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ClinicalTrials.gov public records Last synced Oct 15, 2026, 10:54 AM EDT

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Showing 601–624 of 1,709 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Completed Phase 2 Interventional Results available

An Open Label Study of ANX005 in Participants With, or at Risk for, Manifest Huntington's Disease

NCT04514367
View directory record Official record
Conditions
Huntington Disease
Interventions
ANX005
Drug
Lead sponsor
Annexon, Inc.
Industry
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
6
States / cities
Birmingham, Alabama • Englewood, Colorado • Washington D.C., District of Columbia + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Oct 15, 2026, 10:54 AM EDT
Completed No phase listed Observational

Data Collection in Women With Fabry Disease

NCT00030134
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Female only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 15, 2026, 10:54 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Gait Analysis in Neurological Disease

NCT02994719
View directory record Official record
Conditions
Parkinson's Disease, Parkinsonian Disorders, Atypical Parkinson Disease, Progressive Supranuclear Palsy, Multiple System Atrophy, Corticobasal Degeneration, Gait, Frontal, Huntington Disease, Ataxia, Spinocerebellar
Interventions
Anti-Parkinson medication, Deep Brain Stimulation
Drug · Device
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
18 Years to 85 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Oct 15, 2026, 10:54 AM EDT
Completed No phase listed Observational

PheCheck Feasibility Study

NCT05998109
View directory record Official record
Conditions
Phenylketonurias
Interventions
PheCheck™, HPLC Amino Acid Analyzer, Dried Blood Spot Cards
Device · Diagnostic Test
Lead sponsor
Lumos Diagnostics
Industry
Eligibility
10 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Oct 15, 2026, 10:54 AM EDT
Withdrawn Phase 2 Interventional

A Trial Evaluating the Efficacy, Safety, and Pharmacokinetics of SNC-102 in Subjects With Tourette Syndrome

NCT02217007
View directory record Official record
Conditions
Tourette Syndrome
Interventions
SNC-102 sustained release tablet
Drug
Lead sponsor
Synchroneuron Inc.
Industry
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Chicago, Illinois • Manhasset, New York
Source: ClinicalTrials.gov public record
Updated Apr 13, 2021 · Synced Oct 15, 2026, 10:54 AM EDT
Completed Phase 3 Interventional

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

NCT03614234
View directory record Official record
Conditions
Fabry Disease
Interventions
pegunigalsidase alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
7
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated May 22, 2026 · Synced Oct 15, 2026, 10:54 AM 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 15, 2026, 10:54 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Risk Factors, Costs, and Impacts of ED Boarding

NCT07532564
View directory record Official record
Conditions
Stroke (CVA) or TIA, Diabetes, Asthma (Diagnosis), COPD (Chronic Obstructive Pulmonary Disease), Sepsis, Hypertension, Heart Failure, Pneumonia, Urinary Tract Infection (Diagnosis), Chest Pain, Psychiatric Disorder, Sickle Cell Disease (SCD)
Interventions
Not listed
Lead sponsor
University of Maryland, Baltimore
Other
Eligibility
18 Years and older
Enrollment
30,486 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Oct 15, 2026, 10:54 AM EDT
Terminated Phase 3 Interventional Results available

Efficacy and Safety of Intravenous Phenobarbital in Neonatal Seizures

NCT04320940
View directory record Official record
Conditions
Epilepsy, Benign Neonatal
Interventions
Phenobarbital Sodium Injection
Drug
Lead sponsor
NEMA Research, Inc.
Industry
Eligibility
34 Weeks to 44 Weeks
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
7
States / cities
Little Rock, Arkansas • Washington D.C., District of Columbia • Miami, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 26, 2023 · Synced Oct 15, 2026, 10:54 AM EDT
Completed Phase 2 Interventional Results available

HSCT for High Risk Inherited Inborn Errors

NCT00383448
View directory record Official record
Conditions
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease, Sandhoffs Disease, Wolman Disease, I-Cell Disease, Sanfilippo Syndrome, GM1 Gangliosidosis
Interventions
Clofarabine, Total body Irradiation, Melphalan, Hematopoietic Stem Cell Transplantation, Alemtuzumab, mycophenylate mofetil, Cyclosporine A, Hydroxyurea
Drug · Procedure · Biological + 1 more
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 70 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 11, 2019 · Synced Oct 15, 2026, 10:54 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 15, 2026, 10:54 AM EDT
Recruiting No phase listed Observational

