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ClinicalTrials.gov public records Last synced Sep 17, 2026, 5:02 AM EDT

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Showing 1–24 of 933 matching trials from the live ClinicalTrials.gov search.
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Terminated Phase 1Phase 2 Interventional Results available

Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita

NCT01001598
View directory record Official record
Conditions
Fanconi Anemia, Dyskeratosis Congenita
Interventions
danazol
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
3 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 18, 2019 · Synced Sep 17, 2026, 5:02 AM EDT
Withdrawn Phase 1 Interventional

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

NCT05238324
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Genetic HMI-203
Biological
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 45 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
5
States / cities
Oakland, California • New Haven, Connecticut • Hackensack, New Jersey + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2023 · Synced Sep 17, 2026, 5:02 AM EDT
Terminated Phase 2 Interventional Results available

Genomic Guided Therapy With Dasatinib or Nilutamide in Metastatic Castration-Resistant Prostate Cancer

NCT00918385
View directory record Official record
Conditions
Prostate Cancer
Interventions
Nilutamide, Dasatinib
Drug
Lead sponsor
Duke University
Other
Eligibility
18 Years and older · Male only
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
3
States / cities
Durham, North Carolina • Portland, Oregon • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Oct 28, 2014 · Synced Sep 17, 2026, 5:02 AM EDT
Recruiting Phase 2 Interventional

Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia

NCT04980638
View directory record Official record
Conditions
X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
Interventions
ER004
Biological
Lead sponsor
EspeRare Foundation
Other
Eligibility
18 Years and older · Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2032
U.S. locations
2
States / cities
Los Angeles, California • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 29, 2025 · Synced Sep 17, 2026, 5:02 AM EDT
Withdrawn No phase listed Observational

Breast Cancer BRCA1 Carriers: a Pilot Study

NCT05062174
View directory record Official record
Conditions
BRCA1 Mutation, High-grade Serous Ovarian Cancer, TNBC - Triple-Negative Breast Cancer
Interventions
Mifepristone 200 MG, Prophylactic mastectomy
Drug · Procedure
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
2
States / cities
Carmel, Indiana • Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Dec 14, 2022 · Synced Sep 17, 2026, 5:02 AM EDT
Not listed No phase listed Observational

A Study to Prospectively Assess Disease Progression in Male Children With X-ALD

NCT03278899
View directory record Official record
Conditions
X-Linked Adrenoleukodystrophy
Interventions
Not listed
Lead sponsor
NeuroVia, Inc.
Industry
Eligibility
2 Years to 13 Years · Male only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 7, 2018 · Synced Sep 17, 2026, 5:02 AM EDT
Active, not recruiting Phase 3 Interventional

A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

NCT05606614
View directory record Official record
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
6 Years to 21 Years · Female only
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
8
States / cities
Duarte, California • La Jolla, California • Oakland, California + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Sep 17, 2026, 5:02 AM EDT
Completed Phase 2 Interventional Results available

Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)

NCT01998633
View directory record Official record
Conditions
Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1, Leukocyte Adhesion Deficiency, IPEX
Interventions
Hematopoietic Stem Cell Transplant
Biological
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
4 Months to 45 Years
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
19
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Atlanta, Georgia + 16 more
Source: ClinicalTrials.gov public record
Updated Dec 7, 2022 · Synced Sep 17, 2026, 5:02 AM EDT
Completed Phase 1 Interventional

A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy

NCT02251600
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Deflazacort
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
4 Years to 16 Years · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
4
States / cities
Los Angeles, California • Chicago, Illinois • Rochester, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 17, 2017 · Synced Sep 17, 2026, 5:02 AM EDT
Completed No phase listed Observational

Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990

NCT02064933
View directory record Official record
Conditions
Wiskott-Aldrich Syndrome
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Male only
Enrollment
305 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
38
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 33 more
Source: ClinicalTrials.gov public record
Updated Aug 9, 2020 · Synced Sep 17, 2026, 5:02 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Neurologic Injuries in Adults With Urea Cycle Disorders

NCT00472732
View directory record Official record
Conditions
Brain Diseases, Metabolic, Inborn, Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
Interventions
Not listed
Lead sponsor
Andrea Gropman
Other
Eligibility
18 Years to 60 Years
Enrollment
46 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2010
U.S. locations
2
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 23, 2015 · Synced Sep 17, 2026, 5:02 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 6, 2020 · Synced Sep 17, 2026, 5:02 AM EDT
Not listed No phase listed Observational

