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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Genetic Disease, X-Linked Clear all
Completed No phase listed Observational Accepts healthy volunteers

PET Scans in Normal Volunteers and Patients With Fabry Disease

NCT00005111
View directory record Official record
Conditions
Fabry Disease, Healthy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 23, 2026, 4:20 PM EDT
Completed No phase listed Observational

Natural History of Rett Syndrome & Related Disorders

NCT02738281
View directory record Official record
Conditions
Rett Syndrome, MECP2 Duplication dIsorder, CDKL5 Disorder, FOXG1 Syndrome
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
1,044 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
14
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2021 · Synced Jul 23, 2026, 4:20 PM EDT
Terminated Phase 4 Interventional Results available

Study of the Effects of Fabrazyme Treatment on Lactation and Infants

NCT00230607
View directory record Official record
Conditions
Fabry Disease, Alpha Galactosidase A Deficiency
Interventions
agalsidase beta
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2024
U.S. locations
3
States / cities
Decatur, Georgia • Fairfax, Virginia • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 9, 2024 · Synced Jul 23, 2026, 4:20 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Magnetic Resonance and Optical Imaging of Dystrophic and Damaged Muscle

NCT02168114
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Exercising, Optical and Magnetic Resonance Imaging and Spectroscopy
Other · Procedure
Lead sponsor
University of Florida
Other
Eligibility
10 Years to 55 Years · Male only
Enrollment
17 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2018
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jan 11, 2018 · Synced Jul 23, 2026, 4:20 PM EDT
Recruiting Phase 1Phase 2 Interventional

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

NCT06270316
View directory record Official record
Conditions
Fabry Disease
Interventions
AMT-191
Drug
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
18 Years to 50 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
8
States / cities
Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2025 · Synced Jul 23, 2026, 4:20 PM EDT
Completed No phase listed Observational

Influences on Female Adolescents' Decisions Regarding Testing for Carrier Status of XSCID

NCT00006335
View directory record Official record
Conditions
Severe Combined Immunodeficiency
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
13 Years to 17 Years · Female only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Jul 23, 2026, 4:20 PM 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 20, 2020 · Synced Jul 23, 2026, 4:20 PM EDT
Completed Phase 1 Interventional

Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

NCT03508947
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
WVE-210201, Placebo
Drug
Lead sponsor
Wave Life Sciences USA, Inc.
Industry
Eligibility
5 Years to 18 Years · Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 7, 2019 · Synced Jul 23, 2026, 4:20 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy

NCT03333590
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.GALGT2
Biological
Lead sponsor
Kevin Flanigan
Other
Eligibility
4 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 12, 2025 · Synced Jul 23, 2026, 4:20 PM EDT
Completed Not applicable Interventional

Treatment of Disruptive Behaviors in Fragile X Syndrome

NCT03510156
View directory record Official record
Conditions
Fragile X Syndrome, Disruptive Behavior
Interventions
Behavior analytic treatment
Behavioral
Lead sponsor
Stanford University
Other
Eligibility
3 Years to 10 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated May 3, 2022 · Synced Jul 23, 2026, 4:20 PM EDT
Enrolling by invitation Phase 3 Interventional

A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

NCT05967351
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
Male only
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2033
U.S. locations
22
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 19 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2026 · Synced Jul 23, 2026, 4:20 PM EDT
Not yet recruiting Phase 2 Interventional

Inhibition of Aggressive Behavior in Participants With Fragile X Syndrome

NCT07328529
View directory record Official record
Conditions
Aggressive Behavior in Fragile X Syndome
Interventions
SRX246, Placebo
Drug
Lead sponsor
Azevan Pharmaceuticals
Industry
Eligibility
18 Years to 45 Years · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
Sacramento, California • Chicago, Illinois • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 8, 2026 · Synced Jul 23, 2026, 4:20 PM EDT
Terminated Phase 1 Interventional

Hemophilia B Gene Therapy With AAV8 Vector

NCT01620801
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV8-hFIX19
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 11, 2019 · Synced Jul 23, 2026, 4:20 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias

NCT02413450
View directory record Official record
Conditions
Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS), Brugada Syndrome (BrS), Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), Early Repolarization Syndrome (ERS), Arrhythmogenic Cardiomyopathy (AC, ARVD/C), Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy (DCM), Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Normal Control Subjects
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 85 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2031
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Jul 23, 2026, 4:20 PM EDT
Completed Phase 3 Interventional

A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)

NCT00018109
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
glutamine, creatine monohydrate
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
5 Years to 10 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 23, 2026, 4:20 PM EDT
Completed Phase 2 Interventional Results available

Effect of Oral Cimetidine in the Protoporphyrias

NCT05020184
View directory record Official record
Conditions
Erythropoietic Protoporphyria, X-linked Protoporphyria
Interventions
Cimetidine, Placebo
Drug
Lead sponsor
Amy K. Dickey, M.D.
Other
Eligibility
15 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
3
States / cities
Boston, Massachusetts • Winston-Salem, North Carolina • Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 28, 2026 · Synced Jul 23, 2026, 4:20 PM EDT
Recruiting Phase 3 Interventional

Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

NCT06003387
View directory record Official record
Conditions
Hemophilia B
Interventions
CSL222 (AAV5-hFIXco-Padua)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
3
States / cities
San Diego, California • Ann Arbor, Michigan • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Jul 23, 2026, 4:20 PM EDT
Recruiting Phase 1Phase 2 Interventional

Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease

NCT06325709
View directory record Official record
Conditions
Chronic Granulomatous Disease (CGD), X-Linked Chronic Granulomatous Disease
Interventions
Plerixafor, Filgrastim, Palifermin, Busulfan, Base-edited hematopoietic stem and progenitor cells, Sirolimus
Drug · Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 75 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 23, 2026, 4:20 PM EDT
Completed Phase 2 Interventional Results available

Stem Cell Transplant for Immunologic or Histiocytic Disorders

NCT00176865
View directory record Official record
Conditions
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Deficiency Syndromes, Langerhans-Cell Histiocytosis
Interventions
Stem Cell Transplant, Fludarabine, Melphalan, Anti-thymocyte globulin (ATG), Campath 1H, Cyclosporin A, Mycophenolate mofetil, Intravenous immunoglobulin (IVIG)
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 35 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 27, 2017 · Synced Jul 23, 2026, 4:20 PM EDT
Completed No phase listed Observational

Hunter Outcome Survey (HOS)

NCT03292887
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Not listed
Enrollment
1,443 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2023
U.S. locations
1
States / cities
Lexington, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 27, 2024 · Synced Jul 23, 2026, 4:20 PM EDT
Terminated Not applicable Interventional

Osteoporosis and MRI Study in Hemophilia

NCT01460147
View directory record Official record
Conditions
Bone Mineral Density, Hemophilia A, Hemophilia B
Interventions
DXA scan + MRI
Other
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
25 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 21, 2021 · Synced Jul 23, 2026, 4:20 PM EDT
Completed Phase 2 Interventional Results available

A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

NCT02760264
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vamorolone 0.25 mg/kg/day, Vamorolone 0.75 mg/kg/day, Vamorolone 2.0 mg/kg/day, Vamorolone 6.0 mg/kg/day
Drug
Lead sponsor
ReveraGen BioPharma, Inc.
Industry
Eligibility
4 Years to 6 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
6
States / cities
Davis, California • Gainesville, Florida • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 1, 2019 · Synced Jul 23, 2026, 4:20 PM EDT