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ClinicalTrials.gov public records Last synced Oct 4, 2026, 5:34 PM EDT

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

Showing 73–96 of 1,705 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Terminated Early Phase 1 Interventional

Permeability MRI in Cerebral Cavernous Malformations Type 1 in New Mexico: Effects of Statins

NCT01764451
View directory record Official record
Conditions
Cavernous Angioma, Familial, Cerebral Cavernous Malformations, Cerebral Cavernous Hemangioma
Interventions
Simvastatin
Drug
Lead sponsor
University of New Mexico
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Albuquerque, New Mexico
Source: ClinicalTrials.gov public record
Updated Mar 12, 2024 · Synced Oct 4, 2026, 5:34 PM EDT
Completed Phase 2Phase 3 Interventional Results available

A Trial of Levodopa in Angelman Syndrome

NCT01281475
View directory record Official record
Conditions
Angelman Syndrome
Interventions
Levodopa, Placebo Oral Capsule
Drug
Lead sponsor
Wen-Hann Tan
Other
Eligibility
4 Years to 12 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
7
States / cities
San Diego, California • San Francisco, California • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2020 · Synced Oct 4, 2026, 5:34 PM EDT
Recruiting No phase listed Observational

Toward Ubiquitous Lower Limb Exoskeleton Use in Children and Young Adults

NCT06998134
View directory record Official record
Conditions
Muscle Weakness, Problems Moving Their Arms and Legs
Interventions
Extension assist knee ankle foot orthosis (EA-KAFO)
Device
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
5 Years to 25 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 23, 2026 · Synced Oct 4, 2026, 5:34 PM EDT
Not listed Phase 4 Interventional

A Pilot Study Assessing Impulsivity in Patients With Huntington's Disease on Xenazine (Tetrabenazine)

NCT02509793
View directory record Official record
Conditions
Huntington's Disease
Interventions
Tetrabenazine
Drug
Lead sponsor
William Ondo, MD
Other
Eligibility
18 Years to 80 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 2, 2022 · Synced Oct 4, 2026, 5:34 PM EDT
Completed Phase 3 Interventional Results available

Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

NCT00272792
View directory record Official record
Conditions
Phenylketonurias
Interventions
Sapropterin Dihydrochloride, Placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
4 Years to 12 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006
U.S. locations
9
States / cities
Los Angeles, California • Oakland, California • Sacramento, California + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2015 · Synced Oct 4, 2026, 5:34 PM EDT
Completed Phase 3 Interventional Results available

Safety and Tolerability of Once-daily Oral Aripiprazole in Children and Adolescents With Tourette's Disorder

NCT01727713
View directory record Official record
Conditions
Tourette's Disorder, Tic Disorder
Interventions
Aripiprazole
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
7 Years to 18 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
26
States / cities
Dothan, Alabama • Goodyear, Arizona • Orange, California + 23 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2015 · Synced Oct 4, 2026, 5:34 PM EDT
Completed Not applicable Interventional

Theta Burst Stimulation to Improve Inhibitory Motor Physiology in Tourette Syndrome

NCT05628805
View directory record Official record
Conditions
Tourette Syndrome, Tourette Syndrome in Children, Tourette Syndrome in Adolescence, Tourette Syndrome, Modifier of
Interventions
iTBS
Device
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
10 Years to 21 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Oct 4, 2026, 5:34 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA)

NCT02316314
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Cardiac magnetic resonance imaging (CMR), Exercise-stress test, Echocardiogram (ECHO), Cardiac-related blood studies
Procedure
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
12 Years to 50 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 4, 2026, 5:34 PM EDT
Completed No phase listed Observational

The Natural History of Metachromatic Leukodystrophy Study (HOME Study)

NCT04628364
View directory record Official record
Conditions
Metachromatic Leukodystrophy
Interventions
Not listed
Lead sponsor
National Organization for Rare Disorders
Other
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Danbury, Connecticut
Source: ClinicalTrials.gov public record
Updated Jun 8, 2025 · Synced Oct 4, 2026, 5:34 PM EDT
Recruiting No phase listed Observational

Hepatic Histopathology in Urea Cycle Disorders

NCT04908319
View directory record Official record
Conditions
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia, Carbamyl Phosphate Synthetase Deficiency, NAGS Deficiency
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 20, 2025 · Synced Oct 4, 2026, 5:34 PM EDT
Recruiting Phase 3 Interventional

KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases

NCT06451757
View directory record Official record
Conditions
Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
Interventions
Sonlicromanol, Placebo
Drug
Lead sponsor
Khondrion BV
Industry
Eligibility
18 Years and older
Enrollment
220 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Boston, Massachusetts • Cleveland, Ohio • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Oct 4, 2026, 5:34 PM EDT
Recruiting Phase 2 Interventional