A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders

NCT00596999
View directory record Official record
Conditions
Hematologic Malignancies
Interventions
UCB and HPDSC
Procedure
Lead sponsor
Celgene Corporation
Industry
Eligibility
Up to 55 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated Apr 9, 2012 · Synced Sep 17, 2026, 5:02 AM EDT
Recruiting Phase 3 Interventional

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

NCT07080905
View directory record Official record
Conditions
Hemophilia B
Interventions
CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
138 Months to 206 Months · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2033
U.S. locations
5
States / cities
Orange, California • Gainesville, Florida • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Sep 17, 2026, 5:02 AM EDT
Completed Phase 2 Interventional Results available

A Study of OV101 in Individuals With Fragile X Syndrome

NCT03697161
View directory record Official record
Conditions
Fragile X Syndrome (FXS)
Interventions
OV101 (gaboxadol)
Drug
Lead sponsor
Ovid Therapeutics Inc.
Other
Eligibility
13 Years to 22 Years · Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
6
States / cities
Sacramento, California • Aurora, Colorado • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2024 · Synced Sep 17, 2026, 5:02 AM EDT
Completed No phase listed Observational

Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)

NCT03349242
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
MeiraGTx UK II Ltd
Industry
Eligibility
5 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
6
States / cities
La Jolla, California • Stanford, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 19, 2024 · Synced Sep 17, 2026, 5:02 AM EDT
Recruiting No phase listed Observational

Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)

NCT07567131
View directory record Official record
Conditions
Erythropoietic Protoporphyria (EPP), X-Linked Porphyria (XLP)
Interventions
Not listed
Lead sponsor
Portal Therapeutics, Inc.
Industry
Eligibility
12 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
5
States / cities
Boston, Massachusetts • New York, New York • Wake Forest, North Carolina + 2 more
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced Sep 17, 2026, 5:02 AM EDT
Active, not recruiting No phase listed Observational

Long-Term Follow-up of Subjects Who Were Treated With ST-920

NCT05039866
View directory record Official record
Conditions
Fabry Disease, Fabry Disease, Cardiac Variant
Interventions
ST-920
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2039
U.S. locations
8
States / cities
Irvine, California • Tampa, Florida • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Sep 17, 2026, 5:02 AM EDT
Recruiting No phase listed Observational

Fabry Disease Registry & Pregnancy Sub-registry

NCT00196742
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
9,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2034
U.S. locations
92
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 72 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Sep 17, 2026, 5:02 AM EDT
Completed No phase listed Observational

Mucopolysaccharidosis Type II Natural History

NCT03529786
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Not listed
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 25, 2022 · Synced Sep 17, 2026, 5:02 AM EDT
Completed Phase 3 Interventional Results available

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

NCT02851797
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
givinostat, placebo
Drug
Lead sponsor
Italfarmaco
Industry
Eligibility
6 Years to 17 Years · Male only
Enrollment
179 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
13
States / cities
Davis, California • San Diego, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2023 · Synced Sep 17, 2026, 5:02 AM EDT
Completed Phase 1 Interventional

Effect of Cinacalcet on Parathyroid Hormone Secretion in Children and Adolescents With Hypophosphatemic Rickets

NCT00195936
View directory record Official record
Conditions
Hypophosphatemic Rickets, X-Linked Dominant
Interventions
Cinacalcet
Drug
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
5 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 18, 2021 · Synced Sep 17, 2026, 5:02 AM EDT
Completed No phase listed Observational

NSAA NON-Interventional Study Protocol

NCT06054971
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Remote Administration of NSAA
Other
Lead sponsor
Red Nucleus Enterprise Solutions, LLC
Industry
Eligibility
4 Years to 12 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Malvern, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 20, 2025 · Synced Sep 17, 2026, 5:02 AM EDT
Not listed Phase 2 Interventional

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

NCT02696044
View directory record Official record
Conditions
Rett Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Center for Rare Neurological Diseases, Norcross, GA
Other
Eligibility
2 Years and older · Female only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Norcross, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 16, 2020 · Synced Sep 17, 2026, 5:02 AM EDT