicalTrials.gov#: NCT04752813
ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)
Only open to adult patients at Stanford
PI: Kate Therkelsen, MD
ClinicalTrials.gov#: NCT05580562
Recurrent High Grade Glioma
Phase I Clinical Trial of Locoregionally (LR) Delivered Autologous B7-H3 Chimeric Antigen Receptor T Cells (B7-H3CART) in Adults with Recurrent Glioblastoma Multiforme (GBM)
PI: Reena Thomas, MD PhD
ClinicalTrials.gov#: NCT05474378
Study to Evaluate Safety, Tolerability, and Optimal Dose of Candidate GBM Vaccine VBI-1901 in Recurrent GBM Subjects
PI: Seema Nagpal, MD
ClinicalTrials.gov#: NCT03382977
A Phase Ib Clinical Trial to Evaluate Early Immunologic Pharmacodynamic Parameters Following Neoadjuvant Anti-PD-1 (Nivolumab), or the Combination of Anti-PD-1 Plus Anti-CTLA-4 (Nivolumab Plus Ipilimumab) in Patients With Surgically Accessible Glioblastoma
PI: Michael Lim, MD
ClinicalTrials.gov#: NCT04606316
Phase I/II, Open-Label Study Evaluating the Efficacy and Pharmacokinetics of Panitumumab-IRDye800 as an Optical Imaging Agent to Detect Neoplasms during Neurosurgical Procedures
PI: Gordon Li, MD
ClinicalTrials.gov#: NCT03510208
Brain and Spine Metastases
A Phase IIa Study Assessing QBS72S For Treating Brain Metastases of Breast Cancer
PI: Melanie Hayden Gephart, MD
ClinicalTrials.gov#: NCT05305365
A Randomized Phase III Trial of Pre-Operative Compared to Post-Operative Stereotactic Radiosurgery in Patients With Resectable Brain Metastases
PI: Scott Soltys, MD
ClinicalTrials.gov#: NCT05438212
A Randomized Phase III TriaL Comparing SingLe- Versus Multi-Fraction Spine STereotActic Radiosurgery for Patients with Spinal Metastases (ALL-STAR)
PI: Erqi Pollum, MD
Primary CNS Lymphoma
An Open-Label Phase II Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Tirabrutinib in Patients with Primary Central Nervous System Lymphoma (PCNSL)
PI: Neel Gupta, MD
ClinicalTrials.gov#: NCT04947319
LGG
Newly Diagnosed
ACNS1831: A Phase 3 Randomized Study of Selumetinib (IND #77782) Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Diagnosis: Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
ACNS1833: A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
Diagnosis: Low-Grade Glioma (LGG)
Recurrent/Refractory/Progressive
PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: LGG/HGG & LGG with NF1
ACNS1931: A Phase 3 Study of Selumetinib (NSC# 748727) or Selumetinib in Combination With Vinblastine for Non-NF1, Non-TSC Patients With Recurrent or Progressive Low-Grade Gliomas (LGGs) Lacking BRAFV600E or IDH1 Mutations
Diagnosis: LGG lacking BRAFV600E or IDH1 Mutations
DIPG
Newly Diagnosed
PBTC060: A Pilot Study of SurVaxM in Children Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma
Diagnosis: Diffuse Intrinsic Pontine Glioma (DIPG)
PBTC048: Optune for Children With High-Grade Glioma or Ependymoma, and Optune With Radiation Therapy for Children With DIPG
Diagnosis: Diffuse Intrinsic Pontine Glioma (DIPG)
GD2 CAR-T: Phase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor (CAR) T cells (GD2CART) for Diffuse Intrinsic Pontine Gliomas (DIPG) and Spinal Diffuse Midline Glioma (DMG)
Diagnosis: Diffuse Intrinsic Pontine Glioma (DIPG) and Spinal Cord Diffuse Midline Glioma (DMG) that is H3K27 mutated
ACNS1821: A Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed Pediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)
Diagnosis: Diffuse Intrinsic Pontine Glioma (DIPG)
Recurrent/Refractory/Progressive
PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Recurrent, progressive, or refractory primary CNS tumor with evidence of genetic activation of the MET pathway
HGG
Newly Diagnosed
ACNS1723: A Phase 2 Study of Dabrafenib (NSC# 763760) With Trametinib (NSC# 763093) After Local Irradiation in Newly-Diagnosed BRAF V600-Mutant High-Grade Glioma (HGG)
Diagnosis: BRAF V600-Mutant HGG
ACNS1821: A Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed Pediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)
Diagnosis: High-Grade Glioma (HGG)
Recurrent/Refractory/Progressive
PBTC060: A Pilot Study of SurVaxM in Children Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma
Diagnosis: High Grade Glioma (HGG)
PBTC048: A Feasibility Trial of Optune for Children with Recurrent or Progressive Supratentorial High-Grade Glioma and Ependymoma
