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ClinicalTrials.gov public records Last synced Oct 2, 2026, 9:28 PM EDT

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Showing 49–72 of 934 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Diseases, X-Linked Clear all
Terminated Phase 3 Interventional Results available

Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome

NCT03848832
View directory record Official record
Conditions
Rett Syndrome, RTT
Interventions
GWP42003-P, Placebo
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
2 Years to 18 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
14
States / cities
Birmingham, Alabama • San Diego, California • Aurora, Colorado + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2022 · Synced Oct 2, 2026, 9:28 PM EDT
Completed No phase listed Observational

Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)

NCT03319030
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
2 Years to 17 Years · Male only
Enrollment
43 participants
Timeline
2017 – 2018
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 4, 2019 · Synced Oct 2, 2026, 9:28 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 2, 2026, 9:28 PM EDT
Completed Not applicable Interventional

Theta Burst Stimulation to Improve Inhibitory Motor Physiology in Tourette Syndrome

NCT05628805
View directory record Official record
Conditions
Tourette Syndrome, Tourette Syndrome in Children, Tourette Syndrome in Adolescence, Tourette Syndrome, Modifier of
Interventions
iTBS
Device
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
10 Years to 21 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Oct 2, 2026, 9:28 PM EDT
Completed Phase 1 Interventional

Stem Cell Transplant Following Low-Intensity Chemotherapy to Treat Chronic Granulomatous Disease

NCT00001765
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Nexell Isolex with T-cell Depletion, Baxter isolex 300i
Device
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 2, 2026, 9:28 PM EDT
Recruiting No phase listed Observational

Hepatic Histopathology in Urea Cycle Disorders

NCT04908319
View directory record Official record
Conditions
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia, Carbamyl Phosphate Synthetase Deficiency, NAGS Deficiency
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 20, 2025 · Synced Oct 2, 2026, 9:28 PM EDT
Completed No phase listed Observational

Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)

NCT02057705
View directory record Official record
Conditions
Myotubular Myopathy
Interventions
Not listed
Lead sponsor
Valerion Therapeutics, LLC
Industry
Eligibility
Not listed
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 7, 2018 · Synced Oct 2, 2026, 9:28 PM EDT
Completed Phase 3 Interventional

Study Evaluating rFIX; BeneFIX in Severe Hemophilia B

NCT00037557
View directory record Official record
Conditions
Hemophilia B
Interventions
BeneFIX
Drug
Lead sponsor
Wyeth is now a wholly owned subsidiary of Pfizer
Industry
Eligibility
Up to 5 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
6
States / cities
Aurora, Colorado • Detroit, Michigan • New Brunswick, New Jersey + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2007 · Synced Oct 2, 2026, 9:28 PM EDT
Withdrawn Phase 1Phase 2 Interventional

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

NCT02704325
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.GALGT2, PLACEBO (Saline)
Biological · Other
Lead sponsor
Kevin Flanigan
Other
Eligibility
9 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 6, 2018 · Synced Oct 2, 2026, 9:28 PM EDT
Completed Phase 2 Interventional

Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)

NCT05291091
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
Sevasemten 10 mg, Sevasemten 5 mg, Sevasemten 12.5 mg, Placebo
Drug
Lead sponsor
Edgewise Therapeutics, Inc.
Industry
Eligibility
12 Years to 50 Years · Male only
Enrollment
244 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
26
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 2, 2026, 9:28 PM EDT
Recruiting No phase listed Observational

Monogenic Kidney Stone - Genetic Testing

NCT03305835
View directory record Official record
Conditions
Rare Kidney Stone Diseases
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
6,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 2, 2026, 9:28 PM EDT
Completed No phase listed Observational

Biomarker and DNA Collection in Subjects Participating in Protocol 22003

NCT00892580
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
STX209
Drug
Lead sponsor
Seaside Therapeutics, Inc.
Industry
Eligibility
6 Years to 17 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
8
States / cities
Phoenix, Arizona • Los Angeles, California • New Haven, Connecticut + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2013 · Synced Oct 2, 2026, 9:28 PM EDT
Recruiting Phase 3 Interventional

