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ClinicalTrials.gov public records Last synced Oct 17, 2026, 11:38 AM EDT

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

Showing 1057–1080 of 1,710 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Completed Phase 3 Interventional Results available

An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

NCT01819727
View directory record Official record
Conditions
Phenylketonuria
Interventions
BMN 165
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 70 Years
Enrollment
261 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
31
States / cities
La Jolla, California • Oakland, California • Aurora, Colorado + 28 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2019 · Synced Oct 17, 2026, 11:38 AM 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 17, 2026, 11:38 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 17, 2026, 11:38 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells

NCT00001234
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase cDNA
Genetic
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1988 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 17, 2026, 11:38 AM EDT
Completed No phase listed Observational

Genzyme Osteopenia/Osteoporosis Study

NCT02785744
View directory record Official record
Conditions
Gaucher Disease
Interventions
Gaucher disease DNA mutation analysis
Other
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 23, 2021 · Synced Oct 17, 2026, 11:38 AM EDT
Terminated Phase 1 Interventional Results available

Study to Compare Capsule and Liquid Formulations of Enzalutamide After Single Dose Administration Under Fasting Conditions in Prostate Cancer

NCT03478904
View directory record Official record
Conditions
Prostate Cancer
Interventions
Enzalutamide Liquid, Enzalutamide Capsule
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 10, 2021 · Synced Oct 17, 2026, 11:38 AM EDT
Terminated Phase 2 Interventional Results available

Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex

NCT03363763
View directory record Official record
Conditions
Angiofibroma of Face, Tuberous Sclerosis
Interventions
Sirolimus 0.2%, Sirolimus 0.4%, Placebo ointment
Drug
Lead sponsor
Aucta Pharmaceuticals, Inc
Industry
Eligibility
2 Years to 21 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
7
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 4 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2025 · Synced Oct 17, 2026, 11:38 AM EDT
Recruiting No phase listed Observational

Advancing Integrated Therapies for Gaucher Disease

NCT07758816
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Yale University
Other
Eligibility
3 Months to 75 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Oct 17, 2026, 11:38 AM EDT
Completed Phase 3 Interventional Results available

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

NCT00158600
View directory record Official record
Conditions
Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
alglucosidase alfa, Placebo
Biological · Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
8 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
5
States / cities
Beverly Hills, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 28, 2015 · Synced Oct 17, 2026, 11:38 AM EDT
Completed Phase 2 Interventional Results available

Impact of Probiotics on Urinary Symptoms in Spinal Cord Injury SCI and SB

NCT02748317
View directory record Official record
Conditions
Lower Urinary Tract Symptoms
Interventions
Lactobacillus rhamnosus GG
Drug
Lead sponsor
Medstar Health Research Institute
Other
Eligibility
18 Years and older
Enrollment
96 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
2
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated May 12, 2020 · Synced Oct 17, 2026, 11:38 AM EDT
Recruiting Phase 1Phase 2 Interventional

AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy

NCT06063850
View directory record Official record
Conditions
Mesial Temporal Lobe Epilepsy
Interventions
AAV9-hSyn1-miGRIK2
Genetic
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
18
States / cities
Birmingham, Alabama • Phoenix, Arizona • Palo Alto, California + 15 more
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Oct 17, 2026, 11:38 AM EDT
Completed Phase 4 Interventional Results available

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

NCT01410890
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD II)
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
4
States / cities
Valhalla, New York • Cincinnati, Ohio • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 28, 2022 · Synced Oct 17, 2026, 11:38 AM EDT
Completed No phase listed Observational

Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)

NCT02998710
View directory record Official record
Conditions
Homocystinuria Due to CBS Deficiency
Interventions
Not listed
Lead sponsor
Travere Therapeutics, Inc.
Industry
Eligibility
1 Year to 65 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
7
States / cities
Aurora, Colorado • Washington D.C., District of Columbia • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 17, 2026, 11:38 AM EDT
Terminated Phase 3 Interventional

Creatine Safety, Tolerability, & Efficacy in Huntington's Disease (CREST-E)

NCT00712426
View directory record Official record
Conditions
Huntington's Disease
Interventions
Creatine Monohydrate, Placebo
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
553 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
38
States / cities
Birmingham, Alabama • Irvine, California • Sacramento, California + 35 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2016 · Synced Oct 17, 2026, 11:38 AM EDT
Recruiting No phase listed Observational

Patterns of Neurodevelopmental Disorders

NCT06213090
View directory record Official record
Conditions
Neurodevelopmental Disorders, Autism Spectrum Disorder, Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal Infection, Pediatric Acute-Onset Neuropsychiatric Syndrome, Down Syndrome, Epilepsy, Mitochondrial Encephalomyopathies, Cerebral Folate Deficiency
Interventions
Not listed
Lead sponsor
Richard Frye
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 17, 2026, 11:38 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 17, 2026, 11:38 AM 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 17, 2026, 11:38 AM EDT
Completed No phase listed Observational

Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease

NCT00244582
View directory record Official record
Conditions
Type 2 Gaucher Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Up to 5 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 17, 2026, 11:38 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.

NCT04526197
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, Celecoxib
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Aug 21, 2023 · Synced Oct 17, 2026, 11:38 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Androgen Receptor, Implications for Health and Wellbeing: Natural History Study of Individuals With Androgen Insensitivity

NCT04708431
View directory record Official record
Conditions
Androgen Insensitivity Syndrome, Metabolic Parameters in AIS, CAIS, PAIS and MAIS, Tumor Formation in AIS, CAIS, PAIS and MAIS, Sexual Function AIS, CAIS, PAIS and MAIS
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
8 Years to 99 Years
Enrollment
650 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2040
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 17, 2026, 11:38 AM 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 17, 2026, 11:38 AM 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 17, 2026, 11:38 AM EDT
Completed Not applicable Interventional

GoalKeeper: Intelligent Information Sharing for Children With Medical Complexity

NCT03620071
View directory record Official record
Conditions
Childhood Cancer, Cerebral Palsy, Chronic Lung Disease, Congenital Heart Disease, Congenital Metabolic Disorder, Gastrostomy
Interventions
GoalKeeper, Standard Care
Other
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Sep 26, 2023 · Synced Oct 17, 2026, 11:38 AM EDT
Recruiting No phase listed Observational

Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

NCT00029965
View directory record Official record
Conditions
Neurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Day to 100 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 17, 2026, 11:38 AM EDT