Diagnosis: Supratentorial HGG
PBTC-045: A Safety and Preliminary Efficacy trial of MK-3475 (pembrolizumab; anti-PD-1) in children with recurrent, progressive or refractory high-grade gliomas (HGG), DIPGs and hypermutated brain tumors
Diagnosis: Stratum B- histologically confirmed diagnosis of a non-brainstem high-grade glioma (NB-HGG) that is recurrent, progressive or refractory following therapy which included radiotherapy. Spinal primary disease is eligible
PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Recurrent, progressive, or refractory primary CNS tumor with evidence of genetic activation of the MET pathway
PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: Phase 2 stratum 4: HGG with BRAF V600E/D/K mutation
Ependymoma
Newly Diagnosed
There are currently no open trials
Recurrent/Refractory/Progressive
PBTC060: A Pilot Study of SurVaxM in Children Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma
Diagnosis: Ependymoma
PBTC048: A Feasibility Trial of Optune for Children with Recurrent or Progressive Supratentorial High-Grade Glioma and Ependymoma
Diagnosis: Supratentorial ependymoma
MEDULLOBLASTOMA
Newly Diagnosed
ACNS1422: A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients
Diagnosis: Medulloblastoma
Recurrent/Refractory/Progressive
Diagnosis: Medulloblastoma
PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Recurrent, progressive, or refractory primary CNS tumor with evidence of genetic activation of the MET pathway
PBTC-053: A Pediatric Brain Tumor Consortium Phase I/ II and Surgical Study of CX-4945 in Patients with Recurrent SHH Medulloblastoma
Diagnosis: Medulloblastoma
ATRT
Newly Diagnosed
None
Recurrent/Refractory/Progressive
PEPN2121: A Phase 1/2 Study of Tiragolumab (NSC# 827799, IND# 161266) and Atezolizumab (NSC# 783608, IND# 161266) in Patients with Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
Diagnosis: Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
Neurofibromatosis-1 (NF1)
Newly Diagnosed
ACNS1831- A Phase 3 Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Diagnosis: NF1 associated LGG
Recurrent/Refractory/Progressive
PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: NF1-associated recurrent or progressive gliomas
Other- nontreatment
NF1-OPG Natural History Study: Developing Evidence-Based Criteria for Initiating Treatment for Neurofibromatosis Type 1 Associated Optic Pathway Glioma/CTF 004: The Collection of Blood Samples from Patients with NF1 for Research Purposes
Diagnosis: Optic pathway glioma
OTHER STUDIES & MOLECULAR BASED THERAPIES
ACNS2021: A Phase 2 Trial of Chemotherapy followed by Response-Based Whole Ventricular & Spinal Canal Irradiation (WVSCI) for Patients with Localized Non-Germinomatous Central Nervous System Germ Cell Tumor
Diagnosis: Newly diagnosed with localized primary CNS NGGCT of the suprasellar and/or pineal region
ACCL2031: A Phase 3 Randomized, Placebo-Controlled Trial Evaluating Memantine (IND# 149832) for Neurocognitive Protection in Children Undergoing Cranial Radiotherapy as Part of Treatment for Primary Central Nervous System Tumors
Diagnosis: Primary Central Nervous System Tumors
G042286: A Phase I/II, Open-Label, Multicenter, Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors for Whom Prior Treatment Has Proven to be Ineffective or for Whom There is No Satisfactory Treatment Available
Diagnosis: CNS or solid tumors harboring ALK gene fusions
LOXO RET: A Phase 1/2 Study of the Oral RET Inhibitor LOXO 292 in Pediatric Patients With Advanced RET-Altered Solid or Primary Central Nervous System Tumors
Diagnosis: Advanced or metastatic solid or primary CNS tumor which has failed standard of care therapies
POE16-01: A Phase I/II Study of Neratinib in Pediatric Patients with Relapsed/ Refractory Solid Tumors or Hematologic Malignancies
Diagnosis: Solid & Central Nervous System (CNS) Tumor, Lymphoma, or Leukemia-
ALTE07C1: Neuropsychological, Social, Emotional and Behavioral Outcomes in Children with Cancer
Diagnosis: Non-disease specific, nontreatment
LOXO-ON-TRK: Prospective Non-interventional study in patients with locally advanced or metastatic TRK fusion cancer treated with larotrectinib
Diagnosis: Non-treatment. locally advanced or metastatic solid tumor harboring an NTRK gene fusion
