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ClinicalTrials.gov public records Last synced May 22, 2026, 3:47 AM EDT

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

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Brain Malformation Clear all
Completed No phase listed Observational
View directory record Official record
Conditions
Tourette Syndrome, Tourette Syndrome in Children, Tourette Syndrome in Adolescence, Tic Disorder, Childhood, Tics
Interventions
Not listed
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
8 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 9, 2025 · Synced May 22, 2026, 3:47 AM EDT
Terminated Phase 3 Interventional Results available
View directory record Official record
Conditions
Ataxia Telangiectasia, Genetic Syndrome
Interventions
EryDex System
Combination Product
Lead sponsor
Quince Therapeutics S.p.A.
Industry
Eligibility
6 Years and older
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Los Angeles, California • Baltimore, Maryland • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2024 · Synced May 22, 2026, 3:47 AM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
DT-216, DT-216 matching Placebo
Drug
Lead sponsor
Design Therapeutics, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Mar 27, 2023 · Synced May 22, 2026, 3:47 AM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 22, 2016 · Synced May 22, 2026, 3:47 AM EDT
Recruiting Not applicable Interventional
View directory record Official record
Conditions
Pseudohypoparathyroidism Type 1A, Albright Hereditary Osteodystrophy, Pseudopseudohypoparathyroidism
Interventions
Neurocognitive and psychosocial testing
Behavioral
Lead sponsor
Connecticut Children's Medical Center
Other
Eligibility
2 Months to 89 Years
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2030
U.S. locations
1
States / cities
Hartford, Connecticut
Source: ClinicalTrials.gov public record
Updated May 27, 2025 · Synced May 22, 2026, 3:47 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Wilson Disease
Interventions
UX701, Standard of Care (SOC)
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2034
U.S. locations
11
States / cities
Los Angeles, California • Redwood City, California • Sacramento, California + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced May 22, 2026, 3:47 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Shoji Yano
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jan 17, 2013 · Synced May 22, 2026, 3:47 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Pompe Disease
Interventions
respiratory muscle strength training
Other
Lead sponsor
Duke University
Other
Eligibility
3 Years to 99 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 29, 2023 · Synced May 22, 2026, 3:47 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Lafora Disease
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
5 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Los Angeles, California • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Sep 27, 2022 · Synced May 22, 2026, 3:47 AM EDT
Enrolling by invitation No phase listed Observational
View directory record Official record
Conditions
Lennox Gastaut Syndrome (LGS)
Interventions
fenfluramine HCl
Drug
Lead sponsor
University of Chicago
Other
Eligibility
2 Years to 35 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced May 22, 2026, 3:47 AM EDT
Active, not recruiting Phase 2 Interventional Results available
View directory record Official record
Conditions
Metachromatic Leukodystrophy (MLD)
Interventions
SHP611
Drug
Lead sponsor
Shire
Industry
Eligibility
6 Months to 72 Months
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
11
States / cities
Torrance, California • Aurora, Colorado • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced May 22, 2026, 3:47 AM EDT
Recruiting Not applicable Interventional
View directory record Official record
Conditions
Tetraplegia, Spinal Cord Injuries, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked in Syndrome, Muscular Dystrophy
Interventions
Placement of the BrainGate2 sensor(s) into the motor-related cortex
Device
Lead sponsor
Leigh R. Hochberg, MD, PhD.
Other
Eligibility
18 Years to 80 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2038
U.S. locations
5
States / cities
Sacramento, California • Stanford, California • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 2, 2025 · Synced May 22, 2026, 3:47 AM EDT
Not listed No phase listed Observational
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 12, 2019 · Synced May 22, 2026, 3:47 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Hemianopia, Hemianopsia, Peripheral Visual Field Defect, Blindness, Retinitis Pigmentosa, Glaucoma
Interventions
Collision warning device
Device
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
18 Years and older
Enrollment
49 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 9, 2021 · Synced May 22, 2026, 3:47 AM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Wilson Disease
Interventions
Not listed
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
7
States / cities
Los Angeles, California • Sacramento, California • New Haven, Connecticut + 4 more
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced May 22, 2026, 3:47 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Unverricht-Lundborg Disease
Interventions
Placebo, BRV 2.5 mg, BRV 25 mg, BRV 50 mg
Other · Drug
Lead sponsor
UCB Pharma
Industry
Eligibility
16 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
4
States / cities
San Francisco, California • Gainesville, Florida • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated May 30, 2023 · Synced May 22, 2026, 3:47 AM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
Prion Disease
Interventions
PrP-siRNA
Drug
Lead sponsor
Broad Institute of MIT and Harvard
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
4
States / cities
Boston, Massachusetts • Rochester, Minnesota • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated May 10, 2026 · Synced May 22, 2026, 3:47 AM EDT
Completed Phase 1 Interventional
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 14, 2015 · Synced May 22, 2026, 3:47 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Behcet's Disease, Churg-Strauss Syndrome, Vasculitis, Central Nervous System, Giant Cell Arteritis, Wegener Granulomatosis, Henoch-Schoenlein Purpura, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu's Arteritis
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
18 Years and older
Enrollment
386 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jul 14, 2014 · Synced May 22, 2026, 3:47 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Idebenone, Placebo
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
8 Years to 17 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
2
States / cities
Los Angeles, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 10, 2023 · Synced May 22, 2026, 3:47 AM EDT
Terminated Phase 1 Interventional
View directory record Official record
Conditions
Fabry Disease
Interventions
Iohexol
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
5 Years to 25 Years · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
5
States / cities
La Jolla, California • Decatur, Georgia • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 15, 2020 · Synced May 22, 2026, 3:47 AM EDT
Terminated Phase 2Phase 3 Interventional Results available
View directory record Official record
Conditions
Prematurity, Bronchopulmonary Dysplasia, Intraventricular Hemorrhage, Periventricular Leukomalacia
Interventions
inhaled nitric oxide, oxygen
Drug
Lead sponsor
University of Chicago
Other
Eligibility
2 Hours to 72 Hours
Enrollment
273 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2023
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Oct 16, 2024 · Synced May 22, 2026, 3:47 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Neurofibromatosis Type 1, Pediatric Brain Tumor
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 18 Years
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated May 8, 2017 · Synced May 22, 2026, 3:47 AM EDT
Not listed No phase listed Observational
View directory record Official record
Conditions
Essential Tremor, Parkinson Disease, Huntington Disease, Dystonia, Primary, Spinocerebellar Ataxias, Movement Disorders
Interventions
Not listed
Lead sponsor
Rutgers, The State University of New Jersey
Other
Eligibility
18 Years to 80 Years
Enrollment
210 participants
Timeline
2019 – 2022
U.S. locations
1
States / cities
Newark, New Jersey
Source: ClinicalTrials.gov public record
Updated Sep 20, 2022 · Synced May 22, 2026, 3:47 AM EDT