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ClinicalTrials.gov public records Last synced Oct 8, 2026, 5:58 AM EDT

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Showing 145–168 of 934 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Diseases, X-Linked Clear all
Completed No phase listed Observational

Review of Kidney Biopsies of Dent Disease Patients

NCT02022189
View directory record Official record
Conditions
Dent Disease
Interventions
Renal Biopsy
Procedure
Lead sponsor
Mayo Clinic
Other
Eligibility
Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 22, 2015 · Synced Oct 8, 2026, 5:58 AM EDT
Completed Phase 2 Interventional Results available

Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

NCT02167217
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Prednisolone
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Month to 30 Months · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
6
States / cities
Sacramento, California • Orlando, Florida • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2018 · Synced Oct 8, 2026, 5:58 AM EDT
Terminated No phase listed Observational

The Natural History of Reproductive and Overall Health in Girls and Women With a Pre-Mutation in the FMR1 Gene; Creation of a Patient Registry

NCT01187524
View directory record Official record
Conditions
Fragile X Syndrome, FMR1 Premutation, Primary Ovairan Insufficiency, Premature Ovarian Failure, Premature Menopause
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years and older · Female only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 6, 2017 · Synced Oct 8, 2026, 5:58 AM EDT
Completed No phase listed Observational

Genetic Analysis of Oculocerebrorenal Syndrome of Lowe

NCT00359515
View directory record Official record
Conditions
Lowe Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Male only
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 8, 2026, 5:58 AM EDT
Not listed No phase listed Observational

Extension Study of XLHED-Affected Male Subjects Treated With EDI200 in Protocol ECP-002

NCT01992289
View directory record Official record
Conditions
X-linked Hypohidrotic Ectodermal Dysplasia
Interventions
EDI200
Drug
Lead sponsor
Edimer Pharmaceuticals
Industry
Eligibility
6 Months to 1 Year · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2025
U.S. locations
3
States / cities
San Francisco, California • Washington D.C., District of Columbia • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Sep 5, 2017 · Synced Oct 8, 2026, 5:58 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy

NCT01207908
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
IGF-1
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
5 Years and older · Male only
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 20, 2021 · Synced Oct 8, 2026, 5:58 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Open-label Extension of the HOPE-2 Trial

NCT04428476
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Deramiocel (CAP-1002)
Biological
Lead sponsor
Capricor Inc.
Industry
Eligibility
10 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
5
States / cities
Sacramento, California • Aurora, Colorado • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 8, 2026, 5:58 AM EDT
Completed Phase 3 Interventional

Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome

NCT01282268
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
arbaclofen, placebo
Drug
Lead sponsor
Seaside Therapeutics, Inc.
Industry
Eligibility
12 Years to 50 Years
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2011
U.S. locations
24
States / cities
Phoenix, Arizona • Long Beach, California • Sacramento, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2013 · Synced Oct 8, 2026, 5:58 AM EDT
Completed Phase 4 Interventional Results available

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

NCT06019728
View directory record Official record
Conditions
Fabry's Disease
Interventions
AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine, Dexamethasone, Montelukast, Loratadine, Cetirizine, Fexofenadine
Drug
Lead sponsor
Sanofi
Industry
Eligibility
2 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
5
States / cities
Santa Monica, California • Atlanta, Georgia • Grand Rapids, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced Oct 8, 2026, 5:58 AM EDT
Completed Phase 2 Interventional Results available

Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease

NCT02489344
View directory record Official record
Conditions
Fabry Disease
Interventions
GZ/SAR402671
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older · Male only
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
3
States / cities
Atlanta, Georgia • Cincinnati, Ohio • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Mar 23, 2021 · Synced Oct 8, 2026, 5:58 AM EDT
Terminated No phase listed Observational

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

NCT04628871
View directory record Official record
Conditions
Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
Interventions
SB-318, SB-913, SB-FIX
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 8, 2026, 5:58 AM EDT
Terminated Phase 1Phase 2 Interventional

BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

NCT06611436
View directory record Official record
Conditions
Hemophilia B, Hemophilia B, Moderately Severe or Severe
Interventions
BE-101
Drug
Lead sponsor
Be Biopharma
Industry
Eligibility
18 Years and older · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
5
States / cities
Davis, California • Washington D.C., District of Columbia • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 8, 2026, 5:58 AM EDT
Completed Phase 2 Interventional Results available

Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome

NCT01254045
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
placebo, oxytocin 24IU, oxytocin 48IU
Drug
Lead sponsor
Stanford University
Other
Eligibility
13 Years to 29 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Mar 18, 2020 · Synced Oct 8, 2026, 5:58 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy

NCT06851767
View directory record Official record
Conditions
X-linked Severe Combined Immunodeficiency, X-SCID, XSCID
Interventions
Plerixafor, Filgrastim, Palifermin, Busulfan, Base-edited hematopoietic stem and progenitor cells
Genetic · Drug · Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 99 Years · Male only
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2034
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 8, 2026, 5:58 AM 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, 5:58 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)

NCT04944940
View directory record Official record
Conditions
Spinal and Bulbar Muscular Atrophy, Kennedys Disease, Motor Neuron Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 120 Years · Male only
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 8, 2026, 5:58 AM EDT
Recruiting No phase listed Observational

Prospective Research Rare Kidney Stones (ProRKS)

NCT02780297
View directory record Official record
Conditions
Hyperoxaluria, Cystinuria, Dent Disease, Lowe Syndrome, Adenine Phosphoribosyltransferase Deficiency
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
220 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
8
States / cities
Birmingham, Alabama • Jacksonville, Florida • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2026 · Synced Oct 8, 2026, 5:58 AM EDT
Recruiting No phase listed Observational

Abbott Vascular Medical Device Registry

NCT04573660
View directory record Official record
Conditions
Acute Myocardial Infarction, Restenoses, Coronary, Coronary Artery Lesions, Venous Embolism, Arterial Embolism
Interventions
Coronary and peripheral stents, Pacing catheters, Vascular plugs, Measurement and imaging (FFR and OCT), Peripheral dilatation catheters, Coronary dilatation catheters, Coronary and peripheral guidewires, Vessel closure/compression devices, Vascular access introducers
Device
Lead sponsor
Abbott Medical Devices
Industry
Eligibility
18 Years and older
Enrollment
3,784 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2031
U.S. locations
7
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 3, 2025 · Synced Oct 8, 2026, 5:58 AM 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 4, 2022 · Synced Oct 8, 2026, 5:58 AM EDT
Completed No phase listed Observational

Cerebral Blood Flow During CPB During Cardiac Surgery and the Presence of Post op Delirium

NCT02084394
View directory record Official record
Conditions
Delirium
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
50 Years and older
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 9, 2016 · Synced Oct 8, 2026, 5:58 AM EDT
Completed Phase 3 Interventional Results available

Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

NCT01027884
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne, Ambulatory Care
Interventions
Placebo, Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
10 Years to 18 Years · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
9
States / cities
Sacramento, California • Aurora, Colorado • Gainesville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2015 · Synced Oct 8, 2026, 5:58 AM EDT
Completed No phase listed Observational

Health-related Quality of Life in Rare Kidney Stone

NCT02124395
View directory record Official record
Conditions
Primary Hyperoxaluria, Cystinuria, Adenine Phosphoribosyl Transferase Deficiency, Dent Disease
Interventions
Health-related Quality of Life in Rare Kidney Stone Questionnaire
Other
Lead sponsor
NYU Langone Health
Other
Eligibility
5 Years and older
Enrollment
365 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
2
States / cities
Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 2, 2022 · Synced Oct 8, 2026, 5:58 AM EDT
Terminated Phase 2 Interventional Results available

A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy

NCT02310763
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06252616, Placebo
Biological · Drug
Lead sponsor
Pfizer
Industry
Eligibility
6 Years to 15 Years · Male only
Enrollment
121 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
30
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 14 more
Source: ClinicalTrials.gov public record
Updated Dec 7, 2020 · Synced Oct 8, 2026, 5:58 AM EDT
Recruiting No phase listed Observational

Longitudinal Study of Neurodegenerative Disorders

NCT03333200
View directory record Official record
Conditions
MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Interventions
Palliative Care, Hematopoetic Stem Cell Transplantation
Other · Biological
Lead sponsor
University of Pittsburgh
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2035
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 9, 2026 · Synced Oct 8, 2026, 5:58 AM EDT