APEC14B1: The Project: Everychild Protocol: A Registry, Eligibility, Screening, Biology, and Outcome Study
Diagnosis: Registry or eligibility arm
APEC1621: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)
Diagnosis: CNS diagnosis with specific mutation
- APEC1621F: Phase 2 subprotocol of Ensartinib in patients with tumors harboring ALK or ROS1 genomic alterations
- APEC1621S: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol
FURTHER INFORMATION AND SUMMARIES FOR HEALTH CARE PROFESSIONALS
Pediatric Clinical Research Group
The division of child neurology maintains a broad clinical research portfolio which typically includes 15-20 active studies in pediatric epilepsy, neurogenetics, pediatric stroke, leukodystrophies, and neonatal neurology. Our research team includes a full time clinical research manager, data analyst, research administrative associate, and three full time clinical research coordinators.
LGG
Newly Diagnosed
ACNS1831: A Phase 3 Randomized Study of Selumetinib (IND #77782) Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Diagnosis: Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
ACNS1833: A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
Diagnosis: Low-Grade Glioma (LGG)
Recurrent/Refractory/Progressive
PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: LGG/HGG & LGG with NF1
ACNS1931: A Phase 3 Study of Selumetinib (NSC# 748727) or Selumetinib in Combination With Vinblastine for Non-NF1, Non-TSC Patients With Recurrent or Progressive Low-Grade Gliomas (LGGs) Lacking BRAFV600E or IDH1 Mutations
Diagnosis: LGG lacking BRAFV600E or IDH1 Mutations
DIPG
Newly Diagnosed
PBTC060: A Pilot Study of SurVaxM in Children Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma
Diagnosis: Diffuse Intrinsic Pontine Glioma (DIPG)
PBTC048: Optune for Children With High-Grade Glioma or Ependymoma, and Optune With Radiation Therapy for Children With DIPG
Diagnosis: Diffuse Intrinsic Pontine Glioma (DIPG)
GD2 CAR-T: Phase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor (CAR) T cells (GD2CART) for Diffuse Intrinsic Pontine Gliomas (DIPG) and Spinal Diffuse Midline Glioma (DMG)
Diagnosis: Diffuse Intrinsic Pontine Glioma (DIPG) and Spinal Cord Diffuse Midline Glioma (DMG) that is H3K27 mutated
ACNS1821: A Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed Pediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)
Diagnosis: Diffuse Intrinsic Pontine Glioma (DIPG)
Recurrent/Refractory/Progressive
PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Recurrent, progressive, or refractory primary CNS tumor with evidence of genetic activation of the MET pathway
HGG
Newly Diagnosed
ACNS1723: A Phase 2 Study of Dabrafenib (NSC# 763760) With Trametinib (NSC# 763093) After Local Irradiation in Newly-Diagnosed BRAF V600-Mutant High-Grade Glioma (HGG)
Diagnosis: BRAF V600-Mutant HGG
ACNS1821: A Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed Pediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)
Diagnosis: High-Grade Glioma (HGG)
Recurrent/Refractory/Progressive
PBTC060: A Pilot Study of SurVaxM in Children Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma
Diagnosis: High Grade Glioma (HGG)
PBTC048: A Feasibility Trial of Optune for Children with Recurrent or Progressive Supratentorial High-Grade Glioma and Ependymoma
Diagnosis: Supratentorial HGG
PBTC-045: A Safety and Preliminary Efficacy trial of MK-3475 (pembrolizumab; anti-PD-1) in children with recurrent, progressive or refractory high-grade gliomas (HGG), DIPGs and hypermutated brain tumors
Diagnosis: Stratum B- histologically confirmed diagnosis of a non-brainstem high-grade glioma (NB-HGG) that is recurrent, progressive or refractory following therapy which included radiotherapy. Spinal primary disease is eligible
PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Recurrent, progressive, or refractory primary CNS tumor with evidence of genetic activation of the MET pathway
PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: Phase 2 stratum 4: HGG with BRAF V600E/D/K mutation
Ependymoma
Newly Diagnosed
There are currently no open trials
Recurrent/Refractory/Progressive
PBTC060: A Pilot Study of SurVaxM in Children Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma
Diagnosis: Ependymoma