UCSD Image-Guided Cognitive-Sparing Radiosurgery for Intracranial Lesions

NCT04343157
View directory record Official record
Conditions
Brain Metastases, Adult, Neurocognitive Function, Cancer, Meningioma of Brain, Arteriovenous Malformations, Cerebral
Interventions
Cognitive Sparing Brain Stereotactic Radiosurgery (SRS)
Radiation
Lead sponsor
Jona Hattangadi-Gluth
Other
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 4, 2026, 5:34 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome

NCT02679079
View directory record Official record
Conditions
Tourette Syndrome
Interventions
NBI-98854, Placebo
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
6 Years to 17 Years
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
44
States / cities
Sun City, Arizona • Springdale, Arkansas • Long Beach, California + 41 more
Source: ClinicalTrials.gov public record
Updated Jan 15, 2021 · Synced Oct 4, 2026, 5:34 PM EDT
Completed Phase 2 Interventional Results available

Study of MK-3475 (Pembrolizumab) in Patients With Microsatellite Unstable (MSI) Tumors (Cohort D)

NCT04098068
View directory record Official record
Conditions
High Tumor Mutation Burden, High TMB (Tumor Mutation Burden), MSS (Microsatellite Stable)
Interventions
MK-3475
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
3
States / cities
Baltimore, Maryland • New York, New York • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 30, 2025 · Synced Oct 4, 2026, 5:34 PM EDT
No Longer Available No phase listed Expanded access

Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

NCT00962260
View directory record Official record
Conditions
Gaucher Disease
Interventions
Plant cell expressed recombinant glucocerebrosidase (prGCD)
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
U.S. locations
14
States / cities
La Jolla, California • Aurora, Colorado • Coral Springs, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 7, 2018 · Synced Oct 4, 2026, 5:34 PM EDT
Completed Phase 2 Interventional Results available

Ecopipam Tablets to Study Tourette's Syndrome in Children and Adolescents

NCT04007991
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Ecopipam, Placebo
Drug
Lead sponsor
Emalex Biosciences Inc.
Industry
Eligibility
6 Years to 17 Years
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
53
States / cities
Dothan, Alabama • Phoenix, Arizona • Anaheim, California + 45 more
Source: ClinicalTrials.gov public record
Updated Oct 4, 2023 · Synced Oct 4, 2026, 5:34 PM EDT
Terminated Phase 1 Interventional

Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

NCT05222178
View directory record Official record
Conditions
Phenylketonurias, PAH Deficiency, Phenylketonuria
Interventions
HMI-103
Drug
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
2
States / cities
Topeka, Indiana • Lancaster, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 10, 2023 · Synced Oct 4, 2026, 5:34 PM EDT
Recruiting No phase listed Observational

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

NCT01665326
View directory record Official record
Conditions
Pompe Disease
Interventions
Observational
Other
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2029
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 4, 2026, 5:34 PM EDT
Completed Not applicable Interventional Results available

Augmentation of Brief Habit Reversal Training With D-cycloserine or Placebo

NCT02582515
View directory record Official record
Conditions
Tourette Disorder, Chronic Tic Disorder
Interventions
D-cycloserine, Placebo
Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
8 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 25, 2019 · Synced Oct 4, 2026, 5:34 PM EDT
Completed Phase 1 Interventional Results available

Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease

NCT01065558
View directory record Official record
Conditions
Lesch-Nyhan Disease
Interventions
Ecopipam
Drug
Lead sponsor
Psyadon Pharma
Industry
Eligibility
6 Years to 65 Years · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
2
States / cities
San Diego, California • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 18, 2013 · Synced Oct 4, 2026, 5:34 PM EDT
Not listed Phase 1 Interventional

Nilotinib in Huntington's Disease

NCT03764215
View directory record Official record
Conditions
Huntington Disease
Interventions
Nilotinib 150 MG
Drug
Lead sponsor
Georgetown University
Other
Eligibility
25 Years to 90 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 20, 2020 · Synced Oct 4, 2026, 5:34 PM EDT
Not yet recruiting No phase listed Observational

Multi-Modal Digital Monitoring of Disease Symptoms in Spinocerebellar Ataxia

NCT07851376
View directory record Official record
Conditions
Spinocerebellar Ataxia (SCA)
Interventions
Not listed
Lead sponsor
BioSensics
Industry
Eligibility
18 Years to 75 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
2
States / cities
Lutherville, Maryland • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 4, 2026, 5:34 PM EDT
Completed Phase 2 Interventional

Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria

NCT00104260
View directory record Official record
Conditions
Phenylketonurias
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
8 Years and older
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
13
States / cities
Los Angeles, California • Oakland, California • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Apr 9, 2007 · Synced Oct 4, 2026, 5:34 PM 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 Sep 30, 2026 · Synced Oct 4, 2026, 5:34 PM EDT