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ClinicalTrials.gov public records Last synced Oct 28, 2026, 10:29 AM EDT

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Showing 1585–1608 of 1,713 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Completed Phase 4 Interventional Results available

A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease

NCT00074984
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta), Placebo
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
19
States / cities
Birmingham, Alabama • Los Angeles, California • San Francisco, California + 16 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2013 · Synced Oct 28, 2026, 10:29 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Neuroimaging of Dystonia

NCT02046447
View directory record Official record
Conditions
Primary Cervical Dystonia, DYT 1 Dystonia
Interventions
Primary Cervical Dystonia (Trihexyphenidyl), Controls Primary Cervical Dystonia (Trihexyphenidyl), DYT 1 Dystonia (Healthy Control), DYT 1 Dystonia
Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
7 Years to 70 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Dec 22, 2017 · Synced Oct 28, 2026, 10:29 AM EDT
Not listed Phase 2Phase 3 Interventional

MIST II PFO-Migraine Trial With BioSTAR® Bioabsorbable Septal Repair Implant

NCT00283738
View directory record Official record
Conditions
Migraine, Aura, Patent Foramen Ovale
Interventions
BioSTAR Septal Repair Implant System, Sham Procedure
Device · Procedure
Lead sponsor
NMT Medical
Industry
Eligibility
18 Years to 60 Years
Enrollment
610 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
2
States / cities
Stamford, Connecticut • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jan 25, 2008 · Synced Oct 28, 2026, 10:29 AM EDT
Withdrawn Phase 1Phase 2 Interventional

Mobile Phone App for Depression and Anxiety in Young Men Who Are Attracted to Men

NCT02072252
View directory record Official record
Conditions
Depressive Symptoms, Anxiety Generalized
Interventions
Mobile Phone Application
Behavioral
Lead sponsor
Northwestern University
Other
Eligibility
17 Years to 20 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 17, 2017 · Synced Oct 28, 2026, 10:29 AM EDT
Completed Phase 1 Interventional

Human Placental-Derived Stem Cell Transplantation

NCT01586455
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Interventions
Human Placental Derived Stem Cell
Drug
Lead sponsor
New York Medical College
Other
Eligibility
0 Years to 55 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
3
States / cities
Denver, Colorado • Valhalla, New York • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 25, 2022 · Synced Oct 28, 2026, 10:29 AM EDT
Completed Phase 2 Interventional Results available

Open-Label Safety and Tolerability Study of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

NCT03444038
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Valbenazine
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
6 Years to 18 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
36
States / cities
Sun City, Arizona • Anaheim, California • San Diego, California + 31 more
Source: ClinicalTrials.gov public record
Updated Feb 23, 2022 · Synced Oct 28, 2026, 10:29 AM EDT
Enrolling by invitation Phase 3 Interventional

A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)

NCT06431893
View directory record Official record
Conditions
Homocystinuria
Interventions
Pegtibatinase
Drug
Lead sponsor
Travere Therapeutics, Inc.
Industry
Eligibility
5 Years to 65 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
13
States / cities
Phoenix, Arizona • New Haven, Connecticut • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 28, 2026, 10:29 AM EDT
Completed No phase listed Observational

Cardiac Rhythm Abnormalities in Patients With Refractory Epilepsy at High Risk for Sudden Death

NCT00203333
View directory record Official record
Conditions
Refractory Epilepsy, Arrhythmia, Sudden Death
Interventions
Medtronic REVEAL
Device
Lead sponsor
Thomas Jefferson University
Other
Eligibility
16 Years to 49 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2010
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 18, 2016 · Synced Oct 28, 2026, 10:29 AM 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 28, 2026, 10:29 AM EDT
Terminated Phase 3 Interventional

A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy

NCT00545597
View directory record Official record
Conditions
Adrenomyeloneuropathy, Adrenoleukodystrophy
Interventions
Lorenzo's oil
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 2, 2012 · Synced Oct 28, 2026, 10:29 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals

NCT06117020
View directory record Official record
Conditions
Muscle Spasticity, Hypertonia, Muscle, Cerebral Palsy, Multiple Sclerosis, Stroke, Hereditary Spastic Paraplegia, Dystonia, Spinal Cord Injuries
Interventions
MTR-601
Drug
Lead sponsor
Motric Bio
Industry
Eligibility
18 Years to 45 Years
Enrollment
89 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2024
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Jun 5, 2026 · Synced Oct 28, 2026, 10:29 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease

