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ClinicalTrials.gov public records Last synced Oct 8, 2026, 3:20 PM EDT

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Showing 169–192 of 934 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Diseases, X-Linked Clear all
Completed No phase listed Observational

ATHN 11: Liver Transplantation Outcomes Study

NCT04055051
View directory record Official record
Conditions
Hemophilia A and B, Liver Transplantation
Interventions
Not listed
Lead sponsor
American Thrombosis and Hemostasis Network
Network
Eligibility
18 Years and older
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
15
States / cities
Los Angeles, California • San Francisco, California • Peoria, Illinois + 12 more
Source: ClinicalTrials.gov public record
Updated May 10, 2023 · Synced Oct 8, 2026, 3:20 PM EDT
Not listed No phase listed Observational

Pulmonary Disease and Exercise Tolerance in Boys With Fabry Disease

NCT01304875
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
8 Years to 18 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2010
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 27, 2013 · Synced Oct 8, 2026, 3:20 PM EDT
Terminated Phase 3 Interventional Results available

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

NCT01247207
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Male only
Enrollment
265 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2026
U.S. locations
29
States / cities
Phoenix, Arizona • Los Angeles, California • Palo Alto, California + 25 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 8, 2026, 3:20 PM EDT
Completed Phase 2 Interventional Results available

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease

NCT00304512
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years · Female only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Oct 3, 2018 · Synced Oct 8, 2026, 3:20 PM EDT
Terminated Phase 3 Interventional Results available

Open-Label Phase 3 Long-Term Safety Study of Migalastat

NCT01458119
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat hydrochloride
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
16 Years and older
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
11
States / cities
Atlanta, Georgia • Chicago, Illinois • Kansas City, Kansas + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2018 · Synced Oct 8, 2026, 3:20 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

THC Crossover Study

NCT04429568
View directory record Official record
Conditions
THC, Cannabis, Cannabis Smoking, Cannabis Use, Unspecified, Cigarette Smoking, Tobacco Use, Nicotine Dependence, Nicotine Withdrawal, Cardiovascular Risk Factor
Interventions
Smoked Cannabis, Vaped Cannabis, Tobacco Cigarette
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
21 Years and older
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2024
U.S. locations
2
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 1, 2024 · Synced Oct 8, 2026, 3:20 PM EDT
Completed Not applicable Interventional Results available

Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

NCT02162420
View directory record Official record
Conditions
Dyskeratosis Congenita, Aplastic Anemia
Interventions
Alemtuzumab, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem Cell Transplant, Anti-thymocyte globulin
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
0 Years to 70 Years
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2025
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 8, 2025 · Synced Oct 8, 2026, 3:20 PM EDT
Recruiting Phase 1 Interventional

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

NCT04626674
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
2 Years and older · Male only
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
12
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Oct 8, 2026, 3:20 PM EDT
Recruiting Phase 1 Interventional

NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)

NCT05189925
View directory record Official record
Conditions
Chronic Granulomatous Disease, Infection
Interventions
gp91 Grans
Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 75 Years · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 8, 2026, 3:20 PM EDT
Suspended Phase 2 Interventional

A Study to Assess the Tolerability of a Single Dose of STX107 in Adults With Fragile X Syndrome

NCT01325740
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
STX107
Drug
Lead sponsor
Seaside Therapeutics, Inc.
Industry
Eligibility
18 Years to 50 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
5
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 20, 2012 · Synced Oct 8, 2026, 3:20 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Analysis of the Nervous System in Patients With Fabry's Disease

NCT00001491
View directory record Official record
Conditions
Fabry's Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
325 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1995 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 8, 2026, 3:20 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

NCT00630747
View directory record Official record
Conditions
Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
Interventions
Idursulfase
Biological
Lead sponsor
Shire
Industry
Eligibility
5 Years and older · Male only
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
16
States / cities
Phoenix, Arizona • Oakland, California • Denver, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced Oct 8, 2026, 3:20 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Cancer in Inherited Bone Marrow Failure Syndromes

NCT00027274
View directory record Official record
Conditions
Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome, Inherited Bone Marrow Failure Syndrome, Aplastic Anemia
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Day to 100 Years
Enrollment
4,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2001
U.S. locations
2
States / cities
Bethesda, Maryland • Rockville, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 8, 2026, 3:20 PM EDT
Completed Phase 1 Interventional

Sodium Nitrate for Muscular Dystrophy

NCT02434627
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
Sodium Nitrate
Drug
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
15 Years to 45 Years · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jan 13, 2020 · Synced Oct 8, 2026, 3:20 PM EDT
Recruiting No phase listed Observational

Brain Development Research Program

NCT00305305
View directory record Official record
Conditions
Brain Disorders, Aicardi Syndrome
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2027
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 8, 2026, 3:20 PM EDT
Completed Not applicable Interventional Results available

Low Cost OCT for Point of Care

NCT03323307
View directory record Official record
Conditions
Optical Coherence Tomography (OCT) of the Retina
Interventions
OCT imaging
Device
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 5, 2019 · Synced Oct 8, 2026, 3:20 PM EDT
Completed No phase listed Observational

Protein Synthesis in the Brain of Patients With Fragile X Syndrome

NCT00362843
View directory record Official record
Conditions
Fragile X Syndrome, Healthy Volunteers
Interventions
[15-O]water
Drug
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
18 Years to 24 Years · Male only
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2024 · Synced Oct 8, 2026, 3:20 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

NCT03802799
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
ZYN002 - Cannabidiol Transdermal Gel
Drug
Lead sponsor
Zynerba Pharmaceuticals, Inc.
Industry
Eligibility
3 Years to 29 Years
Enrollment
476 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
19
States / cities
Phoenix, Arizona • Culver City, California • Irvine, California + 16 more
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Oct 8, 2026, 3:20 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 12, 2018 · Synced Oct 8, 2026, 3:20 PM EDT
Terminated Early Phase 1 Interventional

Gene Therapy for Chronic Granulomatous Disease

NCT00394316
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 55 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 5, 2018 · Synced Oct 8, 2026, 3:20 PM EDT
Completed Not applicable Interventional Results available

Oral Iron for Erythropoietic Protoporphyrias

NCT02979249
View directory record Official record
Conditions
Erythropoietic Protoporphyria, EPP, X-linked Protoporphyria, XLP
Interventions
Oral Iron
Drug
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
6
States / cities
Birmingham, Alabama • San Francisco, California • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2020 · Synced Oct 8, 2026, 3:20 PM EDT
Terminated Phase 2 Interventional Results available

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

NCT00759876
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
3
States / cities
Cincinnati, Ohio • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 29, 2020 · Synced Oct 8, 2026, 3:20 PM 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 8, 2026, 3:20 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients

NCT02035605
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
ALN-AT3SC, Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
51 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2017
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 19, 2020 · Synced Oct 8, 2026, 3:20 PM EDT