PBTC048: A Feasibility Trial of Optune for Children with Recurrent or Progressive Supratentorial High-Grade Glioma and Ependymoma
Diagnosis: Supratentorial ependymoma
MEDULLOBLASTOMA
Newly Diagnosed
ACNS1422: A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients
Diagnosis: Medulloblastoma
Recurrent/Refractory/Progressive
Diagnosis: Medulloblastoma
PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Recurrent, progressive, or refractory primary CNS tumor with evidence of genetic activation of the MET pathway
PBTC-053: A Pediatric Brain Tumor Consortium Phase I/ II and Surgical Study of CX-4945 in Patients with Recurrent SHH Medulloblastoma
Diagnosis: Medulloblastoma
ATRT
Newly Diagnosed
None
Recurrent/Refractory/Progressive
PEPN2121: A Phase 1/2 Study of Tiragolumab (NSC# 827799, IND# 161266) and Atezolizumab (NSC# 783608, IND# 161266) in Patients with Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
Diagnosis: Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
Neurofibromatosis-1 (NF1)
Newly Diagnosed
ACNS1831- A Phase 3 Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Diagnosis: NF1 associated LGG
Recurrent/Refractory/Progressive
PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: NF1-associated recurrent or progressive gliomas
Other- nontreatment
NF1-OPG Natural History Study: Developing Evidence-Based Criteria for Initiating Treatment for Neurofibromatosis Type 1 Associated Optic Pathway Glioma/CTF 004: The Collection of Blood Samples from Patients with NF1 for Research Purposes
Diagnosis: Optic pathway glioma
OTHER STUDIES & MOLECULAR BASED THERAPIES
ACNS2021: A Phase 2 Trial of Chemotherapy followed by Response-Based Whole Ventricular & Spinal Canal Irradiation (WVSCI) for Patients with Localized Non-Germinomatous Central Nervous System Germ Cell Tumor
Diagnosis: Newly diagnosed with localized primary CNS NGGCT of the suprasellar and/or pineal region
ACCL2031: A Phase 3 Randomized, Placebo-Controlled Trial Evaluating Memantine (IND# 149832) for Neurocognitive Protection in Children Undergoing Cranial Radiotherapy as Part of Treatment for Primary Central Nervous System Tumors
Diagnosis: Primary Central Nervous System Tumors
G042286: A Phase I/II, Open-Label, Multicenter, Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors for Whom Prior Treatment Has Proven to be Ineffective or for Whom There is No Satisfactory Treatment Available
Diagnosis: CNS or solid tumors harboring ALK gene fusions
LOXO RET: A Phase 1/2 Study of the Oral RET Inhibitor LOXO 292 in Pediatric Patients With Advanced RET-Altered Solid or Primary Central Nervous System Tumors
Diagnosis: Advanced or metastatic solid or primary CNS tumor which has failed standard of care therapies
POE16-01: A Phase I/II Study of Neratinib in Pediatric Patients with Relapsed/ Refractory Solid Tumors or Hematologic Malignancies
Diagnosis: Solid & Central Nervous System (CNS) Tumor, Lymphoma, or Leukemia-
ALTE07C1: Neuropsychological, Social, Emotional and Behavioral Outcomes in Children with Cancer
Diagnosis: Non-disease specific, nontreatment
LOXO-ON-TRK: Prospective Non-interventional study in patients with locally advanced or metastatic TRK fusion cancer treated with larotrectinib
Diagnosis: Non-treatment. locally advanced or metastatic solid tumor harboring an NTRK gene fusion
APEC14B1: The Project: Everychild Protocol: A Registry, Eligibility, Screening, Biology, and Outcome Study
Diagnosis: Registry or eligibility arm
APEC1621: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)
Diagnosis: CNS diagnosis with specific mutation
- APEC1621F: Phase 2 subprotocol of Ensartinib in patients with tumors harboring ALK or ROS1 genomic alterations
- APEC1621S: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol
FURTHER INFORMATION AND SUMMARIES FOR HEALTH CARE PROFESSIONALS
All Neuromuscular Conditions
Subject Database and Specimen Repository for Neuromuscular and Neurodegenerative Disorders
Protocol ID: 23888
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: This is a study that involves collecting clinical information of subjects (patients with any neurological condition or their close family member) and tissue samples to develop a recruitment database and tissue bank, which will be of great value in helping the investigators learn more about various related neurological conditions.
