Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.

Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 6, 2026, 4:20 AM EDT

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

Showing 97–120 of 934 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Diseases, X-Linked Clear all
Completed Phase 1Phase 2 Interventional

Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)

NCT00004454
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
lymphocyte gene therapy
Genetic
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1996
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 6, 2026, 4:20 AM EDT
Not listed No phase listed Observational

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

NCT00005900
View directory record Official record
Conditions
I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy, Mannosidosis, Niemann-Pick Disease, Pulmonary Complications, Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Metachromatic Leukodystrophy, Gaucher's Disease, Wolman Disease
Interventions
Not listed
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 6, 2026, 4:20 AM EDT
Enrolling by invitation Phase 3 Interventional

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

NCT05594992
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
JR-141
Drug
Lead sponsor
JCR Pharmaceuticals Co., Ltd.
Industry
Eligibility
Not listed
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
2
States / cities
Oakland, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 6, 2026, 4:20 AM EDT
Completed Phase 1 Interventional

Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy

NCT04386304
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
(+)-Epicatechin
Drug
Lead sponsor
Epirium Bio Inc.
Industry
Eligibility
16 Years to 59 Years · Male only
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
3
States / cities
Los Angeles, California • Sacramento, California • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 23, 2022 · Synced Oct 6, 2026, 4:20 AM EDT
Terminated Phase 1Phase 2 Interventional

Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B

NCT00076557
View directory record Official record
Conditions
Hemophilia B
Interventions
Adeno-Associated Viral with Human Factor IX
Genetic
Lead sponsor
Avigen
Industry
Eligibility
18 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
3
States / cities
Palo Alto, California • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 4, 2007 · Synced Oct 6, 2026, 4:20 AM EDT
Completed No phase listed Observational

Fabry Outcome Survey (FOS)

NCT03289065
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Not listed
Enrollment
4,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2021
U.S. locations
1
States / cities
Lexington, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 16, 2021 · Synced Oct 6, 2026, 4:20 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Neurologic Injuries in Adults With Urea Cycle Disorders

NCT00472732
View directory record Official record
Conditions
Brain Diseases, Metabolic, Inborn, Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
Interventions
Not listed
Lead sponsor
Andrea Gropman
Other
Eligibility
18 Years to 60 Years
Enrollment
46 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2010
U.S. locations
2
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 24, 2015 · Synced Oct 6, 2026, 4:20 AM EDT
Completed Phase 1Phase 2 Interventional

Open Label Study Investigating Safety and Efficacy of NPL2009 50 mg - 150 mg on Prepulse Inhibition Tests and Continuous Performance Tasks, Adults With Fragile X Syndrome

NCT00637221
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
NPL-2009
Drug
Lead sponsor
Neuropharm
Industry
Eligibility
18 Years to 45 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
2
States / cities
Sacramento, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Apr 27, 2012 · Synced Oct 6, 2026, 4:20 AM EDT
Terminated Phase 1Phase 2 Interventional

BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

NCT06611436
View directory record Official record
Conditions
Hemophilia B, Hemophilia B, Moderately Severe or Severe
Interventions
BE-101
Drug
Lead sponsor
Be Biopharma
Industry
Eligibility
18 Years and older · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
5
States / cities
Davis, California • Washington D.C., District of Columbia • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 6, 2026, 4:20 AM EDT
Withdrawn Phase 2 Interventional

A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy

NCT02036463
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Prednisone, Placebo
Drug
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
3 Years to 6 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 18, 2015 · Synced Oct 6, 2026, 4:20 AM EDT
Completed Phase 1 Interventional

Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy

NCT02376816
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.micro-Dystrophin
Biological
Lead sponsor
Jerry R. Mendell
Other
Eligibility
7 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 24, 2017 · Synced Oct 6, 2026, 4:20 AM EDT
Completed Phase 2 Interventional Results available

Twice Weekly Steroids and Exercise as Therapy for DMD

NCT04322357
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Prednisone, Exercise Training on standard steroid regimen, Exercise training on twice-weekly steroids
Drug · Behavioral
Lead sponsor
University of Florida
Other
Eligibility
5 Years to 9 Years · Male only
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Mar 13, 2026 · Synced Oct 6, 2026, 4:20 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

NCT05280548
View directory record Official record
Conditions
Fabry Disease
Interventions
Venglustat (GZ402671), Agalsidase alfa, Agalsidase beta (GZ419828), Migalastat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
8
States / cities
Birmingham, Alabama • Los Angeles, California • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Oct 6, 2026, 4:20 AM EDT
Active, not recruiting Phase 2 Interventional

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

NCT02171104
View directory record Official record
Conditions
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Interventions
Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
Biological · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Oct 6, 2026, 4:20 AM EDT
Completed Phase 1 Interventional

Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects

NCT01005004
View directory record Official record
Conditions
Pelizaeus-Merzbacher Disease
Interventions
HuCNS-SC cells
Biological
Lead sponsor
StemCells, Inc.
Industry
Eligibility
6 Months to 5 Years · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jan 15, 2015 · Synced Oct 6, 2026, 4:20 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Sweat Duct Imaging in Mother/Newborn Dyads

NCT01342133
View directory record Official record
Conditions
X-Linked Hypohidrotic Ectodermal Dysplasia
Interventions
Not listed
Lead sponsor
Edimer Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 28, 2012 · Synced Oct 6, 2026, 4:20 AM EDT
Terminated No phase listed Observational

Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

NCT02204904
View directory record Official record
Conditions
X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
Interventions
Allo-HSCT
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
6
States / cities
Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated May 21, 2020 · Synced Oct 6, 2026, 4:20 AM EDT
Terminated No phase listed Observational

Kidney Information Network for Disease Research and Education

NCT03321604
View directory record Official record
Conditions
ESRD, Fabry Disease, Rare Diseases, Kidney Diseases, End Stage Renal Disease, Genetic Diseases, X-Linked
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
338 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 24, 2021 · Synced Oct 6, 2026, 4:20 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)

NCT06253507
View directory record Official record
Conditions
p47, Autosomal Recessive, Chronic Granulomatous Disease
Interventions
Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47
Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 6, 2026, 4:20 AM EDT
Completed Phase 3 Interventional Results available

Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B

NCT02234310
View directory record Official record
Conditions
Hemophilia B
Interventions
rFIXFc
Biological
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
11
States / cities
Sacramento, California • Washington D.C., District of Columbia • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 25, 2022 · Synced Oct 6, 2026, 4:20 AM EDT
Recruiting Phase 2Phase 3 Interventional

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

NCT03373968
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Givinostat
Drug
Lead sponsor
Italfarmaco
Industry
Eligibility
7 Years and older · Male only
Enrollment
206 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2029
U.S. locations
10
States / cities
Sacramento, California • San Diego, California • Hartford, Connecticut + 7 more
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 6, 2026, 4:20 AM EDT
Completed Not applicable Interventional

Strength Training in Duchenne Muscular Dystrophy

NCT02421523
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Aim 2 Exercise group, Aim 1 Exercise Dosing, Aim 2 Control group
Procedure
Lead sponsor
University of Florida
Other
Eligibility
7 Years to 10 Years · Male only
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Mar 6, 2019 · Synced Oct 6, 2026, 4:20 AM EDT
Completed Phase 1 Interventional

An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort

NCT02295748
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Deflazacort
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
4 Years and older · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
4
States / cities
Los Angeles, California • Chicago, Illinois • Rochester, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 8, 2017 · Synced Oct 6, 2026, 4:20 AM EDT