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ClinicalTrials.gov public records Last synced Oct 16, 2026, 5:32 AM EDT

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

Showing 793–816 of 1,709 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Terminated No phase listed Observational

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

NCT04909346
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
Interventions
Not listed
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Akron, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 12, 2023 · Synced Oct 16, 2026, 5:32 AM EDT
Completed No phase listed Observational

Metabolic Abnormalities in Children With Epilepsy

NCT00001325
View directory record Official record
Conditions
Generalized Epilepsy, Infantile Spasms, Metabolic Disease, Partial Epilepsy, Seizures
Interventions
18 FDG
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 16, 2026, 5:32 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 16, 2026, 5:32 AM EDT
Recruiting No phase listed Observational

International Congenital Central Hypoventilation Syndrome (CCHS) Registry and CCHS SHARE

NCT03088020
View directory record Official record
Conditions
Congenital Central Hypoventilation Syndrome
Interventions
Not listed
Lead sponsor
Ilya Khaytin
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2032
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 16, 2026, 5:32 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Biomarker Analysis for GBA Associated Parkinson's Disease

NCT03811496
View directory record Official record
Conditions
Parkinson Disease, Gaucher Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
50 Years and older
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jan 23, 2019 · Synced Oct 16, 2026, 5:32 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

NCT01694940
View directory record Official record
Conditions
Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2026
U.S. locations
16
States / cities
San Diego, California • Stanford, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 16, 2026, 5:32 AM EDT
Completed No phase listed Observational

Journey of Patients With Vasculitis From First Symptom to Diagnosis

NCT03410290
View directory record Official record
Conditions
Vasculitis, Systemic Vasculitis, Behcet's Disease, CNS Vasculitis, Cryoglobulinemic Vasculitis, Eosinophilic Granulomatous Vasculitis, Temporal Arteritis, Giant Cell Arteritis, Granulomatosis With Polyangiitis, Wegener Granulomatosis, Henoch Schonlein Purpura, IgA Vasculitis, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu Arteritis, Urticarial Vasculitis
Interventions
Online Questionnaire
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
456 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jun 6, 2018 · Synced Oct 16, 2026, 5:32 AM EDT
Enrolling by invitation Not applicable Interventional

Group Coaching Study for Life Goals

NCT07083713
View directory record Official record
Conditions
Students, Spinal Cord Injury, Ehlers Danlos Syndrome, Care Givers
Interventions
Group Coaching
Behavioral
Lead sponsor
Thomas Jefferson University
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 24, 2025 · Synced Oct 16, 2026, 5:32 AM EDT
Withdrawn Phase 3 Interventional

Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy

NCT04418024
View directory record Official record
Conditions
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Interventions
AG10, Placebo
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 90 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 16, 2026, 5:32 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

A More Engaging Visual Field Test to Increase Use and Reliability in Pediatrics

NCT02157025
View directory record Official record
Conditions
Glaucoma, Suspect, Optic Nerve Hypoplasia and Abnormalities of the Central Nervous System, Congenital Coloboma of the Optic Nerve, Optic Nerve Head Pits, Bilateral Congenital
Interventions
Cartoon video fixation target and cartoon character voice audio instructions during Humphrey perimetry, Usual Care procedures during Humphrey perimetry for children
Device · Other
Lead sponsor
Nova Southeastern University
Other
Eligibility
5 Years to 8 Years
Enrollment
11 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2026
U.S. locations
1
States / cities
Fort Lauderdale, Florida
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 16, 2026, 5:32 AM EDT
Recruiting No phase listed Observational

Phenotype/Genotype Correlations in Movement Disorders

NCT00018889
View directory record Official record
Conditions
Movement Disorder
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
2 Years to 100 Years
Enrollment
2,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 16, 2026, 5:32 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

NCT03737214
View directory record Official record
Conditions
Fabry Disease
Interventions
Lucerastat
Drug
Lead sponsor
Idorsia Pharmaceuticals Ltd.
Industry
Eligibility
18 Years and older
Enrollment
107 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2029
U.S. locations
14
States / cities
Birmingham, Alabama • Irvine, California • Oakland, California + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 16, 2026, 5:32 AM EDT
Recruiting Phase 3 Interventional