Status: Recruiting
Tissue Banking of Blood, Spinal Fluid or Skin Biopsy for the Research of Neurological Diseases
Protocol ID: 16472
NCT: N/A
PI: Dr. Yuen So
Study Coordinator: Shirley Paulose, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: To collect blood, spinal fluid, or skin biopsy specimens to create a tissue bank for current or future neuroscience research, which would help to learn more about various neurological conditions
Status: Recruiting
MOVR Registry
Protocol ID: 52620
NCT: N/A
PI: Dr. Jacinda Sampson
Study Coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Muscular Dystrophy Association
Purpose: Observational study of neuromuscular conditions including ALS, BMD/DMD, FSHD, LGMD, Pompe and SMA
Status: Recruiting soon in clinic
Determination of Standards for Maximal and Submaximal Exercise testing for Individuals with Neuromuscular Disease (CPET)
Protocol ID: 54078
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Sabrina Salvatore, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Understanding exercise testing which will assess how heart and lungs work together to get oxygen to muscles in children and adults with muscular dystrophies
Status: Recruiting
Recruitment Notes: All clinic patients ages 9+ with diagnosed neuromuscular disorders who are able to perform testing on cycle ergometer as well as controls
Amyotrophic Lateral Sclerosis (ALS)
Clinical Procedures to Support Research in ALS (CAPTURE-ALS)
Protocol ID: 54467
NCT03489278
PI: Dr. Yuen So
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NINDS/NIH, MDA
Purpose: Natural History Study of ALS and PLS
Status: Recruiting in clinic
A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients With Fused in Sarcoma Mutations (FUSION)
Protocol ID: 59619
NCT04768972
PI: Dr. John W. Day
Study Coordinator: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: IONIS Pharmaceuticals
Purpose: Assessing the safety and efficacy of ION363 in children with ALS
Status: Recruiting
Recruitment Notes: Confirmed genetic mutation in FUS
Expanded Access Program to ION363 FUS ALS treatment
Protocol ID: N/A
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Expanded Access Program
Status: Recruiting on case-by-case basis
Recruitment Notes:
Becker and Duchenne muscular dystrophy (BMD/DMD)
A Gene Delivery Study to Evaluate the Safety of and Expression From SRP-9001 in Duchenne Muscular Dystrophy (DMD) (ENDEAVOR)
Protocol ID: 59448
NCT04626674
PI: Dr. John W. Day
Study Coordinator: Lesly Welsh, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Sarepta Therapeutics, Inc
Purpose: Assessing the safety and efficacy of SRP-9001 gene therapy in individuals with DMD
Status: Active, not recruiting
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (ENVISION)
Protocol ID: 64248
NCT05881408
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Sarepta Therapeutics, Inc
Purpose: Assessing the safety and efficacy of SRP-9001 gene therapy in individuals with DMD
Status: Active, not recruiting
Long-term follow-up study for all SRP-9001 studies (EXPEDITION)
Protocol ID: 71344
NCT05967351
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Lesly Welsh, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Sarepta Therapeutics, Inc
Purpose: Long term follow-up study of safety and efficacy of SRP-9001
Status: Active, recruiting by invitation only
Recruitment Notes: Requires enrollment in previous Sarepta SRP-9001 trials
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping (EXPLORE44)
Protocol ID: 69829
NCT05670730
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the safety and efficacy of AOC1044 in DMD
Status: Recruiting
Recruitment Notes: Individuals with DMD mutations amenable to exon 44 skipping, ages 7-27
A Phase 1/2 Open-label, Dose Escalation and Dose Expansion Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males with Duchenne Muscular Dystrophy (DMD) (AFFINITY DMD)
