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ClinicalTrials.gov public records Last synced May 22, 2026, 4:49 AM EDT

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

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Genetic Disease, X-Linked Clear all
Completed Phase 3 Interventional
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
BPN14770/ zatolmilast, Placebo
Drug
Lead sponsor
Tetra Discovery Partners
Industry
Eligibility
18 Years to 45 Years · Male only
Enrollment
171 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
17
States / cities
Irvine, California • Orange, California • Sacramento, California + 14 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2025 · Synced May 22, 2026, 4:49 AM EDT
Terminated No phase listed Observational Results available
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Hemophilia B standard half-life, Hemophilia B extended half-life, Hemophilia A standard half-life, Hemophilia A extended half-life
Drug
Lead sponsor
Pfizer
Industry
Eligibility
Not listed
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
3
States / cities
Gainesville, Florida • Las Vegas, Nevada • Fort Worth, Texas
Source: ClinicalTrials.gov public record
Updated May 19, 2019 · Synced May 22, 2026, 4:49 AM EDT
Terminated Phase 1 Interventional
View directory record Official record
Conditions
Fabry Disease
Interventions
Iohexol
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
5 Years to 25 Years · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
5
States / cities
La Jolla, California • Decatur, Georgia • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 15, 2020 · Synced May 22, 2026, 4:49 AM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa
Interventions
rAAV2tYF-GRK1-RPGR
Biological
Lead sponsor
Beacon Therapeutics
Industry
Eligibility
8 Years to 50 Years · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
6
States / cities
Jacksonville, Florida • Boston, Massachusetts • Cincinnati, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2024 · Synced May 22, 2026, 4:49 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
X Linked Hypohidrotic Ectodermal Dysplasia
Interventions
Not listed
Lead sponsor
Edimer Pharmaceuticals
Industry
Eligibility
4 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 26, 2013 · Synced May 22, 2026, 4:49 AM EDT
Terminated Not applicable Interventional
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 May 22, 2026, 4:49 AM EDT
Not listed No phase listed Observational
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 – 2025
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 9, 2023 · Synced May 22, 2026, 4:49 AM EDT
Terminated Phase 2Phase 3 Interventional Results available
View directory record Official record
Conditions
Rett Syndrome
Interventions
Trofinetide
Drug
Lead sponsor
ACADIA Pharmaceuticals Inc.
Industry
Eligibility
2 Years to 5 Years · Female only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
7
States / cities
Birmingham, Alabama • Aurora, Colorado • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2024 · Synced May 22, 2026, 4:49 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced May 22, 2026, 4:49 AM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
AFQ056, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 45 Years
Enrollment
175 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
12
States / cities
Phoenix, Arizona • Sacramento, California • Decatur, Georgia + 9 more
Source: ClinicalTrials.gov public record
Updated Dec 22, 2020 · Synced May 22, 2026, 4:49 AM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
Hemophilia A, Factor VIII Deficiency, Hemophilia, Hemophilia B, Factor IX Deficiency
Interventions
Peripheral Vein Blood draw
Procedure
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
1 Year to 21 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 5, 2021 · Synced May 22, 2026, 4:49 AM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Small Cell Neuroendocrine Carcinoma, Prostate Cancer, Small Cell Carcinoma
Interventions
Apalutamide, Cetrelimab
Drug · Biological
Lead sponsor
Rahul Aggarwal
Other
Eligibility
18 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 14, 2025 · Synced May 22, 2026, 4:49 AM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
6 Years to 65 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
5
States / cities
Aurora, Colorado • Washington D.C., District of Columbia • Philadelphia, Pennsylvania + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 26, 2026 · Synced May 22, 2026, 4:49 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
6 Years to 18 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010
U.S. locations
3
States / cities
Washington D.C., District of Columbia • St Louis, Missouri • Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 10, 2013 · Synced May 22, 2026, 4:49 AM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Hemophilia A, Hemophilia B, Vitamin D Deficiency
Interventions
Vitamin D and calcium
Dietary Supplement
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
2 Years to 21 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 28, 2016 · Synced May 22, 2026, 4:49 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced May 22, 2026, 4:49 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Chronic Granulomatous Disease (CGD)
Interventions
Not listed
Lead sponsor
Ensoma
Industry
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
50
States / cities
Montgomery, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 47 more
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced May 22, 2026, 4:49 AM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Congenital Bleeding Disorder, Haemophilia B
Interventions
nonacog beta pegol
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
3
States / cities
Cincinnati, Ohio • Portland, Oregon • Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Jan 19, 2017 · Synced May 22, 2026, 4:49 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Pelizaeus-Merzbacher Disease, PMD
Interventions
HuCNS-SC transplant in the lead-in phase
Biological
Lead sponsor
StemCells, Inc.
Industry
Eligibility
Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 12, 2016 · Synced May 22, 2026, 4:49 AM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
(+)- Epicatechin
Drug
Lead sponsor
Craig McDonald, MD
Other
Eligibility
8 Years to 17 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Dec 20, 2021 · Synced May 22, 2026, 4:49 AM EDT
Available No phase listed Expanded access
View directory record Official record
Conditions
Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
Interventions
CliniMACS CD34 Reagent System
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
Up to 65 Years
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 19, 2025 · Synced May 22, 2026, 4:49 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
23
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced May 22, 2026, 4:49 AM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 13 Years · Male only
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
3
States / cities
Birmingham, Alabama • Gainesville, Florida • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Feb 26, 2026 · Synced May 22, 2026, 4:49 AM EDT