A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

NCT06056297
View directory record Official record
Conditions
Neutropenia
Interventions
Mavorixafor, Placebo
Drug
Lead sponsor
X4 Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
22
States / cities
Phoenix, Arizona • Irvine, California • La Jolla, California + 19 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2026 · Synced Oct 2, 2026, 9:28 PM EDT
Completed Phase 4 Interventional Results available

A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency

NCT01289847
View directory record Official record
Conditions
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinemia, Hyper-IgM Syndrome, Wiskott-Aldrich Syndrome
Interventions
Gammaplex
Biological
Lead sponsor
Bio Products Laboratory
Other
Eligibility
2 Years to 16 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
8
States / cities
Irvine, California • Los Angeles, California • Centennial, Colorado + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 23, 2014 · Synced Oct 2, 2026, 9:28 PM EDT
Active, not recruiting Phase 2 Interventional

A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa

NCT07174726
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa (XLRP)
Interventions
Adeno-associated virus vector expressing a human RPGR gene
Biological
Lead sponsor
Beacon Therapeutics
Industry
Eligibility
12 Years to 50 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
6
States / cities
Jacksonville, Florida • Miami, Florida • Durham, North Carolina + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2026 · Synced Oct 2, 2026, 9:28 PM EDT
Completed No phase listed Observational

Role of Interferon-gamma 1-b (IFN-γ) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous Disease

NCT03548818
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Interferon Gamma-1B
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
5 Years to 60 Years
Enrollment
9 participants
Timeline
2018 – 2020
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jan 22, 2021 · Synced Oct 2, 2026, 9:28 PM EDT
Completed Phase 1 Interventional Results available

Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease

NCT01065558
View directory record Official record
Conditions
Lesch-Nyhan Disease
Interventions
Ecopipam
Drug
Lead sponsor
Psyadon Pharma
Industry
Eligibility
6 Years to 65 Years · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
2
States / cities
San Diego, California • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 18, 2013 · Synced Oct 2, 2026, 9:28 PM EDT
Completed Phase 2 Interventional

Modified Stem Cell Transplantation Procedure for Treating Chronic Granulomatous Disease

NCT00006417
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Stem cell transplantation
Procedure
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 2, 2026, 9:28 PM EDT
Recruiting No phase listed Observational

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

NCT07127978
View directory record Official record
Conditions
Duchene Muscular Dystrophy
Interventions
Not listed
Lead sponsor
ITF Therapeutics LLC
Industry
Eligibility
6 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
9
States / cities
Little Rock, Arkansas • Washington D.C., District of Columbia • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 2, 2026 · Synced Oct 2, 2026, 9:28 PM EDT
Completed Phase 2 Interventional Results available

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

NCT02060526
View directory record Official record
Conditions
Sanfilippo Syndrome
Interventions
Recombinant human heparan N-sulfatase [rhHNS]
Drug
Lead sponsor
Shire
Industry
Eligibility
12 Months to 48 Months
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
3
States / cities
Torrance, California • Minneapolis, Minnesota • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Oct 2, 2026, 9:28 PM EDT
Terminated No phase listed Observational

Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)

NCT02095015
View directory record Official record
Conditions
Mucopolysaccharidosis (MPS)
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Up to 7 Years · Male only
Enrollment
159 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
7
States / cities
Los Angeles, California • Jacksonville, Florida • Minneapolis, Minnesota + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 17, 2021 · Synced Oct 2, 2026, 9:28 PM EDT
Completed Phase 3 Interventional Results available

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

NCT02795676
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102 (pegunigalsidase alfa), agalsidase beta
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years to 60 Years
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Orange, California + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 2, 2026, 9:28 PM 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 Oct 2, 2026, 9:28 PM EDT