Protocol ID: 52155
NCT05693142
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Yan Yang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: REGENXBIO
Purpose: Assessing the safety and efficacy of RGX-202 gene therapy in children with DMD
Status: Recruiting
Recruitment Notes: Boy ages 4-12, ambulatory, DMD mutations in exons 18 or higher
Charcot-Marie Tooth Disease (CMT)
Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Type (CMT1B), 2A (CMT2A), 4A (CMT4A), 4C (CMT4C), and Others
Protocol ID: 23094
NCT01193075
PI: Dr. Maxwell Greene
Study Coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NIH and MDA
Purpose: Natural history study of CMT
Status: Recruiting in clinic
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J (CMT4J)
Protocol ID: 72459
NCT06151600
PI: Dr. John W. Day
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Elpida Therapeutics
Purpose: Natural history study of CMT4J
Status: Recruiting soon
Myotonic dystrophy (DM)
Outcome validation with functional measures in myotonic dystrophy (MYOCAP/Actimyo)
Protocol ID: 62522
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Outcome validation with functional measures in myotonic dystrophy
Status: Recruiting
Avidity HARBOR trial
Protocol ID: 74759
NCT: TBD
PI: Dr. John W. Day
Study Coordinator: Tia Lum, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the safety and efficacy of Delpacibart etedesiran in people with DM1
Status: Recruiting soon
Recruitment Notes: Ambulatory individuals with DM1 ages 16-65 with hand myotonia
Facioscapulohumeral Muscular Dystrophy (FSHD)
Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
Protocol ID: 57072
NCT04635891
PI: Dr. John W. Day
Study Coordinator: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NIH and MDA
Purpose: Motor Outcomes to Validate Evaluations in FSHD
Status: Recruiting
Myasthenia Gravis (MG)
An Open-Label Uncontrolled Multicenter Study to Evaluate the Pharmacokinetics,Pharmacodynamics, Safety and Activity of Nipocalimab in Children Aged 2 to less than 18 years with Generalized Myasthenia Gravis
Protocol ID: 64037
NCT05265273
Dr. Carolina Tesi-Rocha
Study Coordinator: Yan Yang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Janssen
Purpose: Assessing the safety and efficacy of Nipocalimab in children with gMG
Status: Recruiting
Recruitment Notes: Children with gMG ages 6-18 with suboptimal response to current stable therapy
Pompe disease (PD)
Pompe Disease Registry
Protocol ID: 12372
NCT00231400
PI: Dr. John W. Day
Study Coordinator: Lesly Welsh, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Genzyme/Sanofi
Purpose: To collect information on the subjects with rare diseases like Pompe Disease and other lysosomal storage disorders longitudinally
Status: All Stanford patients diagnosed with Pompe disease
Recruitment Notes: All Stanford patients diagnosed with Pompe disease
Spinal muscular atrophy (SMA)
A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen (ONWARD)
Protocol ID: 59527
NCT04729907
PI: Dr. John W. Day
Study Coordinator: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Assessing the long term safety and efficacy of higher doses of Spinraza in SMA
Status: Active, not recruiting
A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec (RESPOND)
Protocol ID: 58724
NCT04488133
PI: Dr. John W. Day
Study Coordinator: Susan Thomas, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Assessing the safety and efficacy of Spinraza treatment after Zolgensma treatment
Status: Recruiting
Recruiting Notes: Previous treatment with Zolgensma
A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam (ASCEND)
Protocol ID: 62798
NCT05067790
PI: Dr. John W. Day
Study Coordinator: Emily Lien, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Assessing safety and efficacy of higher dose of Spinraza in SMA
Status: Recruiting
Recruiting Notes: Previous treatment with Evrysdi or treatment-naive, non-ambulatory ages 15 to 50
