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ClinicalTrials.gov public records Last synced Sep 30, 2026, 2:33 AM EDT

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Showing 1–24 of 934 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Diseases, X-Linked Clear all
Completed Phase 2 Interventional Results available

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT03406780
View directory record Official record
Conditions
Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
Interventions
CAP-1002, Placebo
Biological · Drug
Lead sponsor
Capricor Inc.
Industry
Eligibility
10 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
9
States / cities
Sacramento, California • Aurora, Colorado • Orlando, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated May 28, 2025 · Synced Sep 30, 2026, 2:33 AM EDT
Completed Phase 3 Interventional

Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients

NCT00220766
View directory record Official record
Conditions
Immunologic Deficiency Syndrome, Agammaglobulinemia, Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome, Common Variable Immunodeficiency
Interventions
Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified, Dextrose, 5% in Water
Drug
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years to 75 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2004
U.S. locations
9
States / cities
Birmingham, Alabama • Denver, Colorado • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2009 · Synced Sep 30, 2026, 2:33 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Fabry Screening Study

NCT01019629
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Baylor Research Institute
Other
Eligibility
18 Years and older
Enrollment
2,724 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Feb 13, 2018 · Synced Sep 30, 2026, 2:33 AM EDT
Terminated Phase 4 Interventional Results available

Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease

NCT01147042
View directory record Official record
Conditions
IFN-Gamma Therapy, CGD Gene Mutation, CGD Response to IFNg, CGD - Chronic Granulomatous Disease, Immunodeficiency Disease
Interventions
IFN-gamma
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 20, 2017 · Synced Sep 30, 2026, 2:33 AM EDT
Active, not recruiting Phase 3 Interventional

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome

NCT03837483
View directory record Official record
Conditions
Wiskott-Aldrich Syndrome
Interventions
OTL-103
Genetic
Lead sponsor
Fondazione Telethon
Other
Eligibility
Up to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced Sep 30, 2026, 2:33 AM EDT
Completed No phase listed Observational

DDX3X Syndrome -The Seaver Autism Center for Research and Treatment is Characterizing DDX3X-related Neurodevelopmental Disorders Using Genetic, Medical, and Neuropsychological Measures.

NCT03718910
View directory record Official record
Conditions
DDX3X, Mental Retardation, X-linked 102, Autism Spectrum Disorder
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
2 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 9, 2026 · Synced Sep 30, 2026, 2:33 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

UTSW HP [13-C] Pyruvate Injection in HCM

NCT03057002
View directory record Official record
Conditions
Cardiomyopathy, Hypertrophic, Dilated Cardiomyopathy, Duchenne Muscular Dystrophy, Cardiac Sarcoidosis, Becker Muscular Dystrophy, Heart Failure With Preserved Ejection Fraction, Heart Failure With Reduced Ejection Fraction
Interventions
Hyperpolarized 13C-Pyruvate
Diagnostic Test
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 60 Years
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2023
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 26, 2025 · Synced Sep 30, 2026, 2:33 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

NCT04251026
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
tividenofusp alfa
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
Up to 18 Years · Male only
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2031
U.S. locations
4
States / cities
Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2025 · Synced Sep 30, 2026, 2:33 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

NCT07113743
View directory record Official record
Conditions
Chronic Granulomatous Disease (CGD)
Interventions
Busulfan, Tocilizumab, Eltrombopag, Sirolimus, pCCLChimGp91lentiviral vector containing the human gp91 phox (CYBB) gene
Drug · Other
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 60 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Sep 30, 2026, 2:33 AM EDT
Completed Phase 1 Interventional

Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)

NCT01482143
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
AFQ056
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
3 Years to 11 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
3
States / cities
Sacramento, California • Chicago, Illinois • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Dec 8, 2020 · Synced Sep 30, 2026, 2:33 AM EDT
Completed No phase listed Observational

Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)

NCT01822184
View directory record Official record
Conditions
Mucopolysaccharidosis (MPS), Hunter Syndrome
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
2 Years to 18 Years · Male only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 17, 2021 · Synced Sep 30, 2026, 2:33 AM EDT
Completed Phase 2 Interventional Results available

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

NCT02286947
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
9
States / cities
Los Angeles, California • Sacramento, California • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2020 · Synced Sep 30, 2026, 2:33 AM EDT
Recruiting Phase 1Phase 2 Interventional

Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists

NCT05463133
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Sirolimus, Cyclophosphamide, Alemtuzumab, Busulfan, Pheripheral blood stem cells, Emapalumab-Izsg, Tociluzumab, Total Body Irradiation
Drug · Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
4 Years to 65 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Sep 30, 2026, 2:33 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 Sep 30, 2026, 2:33 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

NCT05693142
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
RGX-202
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
1 Year and older · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
19
States / cities
Little Rock, Arkansas • Orange, California • Palo Alto, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Sep 30, 2026, 2:33 AM EDT
Completed No phase listed Observational

X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) Carrier Outlook Toward Reproduction Survey

NCT01398813
View directory record Official record
Conditions
Hypohidrotic Ectodermal Dysplasia, X-Linked Hypohidrotic Ectodermal Dysplasia
Interventions
Not listed
Lead sponsor
Edimer Pharmaceuticals
Industry
Eligibility
18 Years and older · Female only
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Portland, Maine
Source: ClinicalTrials.gov public record
Updated Jul 9, 2013 · Synced Sep 30, 2026, 2:33 AM EDT
Completed No phase listed Observational

Mucopolysaccharidosis Type II Natural History

NCT03529786
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Not listed
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 26, 2022 · Synced Sep 30, 2026, 2:33 AM EDT
Completed Phase 1 Interventional

A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy

NCT02595489
View directory record Official record
Conditions
X-linked Adrenoleukodystrophy
Interventions
vitamin D3
Dietary Supplement
Lead sponsor
Stanford University
Other
Eligibility
18 Months to 25 Years · Male only
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 9, 2022 · Synced Sep 30, 2026, 2:33 AM EDT
Completed Phase 3 Interventional Results available

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT01826487
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
Interventions
Ataluren, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
7 Years to 16 Years · Male only
Enrollment
230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
21
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 17 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2020 · Synced Sep 30, 2026, 2:33 AM EDT
Withdrawn No phase listed Observational

Evaluation of Phenotypic Variability in Fabry Disease

NCT03145779
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Year and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 3, 2020 · Synced Sep 30, 2026, 2:33 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

The Role of Family Functioning in Promoting Adaptation in Siblings of Individuals With Duchenne Muscular Dystrophy (DMD)

NCT01386515
View directory record Official record
Conditions
Genetic Disease, Communication
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
13 Years and older
Enrollment
33 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 12, 2019 · Synced Sep 30, 2026, 2:33 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Role Of Phosphorus And FGF 23 In Patients With Dent Disease

NCT02016235
View directory record Official record
Conditions
Dent Disease
Interventions
Phosphorus Supplement, Observation
Drug · Other
Lead sponsor
Mayo Clinic
Other
Eligibility
Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 23, 2020 · Synced Sep 30, 2026, 2:33 AM EDT
Withdrawn Phase 4 Interventional

A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency.

NCT00312767
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
1
States / cities
North Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Sep 30, 2026, 2:33 AM EDT
Completed Phase 2 Interventional Results available

A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

NCT00214500
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 55 Years · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
5
States / cities
Los Angeles, California • Decatur, Georgia • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2018 · Synced Sep 30, 2026, 2:33 AM EDT