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ClinicalTrials.gov public records Last synced Oct 15, 2026, 7:48 PM EDT

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Showing 745–768 of 1,709 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Terminated Phase 2 Interventional Results available

Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)

NCT04475549
View directory record Official record
Conditions
MELAS
Interventions
IW-6463 Tablets
Drug
Lead sponsor
Tisento Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Baltimore, Maryland • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 29, 2024 · Synced Oct 15, 2026, 7:48 PM EDT
Recruiting No phase listed Observational

Systemic Biomarkers of Brain Injury From Hyperammonemia

NCT04602325
View directory record Official record
Conditions
Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I, Fatty Acid Oxidation Disorder, Hypoxic-Ischemic Encephalopathy
Interventions
Not listed
Lead sponsor
Children's National Research Institute
Other
Eligibility
7 Years to 18 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Oct 15, 2026, 7:48 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Sensorimotor and Psychosocial Trajectories in Adolescents With Tic Disorder

NCT06576726
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorder
Interventions
This study involves no intervention.
Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
11 Years to 17 Years
Enrollment
351 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2030
U.S. locations
1
States / cities
Nashvile, Tennessee
Source: ClinicalTrials.gov public record
Updated Nov 24, 2025 · Synced Oct 15, 2026, 7:48 PM EDT
Not listed Phase 3 Interventional

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

NCT04860960
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
Hydroxypropyl-beta-cyclodextrin, Placebo
Drug
Lead sponsor
Cyclo Therapeutics, Inc.
Industry
Eligibility
3 Years and older
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
7
States / cities
Oakland, California • Jacksonville, Florida • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2024 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Not applicable Interventional

Treadmill Walking in Individuals With Dementia With Lewy Bodies and Huntington's Disease

NCT02268617
View directory record Official record
Conditions
Huntington Disease, Lewy Body Disease
Interventions
Treadmill Walking
Other
Lead sponsor
Ohio State University
Other
Eligibility
18 Years to 90 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 27, 2016 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 2 Interventional

Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia

NCT01350440
View directory record Official record
Conditions
Spinocerebellar Ataxia
Interventions
IVIG
Biological
Lead sponsor
University of South Florida
Other
Eligibility
10 Years to 50 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jul 23, 2013 · Synced Oct 15, 2026, 7:48 PM EDT
Terminated Phase 4 Interventional Results available

Immune Tolerance Induction Study

NCT00701701
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
Myozyme® (alglucosidase alfa)
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
1 Month and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2020
U.S. locations
4
States / cities
Louisville, Kentucky • Durham, North Carolina • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2022 · Synced Oct 15, 2026, 7:48 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

University of Central Florida CereBank

NCT06876857
View directory record Official record
Conditions
Biospecimen Donation, Brain Injury, Brain Diseases
Interventions
Not listed
Lead sponsor
University of Central Florida
Other
Eligibility
18 Years and older · Female only
Enrollment
50,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2075
U.S. locations
1
States / cities
Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Mar 14, 2025 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 1 Interventional

Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease

NCT00316498
View directory record Official record
Conditions
Niemann Pick Diseases
Interventions
OGT918
Drug
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
12 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
2
States / cities
Bethesda, Maryland • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers

First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria

NCT05781399
View directory record Official record
Conditions
Phenylketonuria
Interventions
JNT-517 Suspension, Placebo Suspension, JNT-517 Tablet, Placebo Tablet
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
163 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
11
States / cities
Gainesville, Florida • Tampa, Florida • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Oct 15, 2026, 7:48 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Cooperative Huntington's Observational Research Trial

NCT00313495
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
HP Therapeutics Foundation
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2020
U.S. locations
41
States / cities
Birmingham, Alabama • Los Angeles, California • Sacramento, California + 35 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2011 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

AZD2014 In NF2 Patients With Progressive or Symptomatic Meningiomas

NCT02831257
View directory record Official record
Conditions
Neurofibromatosis 2, Meningioma
Interventions
AZD2014
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2020
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 13, 2022 · Synced Oct 15, 2026, 7:48 PM EDT
Completed No phase listed Observational