iSMAC/PNCR
Protocol ID: 31140
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: SMA Foundation, CureSMA, Biogen
Purpose: To study the natural history of Spinal Muscular Atrophy to help with clinical trials in future
Status: Recruiting in clinic
CureSMA registry
Protocol ID: 49895
NCT: N/A
PI: Dr. Jacinda Sampson
Study Coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: CureSMA
Purpose: Clinical data registry with CureSMA
Status: Recruiting in clinic
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab (ONYX)
Protocol ID: 68228
NCT05626855
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Ayesha Zaina, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Scholar Rock
Purpose: Long term safety and efficacy of Apitegromab
Status: Active, not recruiting
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
Protocol ID: 52085
NCT05626855
PI: Dr. John W Day
Study Coordinator: Ayesha Zaina, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avexis/Novartis
Purpose: Assessing the long term effects of AVXS-101
Status: Active, not recruiting
Pediatric Epilepsy and Seizures
Sunflower Syndrome - Self-Induced Photosensitive Epilepsy
PI: Fiona Baumer, MD
Epileptic encephalopathy with continuous spike-and-wave during sleep (CSWS): a review of current treatment practices
PI: Fiona Baumer, MD
Cortical Excitability, Synaptic Plasticity and Learning in Benign Epilepsy with Centrotemporal Spikes (BECTS)
PI: Fiona Baumer, MD
Consortium for Standardizing TMS (Transcranial Mangetic Stimulation) Language Mapping Protocols and Establishing Best Practice Guidelines for Using TMS in Presurgical Evaluation of Language in Children
PI: Fiona Baumer, MD
Consequences of Prolonged Febrile Seizures in Childhood
PI: William Gallentine, DO
Novel Early Imaging Markers Following Febrile Status Epilepticus
PI: William Gallentine, DO
Open-Label Safety and Efficacy Study of Cenobamate (YKP3089) in Pediatric Subjects with Partial-onset (Focal) Seizures
PI: William Gallentine, DO
Brain Connectivity Changes in Benign Epilepsy with Centrotemporal Spikes and other Pediatric Epilepsy Syndromes
PI: Fiona Baumer, MD
Diagnostic, Prognostic, and Therapeutic Applications of TMS for Epilepsy
PI: Fiona Baumer, MD
A Multi-Center Examination of Pediatric Epilepsy Surgery Evaluation in the US
PI: Ann Hyslop, MD
Investigation of Keto-Amino Acids as Novel Therapeutics for Refractory Pediatric Epilepsy
PI: Juliet Knowles, MD, PhD
A Randomized, Double-Blind, Placebo-Controlled Study To Investigate The Efficacy And Safety Of Carisbamate (YKP509) As Adjunctive Treatment For Seizures Associated With Lennox-Gastaut Syndrome In Children And Adults, With Optional Open-Label Extension (SK-LGS)
PI: Brenda Porter, MD, PhD
Children with Lennox-Gastaut Syndrome (LGS) treated with Deep Brain Stimulation (DBS)
PI: Juliet Knowles, MD, PhD
White matter structure and function in children with Lennox-Gastaut Syndrome
PI: Juliet Knowles, MD, PhD
A Multimodal Biomarker-based Predictive Model for Infantile Spasms
PI: Ann Hyslop, MD
PERF Genetic Epilepsy Registry
PI: Juliet Knowles, MD, PhD
MRI Microstructure Mapping to Improve Detection of Focal Cortical Dysplasia
PI: Juliet Knowles, MD, PhD
Study of Oral MC-1 for the Treatment of Patients with PNPO Deficiency
PI: Courtney Wusthoff, MD
Neurogenetic disorders
A Natural History Study of Rare Neurogenetic Disorders
PI: Rebecca Levy, MD, PhD
A Retrospective Case Series of Timothy Syndrome
PI: Rebecca Levy, MD, PhD
SLC13A5 Deficiency:A Prospective Natural History Study
PI: Brenda Porter, MD, PhD
Tuberous Sclerosis Complex
Pediatric Epilepsy Surgery: Decision-Making Survey in Patients with Tuberous Sclerosis
PI: Brenda Porter, MD, PhD
Preventing Epilepsy Using Vigabatrin in Infants with Tuberous Sclerosis Complex (PREVeNT Trial)
PI: Brenda Porter, MD, PhD
Stopping Tuberous Sclerosis Complex Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study (TSC-STEPS)