A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia

NCT07778836
View directory record Official record
Conditions
Friedreich Ataxia, Friedreich's Ataxia
Interventions
Nomlabofusp, Placebo
Drug
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
12 Years to 40 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
4
States / cities
Tampa, Florida • Chicago, Illinois • Eatontown, New Jersey + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2026 · Synced Oct 16, 2026, 5:32 AM EDT
Completed Phase 2 Interventional Results available

Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders

NCT00947544
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100, NaPBA
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
7
States / cities
Los Angeles, California • Washington D.C., District of Columbia • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2024 · Synced Oct 16, 2026, 5:32 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Brain Activation in Vocal and Motor Tics

NCT00026000
View directory record Official record
Conditions
Tourette's Syndrome, Chronic Motor Tic Disorder, Chronic Motor Vocal Disorder
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 16, 2026, 5:32 AM EDT
Completed Phase 3 Interventional

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

NCT05156320
View directory record Official record
Conditions
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
Interventions
Apitegromab, Placebo
Drug
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
2 Years to 21 Years
Enrollment
188 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
26
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Oct 16, 2026, 5:32 AM EDT
Terminated Phase 3 Interventional Results available

An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome

NCT03882918
View directory record Official record
Conditions
Angelman Syndrome
Interventions
OV101
Drug
Lead sponsor
Healx AI
Industry
Eligibility
13 Years to 49 Years
Enrollment
141 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
10
States / cities
Phoenix, Arizona • San Diego, California • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 18, 2025 · Synced Oct 16, 2026, 5:32 AM EDT
Completed Not applicable Interventional

Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.

NCT02654886
View directory record Official record
Conditions
Pompe Disease, Muscle Weakness
Interventions
Resistance Exercise Training
Other
Lead sponsor
University of California, Irvine
Other
Eligibility
16 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Sep 26, 2019 · Synced Oct 16, 2026, 5:32 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I

NCT00439218
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
Westat
Other
Eligibility
2 Months to 48 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
5
States / cities
Stanford, California • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 9, 2010 · Synced Oct 16, 2026, 5:32 AM EDT
Completed Phase 2 Interventional

A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria

NCT06637514
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
JNT-517 Tablet, Placebo
Drug · Other
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
12 Years to 17 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
6
States / cities
Gainesville, Florida • Tampa, Florida • Portland, Oregon + 3 more
Source: ClinicalTrials.gov public record
Updated May 13, 2026 · Synced Oct 16, 2026, 5:32 AM EDT
Active, not recruiting No phase listed Observational

Spinraza in Adult Spinal Muscular Atrophy

NCT03709784
View directory record Official record
Conditions
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type 3
Interventions
Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care, One time survey
Drug · Other
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 70 Years
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
10
States / cities
Phoenix, Arizona • Washington D.C., District of Columbia • Baltimore, Maryland + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 20, 2024 · Synced Oct 16, 2026, 5:32 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Alternatives for Reducing Tics in Tourette Syndrome (TS): A Study of TEV-50717 (Deutetrabenazine) for the Treatment of Tourette Syndrome in Children and Adolescents

NCT03452943
View directory record Official record
Conditions
Tourette Syndrome
Interventions
TEV-50717, Placebo
Drug
Lead sponsor
Teva Branded Pharmaceutical Products R&D, Inc.
Industry
Eligibility
6 Years to 16 Years
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
27
States / cities
Dothan, Alabama • Sun City, Arizona • Rogers, Arkansas + 22 more
Source: ClinicalTrials.gov public record
Updated Nov 9, 2021 · Synced Oct 16, 2026, 5:32 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Genetic Studies of Lysosomal Storage Disorders

NCT00001215
View directory record Official record
Conditions
Lysosomal Storage Disorders, Gaucher Disease, Parkinson Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Week to 110 Years
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1995
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 16, 2026, 5:32 AM EDT
Terminated Not applicable Interventional

Moderate Intensity Exercise and Phenylketonuria

NCT01904708
View directory record Official record
Conditions
Phenylketonuria, PKU
Interventions
Acute Moderate Intensity Exercise
Other
Lead sponsor
Oregon Health and Science University
Other
Eligibility
14 Years to 17 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 16, 2014 · Synced Oct 16, 2026, 5:32 AM EDT