VAlidation of a Lower Cost aneUploidy scrEen

NCT03087357
View directory record Official record
Conditions
Down Syndrome, Trisomy 18, Trisomy 13
Interventions
SmartNIPT
Other
Lead sponsor
Women and Infants Hospital of Rhode Island
Other
Eligibility
18 Years and older · Female only
Enrollment
2,443 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
13
States / cities
Los Angeles, California • Aurora, Colorado • Bridgeport, Connecticut + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2022 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 2 Interventional Results available

Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840

NCT04422431
View directory record Official record
Conditions
Wilson Disease
Interventions
Bis-Choline Tetrathiomolybdate
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
3
States / cities
Sacramento, California • Ann Arbor, Michigan • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 18, 2024 · Synced Oct 15, 2026, 7:48 PM EDT
Recruiting No phase listed Observational

Natural History Study of Patients with HPDL Mutations

NCT05848271
View directory record Official record
Conditions
Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia, Spastic Paraplegia, White Matter Disease, Neonatal Encephalopathy, Mutation, Genetic Disease
Interventions
Patient Registry, Dry blood spots sampling
Other
Lead sponsor
University of California, San Diego
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 30, 2025 · Synced Oct 15, 2026, 7:48 PM EDT
Enrolling by invitation Phase 2 Interventional

Sirolimus for Leigh Syndrome

NCT06843811
View directory record Official record
Conditions
Leigh Syndrome
Interventions
Sirolimus
Drug
Lead sponsor
Matthew Demczko
Other
Eligibility
6 Months to 55 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 31, 2026 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 2 Interventional Results available

Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease

NCT01196871
View directory record Official record
Conditions
Fabry Disease
Interventions
Migalastat HCl, Agalsidase Beta, Agalsidase Alfa
Drug · Biological
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
5
States / cities
Birmingham, Alabama • Decatur, Georgia • Iowa City, Iowa + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 19, 2018 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia

NCT04117711
View directory record Official record
Conditions
Classic Galactosemia
Interventions
AT-007, Placebo
Drug
Lead sponsor
Applied Therapeutics, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
114 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2021
U.S. locations
4
States / cities
Anaheim, California • Atlanta, Georgia • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated May 30, 2024 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 3 Interventional Results available

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

NCT01074944
View directory record Official record
Conditions
Gaucher Disease
Interventions
Eliglustat tartrate
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
170 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
10
States / cities
San Diego, California • New Haven, Connecticut • Decatur, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2017 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Not applicable Interventional Results available

Low Cost OCT for Point of Care

NCT03323307
View directory record Official record
Conditions
Optical Coherence Tomography (OCT) of the Retina
Interventions
OCT imaging
Device
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 5, 2019 · Synced Oct 15, 2026, 7:48 PM EDT
Withdrawn No phase listed Observational

A Study of the Natural Course of SURF1 Deficiency

NCT05277363
View directory record Official record
Conditions
Leigh Syndrome
Interventions
None (Observational study)
Other
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
Up to 18 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated May 20, 2022 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy of tPBM for Epileptiform Activity in Autism

NCT06352372
View directory record Official record
Conditions
Autism Spectrum Disorder, EEG With Periodic Abnormalities, Epilepsy, Neurodevelopmental Disorders, Neurological Disorder
Interventions
noninvasively delivered near infra-red (NIR) light - transcranial Photobiomodulation (tPBM)
Device
Lead sponsor
Richard Frye
Other
Eligibility
4 Years to 16 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 15, 2026, 7:48 PM EDT
Terminated Phase 1Phase 2 Interventional

Liver Cell Transplant for Phenylketonuria

NCT01465100
View directory record Official record
Conditions
Phenylketonuria
Interventions
Preparative Radiation Therapy, Hepatocyte Transplant, Immunosuppression, Liver Evaluation, Neuro-psychological Assessment, Whole body Phe oxidation testing, Liver Biopsy
Radiation · Procedure · Drug + 3 more
Lead sponsor
Ira Fox
Other
Eligibility
14 Years to 55 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 16, 2023 · Synced Oct 15, 2026, 7:48 PM EDT
Completed Phase 3 Interventional Results available

Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity

NCT03746522
View directory record Official record
Conditions
Bardet Biedl Syndrome (BBS), Alström Syndrome (AS)
Interventions
Setmelanotide, Placebo
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
6
States / cities
San Diego, California • Springfield, Massachusetts • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2023 · Synced Oct 15, 2026, 7:48 PM EDT