PI: Brenda Porter, MD, PhD
Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)
PI: Brenda Porter, MD, PhD
Pediatric neuroinflammatory disorders
Project BIG: The Stanford Brain Immune Gut Research Initiative
PI: Keith Van Haren, MD
Biobank for Neuroinflammatory Disorders
PI: Keith Van Haren, MD
Leukodystrophy
A phase 1-3, Double Bind, Randomized Placebo- Controlled Study to Evaluate the Efficacy, Safety, PharmacoKinetics and Pharmacodynamics Intrathecally Administered ION373 in Patients with Alexander Disease (Gene Transfer)
PI: Keith Van Haren, MD
A phase 3 Study of Lenti-D Drug Product After Myeloblative Conditioning Using Busulfan and Fludarabine in Subjetcs 17 Years of Age and Under With Cerebral Adrenoleukodystrophy (CALD) (BMT/GeneTransfer)
PI: Keith Van Haren, MD
The Effect of Vitamin D3 on Markers of Oxidative Stress in Boys with X-Linked Adrenoleukodystrophy (ALD)
PI: Keith Van Haren, MD
New Diagnostic Approaches in Leukodystrophy - The Myelin Disorders Biorepository and Natural History Project
PI: Keith Van Haren, MD
Multiple Sclerosis
Continuation of the analysis of Cytokine Modulation with Tecfidera in Human Immune Cell Subsets using High Dimensional Flow Cytometry and CYTOF Mass Spectrometry
PI: Lawrence Steinman, MD
Single Cell RNA-seq and CITE-seq on B cells in the peripheral blood and CSF of MS patients prior and during therapy with ATA-188
PI: Lawrence Steinman, MD
Neonatal Neurology
Neonatal Seizure Registry
PI: Courtney Wusthoff, MD
High Frequency Oscillations in Neonates
PI: Courtney Wusthoff, MD
Neonatal Seizure Registry- Developmental functional Evaluation (NSR-DEV)
PI: Courtney Wusthoff, MD
Continued Anticonvulsants After Resolution of Neonatal Seizures: A Patient-centered Comparative Effectiveness Study
PI: Courtney Wusthoff, MD
Temporal-spatial quantification of neonatal seizure burden caused by arterial ischemic stroke
PI: Courtney Wusthoff, MD
MRI and EEG findings in preterm neonates with HIE
PI: Courtney Wusthoff, MD
Childhood Stroke
Vascular Effects of Infection in Pediatric Stroke (VIPS II) Study
PI: Sarah Lee, MD
Stanford Children's Health Pediatric Stroke Database
PI: Sarah Lee, MD
International Pediatric Stroke Registry: Risk Factors and Outcome Observational Study
PI: Sarah Lee, MD
Seizures and Children 's Outcomes after Stroke (SCOUTS)
PI: Sarah Lee, MD
Leducq Trans-Atlantic Network of Excellence On Circadian Effects in Stroke (CIRCA-Stroke)
PI: Sarah Lee, MD
Focal Cerebral Arteriopathy Steroid Trial Pediatric Comparative Effectiveness Trial (FOCAS)
PI: Sarah Lee, MD
acutE Perfusion Imaging in Childhood LVO (EPIC-LVO): A Prospective Neuroimaging Pilot Study
PI: Sarah Lee, MD
Pediatric Neurocritical Care
Practices in the Evaluation of Pediatric Brain Death
PI: Courtney Wusthoff, MD
Critical Care EEG Monitoring Research Consortium
PI: Courtney Wusthoff, MD
Creation and Development of Pediatric Neurocritical Care Database
PI: Courtney Wusthoff, MD
Neurofibromatosis Type 1 (NF1)
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
PI: Cynthia Campen, MD
A Phase 2b Trial of MEK ½ Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients with Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) that are Causing Significant Morbidity
PI: Cynthia Campen, MD
Developing Evidence-Based Criteria for Initiating Treatment for NF1-Associated Optic Pathway Glioma: A Natural History Observational Study
PI: Cynthia Campen, MD
Neuroimaging Correlates of Neurocognitive Deficits in Children with NF1
PI: Cynthia Campen, MD
Optic disc drusen
Optic Disc Drusen in children
PI: Shannon Beres, MD
Adult Clinical Trials
For questions about clinical trials please contact our research team at neurotrials@stanford.edu.
Pediatric Clinical Trials
To find out the current status of any pediatric study please contact us at: arushi.gehani@stanford.edu
For pediatric neuro-oncology studies, please contact: Neuroonc@stanfordchildrens.org