NCT06609889
View directory record Official record
Conditions
Lafora Disease
Interventions
ION283
Genetic
Lead sponsor
Berge Minassian
Other
Eligibility
10 Years to 18 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Oct 28, 2026, 10:29 AM EDT
Completed Not applicable Interventional

A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD

NCT03911089
View directory record Official record
Conditions
Urea Cycle Disorder, Inborn Errors of Metabolism
Interventions
UCD Anamix Infant
Other
Lead sponsor
Nutricia Research
Industry
Eligibility
Up to 30 Days
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 9, 2021 · Synced Oct 28, 2026, 10:29 AM EDT
Withdrawn Phase 2 Interventional

High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease

NCT01656590
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
High Protein and Exercise Therapy along-with Nocturnal Enteral Feeding
Other
Lead sponsor
Columbia University
Other
Eligibility
8 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 13, 2015 · Synced Oct 28, 2026, 10:29 AM EDT
Completed Phase 2 Interventional

A Study of the Novel Drug Dimebon in Patients With Huntington's Disease

NCT00497159
View directory record Official record
Conditions
Huntington's Disease
Interventions
Placebo, Dimebon
Other · Drug
Lead sponsor
Medivation, Inc.
Industry
Eligibility
29 Years and older
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jun 8, 2015 · Synced Oct 28, 2026, 10:29 AM EDT
Terminated Phase 2 Interventional Accepts healthy volunteers Results available

Phenylketonuria, Oxidative Stress, and BH4

NCT01395394
View directory record Official record
Conditions
Phenylketonuria
Interventions
Kuvan, Meal Challenge
Drug · Other
Lead sponsor
Emory University
Other
Eligibility
10 Years to 45 Years
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Aug 28, 2014 · Synced Oct 28, 2026, 10:29 AM EDT
Completed Not applicable Interventional

Incorporating teleCBIT Into a Hospital-Based Tic Program

NCT04007913
View directory record Official record
Conditions
Tic Disorders, Tourette Syndrome
Interventions
teleCBIT
Behavioral
Lead sponsor
San Jose State University
Other
Eligibility
5 Years to 65 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
2
States / cities
San Jose, California • Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 5, 2019 · Synced Oct 28, 2026, 10:29 AM EDT
Active, not recruiting Phase 3 Interventional

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

NCT04910776
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
avalglucosidase alfa
Drug
Lead sponsor
Sanofi
Industry
Eligibility
0 Days to 12 Months
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
6
States / cities
Stanford, California • Minneapolis, Minnesota • Hawthorne, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 28, 2026, 10:29 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 28, 2026, 10:29 AM EDT
Recruiting Phase 1Phase 2 Interventional

Safety and Tolerability Study of Human Neural Stem Cells for Huntington's Disease

NCT07451613
View directory record Official record
Conditions
Huntington Disease
Interventions
hNSC-01
Biological
Lead sponsor
Leslie Thompson
Other
Eligibility
18 Years to 65 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 28, 2026, 10:29 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

Metabolic and Hemodynamic Reserve in Pediatric SCA

NCT04406818
View directory record Official record
Conditions
Child, Only, Brain Diseases, Sickle Cell Disease, Anemia, Sickle Cell
Interventions
Carbon Dioxide
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
4 Years to 21 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2027
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 28, 2026, 10:29 AM EDT
Completed Phase 1Phase 2 Interventional

Minocycline in Patients With Huntington's Disease

NCT00029874
View directory record Official record
Conditions
Huntington's Disease
Interventions
Minocycline
Drug
Lead sponsor
FDA Office of Orphan Products Development
Federal
Eligibility
18 Years and older
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 28, 2026, 10:29 AM EDT
Terminated Phase 1Phase 2 Interventional

A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency

NCT05154890
View directory record Official record
Conditions
Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
Interventions
Pegtarviliase IV, Pegtarviliase SC
Drug
Lead sponsor
Aeglea Biotherapeutics
Industry
Eligibility
12 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 27, 2023 · Synced Oct 28, 2026, 10:29 AM EDT
Recruiting Phase 2 Interventional

An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia

NCT06447025
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
CTI-1601
Drug
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
2 Years to 60 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
10
States / cities
Los Angeles, California • San Francisco, California • Gainesville, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 27, 2026 · Synced Oct 28, 2026, 10:29 AM EDT