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ClinicalTrials.gov public records Last synced Oct 22, 2026, 10:29 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 1,268 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Nervous System Heredodegenerative Disorder Clear all
Completed Phase 1 Interventional Results available

Safety and Efficacy of Pembrolizumab (MK-3475) Plus Binimetinib Alone or Pembrolizumab Plus Chemotherapy With or Without Binimetinib in Metastatic Colorectal Cancer (mCRC) Participants (MK-3475-651/KEYNOTE-651)

NCT03374254
View directory record Official record
Conditions
Metastatic Colorectal Cancer
Interventions
Pembrolizumab, Binimetinib, Oxaliplatin, Leucovorin, 5-Fluorouracil [5-FU], Irinotecan
Biological · Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
18 Years and older
Enrollment
116 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
10
States / cities
Duarte, California • Aurora, Colorado • New Haven, Connecticut + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 21, 2024 · Synced Oct 22, 2026, 10:29 AM EDT
Completed Phase 2 Interventional Results available

A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD

NCT02451696
View directory record Official record
Conditions
Epilepsy, Tuberous Sclerosis Complex, Focal Cortical Dysplasia
Interventions
Everolimus
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
2 Years to 40 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 1, 2021 · Synced Oct 22, 2026, 10:29 AM EDT
Completed Not applicable Interventional Results available

Tourette Syndrome Deep Brain Stimulation

NCT02056873
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Continuous DBS, Responsive Brain Stimulation (RBS Setting)
Device
Lead sponsor
University of Florida
Other
Eligibility
21 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 5, 2024 · Synced Oct 22, 2026, 10:29 AM EDT
Terminated Phase 3 Interventional Results available

Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy

NCT05604170
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Ganaxolone
Drug
Lead sponsor
Marinus Pharmaceuticals
Industry
Eligibility
1 Year to 65 Years
Enrollment
117 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
20
States / cities
Little Rock, Arkansas • Los Angeles, California • Orange, California + 17 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2025 · Synced Oct 22, 2026, 10:29 AM EDT
Recruiting No phase listed Observational

Natural History Study of Patients With Neurofibromatosis Type 2

NCT00598351
View directory record Official record
Conditions
Neurofibromatosis
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
8 Years to 75 Years
Enrollment
269 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 22, 2026, 10:29 AM EDT
Terminated Phase 3 Interventional Results available

Long-Term Analgesic Efficacy And Safety Of Tanezumab Alone Or In Combination With Non-Steroidal Anti-Inflammatory Drugs (NSAIDs) Versus NSAIDs Alone In Patients With Osteoarthritis Of The Knee Or Hip

NCT00809354
View directory record Official record
Conditions
Osteoarthritis, Arthritis
Interventions
NSAID, tanezumab
Drug · Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 99 Years
Enrollment
2,720 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
250
States / cities
Birmingham, Alabama • Huntsville, Alabama • Mobile, Alabama + 161 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2021 · Synced Oct 22, 2026, 10:29 AM EDT
Not yet recruiting Phase 2 Interventional

Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome

NCT07328529
View directory record Official record
Conditions
Aggressive Behavior in Fragile X Syndome
Interventions
SRX246, Placebo
Drug
Lead sponsor
Azevan Pharmaceuticals
Industry
Eligibility
18 Years to 45 Years · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Sacramento, California • Chicago, Illinois • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 9, 2026 · Synced Oct 22, 2026, 10:29 AM EDT
Active, not recruiting Phase 2 Interventional

GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease

NCT05686551
View directory record Official record
Conditions
Huntington Disease
Interventions
Tominersen, Placebo
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
25 Years to 50 Years
Enrollment
301 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
18
States / cities
Birmingham, Alabama • Phoenix, Arizona • Sacramento, California + 15 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Oct 22, 2026, 10:29 AM EDT
Recruiting Phase 2 Interventional

Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas

NCT05253131
View directory record Official record
Conditions
MPNST, NF1, Sarcoma
Interventions
Selumetinib + ZEN-3694 ± Durvalumab
Combination Product
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years to 99 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Oct 22, 2026, 10:29 AM EDT
Withdrawn No phase listed Observational

Prospective Identification of Cardiac Amyloidosis by Cardiac Magnetic Resonance Imaging

NCT02462213
View directory record Official record
Conditions
Amyloidosis, Paraproteinemias, Cardiomyopathies
Interventions
Amyloidosis, Cardiac Amyloidosis
Other
Lead sponsor
University of Cincinnati
Other
Eligibility
18 Years to 85 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 26, 2017 · Synced Oct 22, 2026, 10:29 AM EDT
Completed Early Phase 1 Interventional

Pimavanserin Treatment in TS

NCT04794413
View directory record Official record
Conditions
Tourette Syndrome, Tardive Dyskinesia
Interventions
Pimavanserin
Drug
Lead sponsor
Joseph Jankovic
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 12, 2021 · Synced Oct 22, 2026, 10:29 AM EDT
Terminated No phase listed Observational

Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia

NCT05008874
View directory record Official record
Conditions
AMN, AMN Gene Mutation, X-ALD
Interventions
Natural History Observation
Other
Lead sponsor
SwanBio Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
4
States / cities
Stanford, California • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Oct 22, 2026, 10:29 AM EDT
Not yet recruiting Phase 2 Interventional