Prospective Research Rare Kidney Stones (ProRKS)

NCT02780297
View directory record Official record
Conditions
Hyperoxaluria, Cystinuria, Dent Disease, Lowe Syndrome, Adenine Phosphoribosyltransferase Deficiency
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
220 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
8
States / cities
Birmingham, Alabama • Jacksonville, Florida • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2026 · Synced Oct 15, 2026, 10:54 AM EDT
Completed Phase 1 Interventional

Developing Cognitive Training for Tourette Syndrome

NCT01440023
View directory record Official record
Conditions
Tourette Syndrome, Chronic Tic Disorders
Interventions
Comprehensive Behavioral Intervention for Tics (CBIT), Computerized Response Inhibition Training, Computerized Placebo Cognitive Training
Behavioral
Lead sponsor
University of Wisconsin, Milwaukee
Other
Eligibility
9 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 15, 2015 · Synced Oct 15, 2026, 10:54 AM EDT
Completed Phase 2 Interventional Results available

Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease

NCT02228460
View directory record Official record
Conditions
Fabry Disease
Interventions
GZ/SAR402671
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 49 Years · Male only
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
3
States / cities
Atlanta, Georgia • Cincinnati, Ohio • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 15, 2026, 10:54 AM EDT
Active, not recruiting No phase listed Observational

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

NCT04848779
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Alglucosidase alfa GZ419829
Drug
Lead sponsor
Sanofi
Industry
Eligibility
0 Days to 6 Months
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
5
States / cities
Hawthorne, New York • Durham, North Carolina • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Oct 15, 2026, 10:54 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Cerebral Oxygen Metabolism in Children

NCT04157179
View directory record Official record
Conditions
Sickle Cell Disease, Cerebral Stroke
Interventions
Carbon Dioxide
Other
Lead sponsor
Washington University School of Medicine
Other
Eligibility
3 Years to 50 Years
Enrollment
350 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2027
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Sep 11, 2025 · Synced Oct 15, 2026, 10:54 AM EDT
Completed No phase listed Observational

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

NCT01451879
View directory record Official record
Conditions
Pompe Disease
Interventions
Rituximab, Miglustat
Drug
Lead sponsor
University of Florida
Other
Eligibility
Up to 65 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Dec 8, 2017 · Synced Oct 15, 2026, 10:54 AM EDT
Completed Phase 3 Interventional

Caspofungin Acetate Compared With Amphotericin B Liposomal in Treating Patients With Persistent Fever and Neutropenia Following Cancer Treatment

NCT00008359
View directory record Official record
Conditions
Cancer
Interventions
caspofungin acetate, liposomal amphotericin B
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
16 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 18, 2013 · Synced Oct 15, 2026, 10:54 AM EDT
Completed Phase 3 Interventional Results available

Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia

NCT02797080
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
interferon γ-1b
Drug
Lead sponsor
Amgen
Industry
Eligibility
11 Years to 27 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
4
States / cities
Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced Oct 15, 2026, 10:54 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Gut Microbiomes in HD

NCT06448546
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
University of Central Florida
Other
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Jun 18, 2025 · Synced Oct 15, 2026, 10:54 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants

NCT04595513
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Epilepsy
Interventions
TAVT-18 (sirolimus)
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
1 Day to 6 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated May 16, 2024 · Synced Oct 15, 2026, 10:54 AM EDT
Completed No phase listed Observational

A Natural History of Late Onset Tay-Sachs Disease

NCT02851862
View directory record Official record
Conditions
GM2 Gangliosidosis
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
7 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 15, 2026, 10:54 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy

NCT01855360
View directory record Official record
Conditions
Amyloidosis; Heart (Manifestation), Senile Cardiac Amyloidosis
Interventions
Tauroursodeoxycholic Acid and Doxycycline
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 4, 2020 · Synced Oct 15, 2026, 10:54 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 15, 2026, 10:54 AM EDT