GV101 for Friedreich's Ataxia (FA) Treatment

NCT07870590
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
GV101, Placebo
Drug
Lead sponsor
Graviton Bioscience Corporation
Industry
Eligibility
16 Years to 40 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 22, 2026, 10:29 AM EDT
Completed Phase 2 Interventional Results available

Study in PRE-manifest Huntington's Disease of Coenzyme Q10 (UbiquinonE) Leading to Preventive Trials (PREQUEL)

NCT00920699
View directory record Official record
Conditions
Huntington's Disease
Interventions
CoQ10
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
13
States / cities
Sacramento, California • Englewood, Colorado • Miami, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2017 · Synced Oct 22, 2026, 10:29 AM EDT
Recruiting Phase 3 Interventional

Pridopidine Phase 3 Study in Huntington's Disease

NCT07609108
View directory record Official record
Conditions
Huntington Disease
Interventions
Pridopidine, Placebo
Drug
Lead sponsor
Prilenia
Industry
Eligibility
23 Years to 65 Years
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
25
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 21 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 22, 2026, 10:29 AM EDT
Recruiting No phase listed Observational

Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension

NCT07700225
View directory record Official record
Conditions
DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1, Myotonic Dystrophy Type-1, Myotonic Dystrophy, Type 1 (DM1), Myotonic Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
18 Years to 70 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 22, 2026, 10:29 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Speech-in-noise Perception in Autism and Fragile X

NCT06088589
View directory record Official record
Conditions
Autism Spectrum Disorder, Fragile X Syndrome
Interventions
Mismatch negativity
Behavioral
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
15 Years to 35 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2026
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 19, 2026 · Synced Oct 22, 2026, 10:29 AM EDT
Withdrawn Not applicable Interventional Accepts healthy volunteers

Telemedicine Clinical Trial for Cognitive Behavioral Therapy in Familial Dysautonomia

NCT03911063
View directory record Official record
Conditions
Familial Dysautonomia
Interventions
CBT sessions, Talking Sessions
Behavioral
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years to 80 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 28, 2020 · Synced Oct 22, 2026, 10:29 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy

NCT01594853
View directory record Official record
Conditions
X-linked Adrenoleukodystrophy
Interventions
exercise training
Behavioral
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
21 Years to 70 Years · Female only
Enrollment
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 29, 2018 · Synced Oct 22, 2026, 10:29 AM EDT
Terminated No phase listed Observational

Pharmacokinetic (PK) and Pharmacodynamics (PD) Study of Ilera Specific Products

NCT03886753
View directory record Official record
Conditions
ALS, Autism Spectrum Disorder, Cancer, Spasticity, Muscle, Dyskinetic Syndrome, Epilepsy, Glaucoma, Huntington Disease, Inflammatory Bowel Disease (IBD), Multiple Sclerosis, Neuropathy, Opioid Use, Parkinson Disease, HIV/AIDS, Ptsd, Intractable Pain, Sickle Cell Disease, Terminal Illness
Interventions
Registry, PK microsampling of blood
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Oct 22, 2026, 10:29 AM EDT
Active, not recruiting Phase 1 Interventional

A Phase I Study, Evaluating the Safety, Pharmacokinetics and Efficacy of PRJ1-3024 in Subjects With Advanced Solid Tumors

NCT05159700
View directory record Official record
Conditions
Advanced Solid Tumor, Advanced Solid Malignancies
Interventions
PRJ1-3024
Drug
Lead sponsor
Zhuhai Yufan Biotechnologies Co., Ltd
Industry
Eligibility
18 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
6
States / cities
Denver, Colorado • Orlando, Florida • Cincinnati, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 22, 2026, 10:29 AM EDT
Completed Phase 2 Interventional Results available

Phase 2 Study of Bevacizumab in Children and Young Adults With NF 2 and Progressive Vestibular Schwannomas

NCT01767792
View directory record Official record
Conditions
Neurofibromatosis Type 2, Progressive Vestibular Schwannomas
Interventions
Bevacizumab
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
6 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
12
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Atlanta, Georgia + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 12, 2021 · Synced Oct 22, 2026, 10:29 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Androgen Receptor, Implications for Health and Wellbeing: Natural History Study of Individuals With Androgen Insensitivity

NCT04708431
View directory record Official record
Conditions
Androgen Insensitivity Syndrome, Metabolic Parameters in AIS, CAIS, PAIS and MAIS, Tumor Formation in AIS, CAIS, PAIS and MAIS, Sexual Function AIS, CAIS, PAIS and MAIS
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
8 Years to 99 Years
Enrollment
650 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2040
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 20, 2026 · Synced Oct 22, 2026, 10:29 AM EDT
Completed Phase 3 Interventional

Study of Tetrathiomolybdate in Patients With Wilson Disease

NCT00004339
View directory record Official record
Conditions
Wilson Disease
Interventions
tetrathiomolybdate, trientine
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1994
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated May 9, 2006 · Synced Oct 22, 2026, 10:29 AM EDT