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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Terminated Phase 2Phase 3 Interventional Results available

Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)

NCT01433354
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
AFQ056
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
12 Years to 18 Years
Enrollment
119 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
7
States / cities
Sacramento, California • Decatur, Georgia • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2016 · Synced Jul 22, 2026, 2:18 AM EDT
Not listed Phase 1 Interventional

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

NCT03882437
View directory record Official record
Conditions
Danon Disease
Interventions
RP-A501
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2023 · Synced Jul 22, 2026, 2:18 AM EDT
Recruiting Phase 3 Interventional

Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

NCT06003387
View directory record Official record
Conditions
Hemophilia B
Interventions
CSL222 (AAV5-hFIXco-Padua)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
3
States / cities
San Diego, California • Ann Arbor, Michigan • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Jul 22, 2026, 2:18 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers

Tadalafil in Becker Muscular Dystrophy

NCT01070511
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
Tadalafil, Placebo
Drug
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years to 55 Years · Male only
Enrollment
48 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
2
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 19, 2013 · Synced Jul 22, 2026, 2:18 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Rare Diseases Clinical Research Network: Neurophysiological Correlates

NCT03077308
View directory record Official record
Conditions
Rett Syndrome, Preserved Speech Variant, Mecp2 Duplication Syndrome, Rett-related Disorders
Interventions
Auditory and Visual Event-related Potentials and EEG
Procedure
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
2 Years to 65 Years
Enrollment
185 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2021
U.S. locations
5
States / cities
Denver, Colorado • Boston, Massachusetts • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2021 · Synced Jul 22, 2026, 2:18 AM EDT
Completed No phase listed Observational

The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor

NCT01182324
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 4, 2018 · Synced Jul 22, 2026, 2:18 AM EDT
Active, not recruiting 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 – 2030
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated May 2, 2024 · Synced Jul 22, 2026, 2:18 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Study to Evaluate Exicorilant (CORT125281) in Combination With Enzalutamide in Patients With Metastatic Castration-Resistant Prostate Cancer

NCT03437941
View directory record Official record
Conditions
Metastatic Castration-Resistant Prostate Cancer
Interventions
Exicorilant, Enzalutamide, Placebo
Drug
Lead sponsor
Corcept Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
6
States / cities
Scottsdale, Arizona • Detroit, Michigan • Basking Ridge, New Jersey + 3 more
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Jul 22, 2026, 2:18 AM EDT
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 9, 2023 · Synced Jul 22, 2026, 2:18 AM EDT
Completed Phase 1 Interventional

A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN

NCT04973657
View directory record Official record
Conditions
Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)
Interventions
VK0214, Placebo
Drug · Other
Lead sponsor
Viking Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
4
States / cities
Palo Alto, California • Gainesville, Florida • Baltimore, Maryland + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2025 · Synced Jul 22, 2026, 2:18 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

NCT06138639
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SGT-003
Genetic
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
0 Years to 17 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
12
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Jul 22, 2026, 2:18 AM EDT
Enrolling by invitation No phase listed Observational

Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)

NCT05962398
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV5-hFIXco-Padua
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older · Male only
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2035
U.S. locations
15
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 11 more
Source: ClinicalTrials.gov public record
Updated Jul 17, 2025 · Synced Jul 22, 2026, 2:18 AM EDT
Terminated Phase 3 Interventional Results available

A Study of Tadalafil for Duchenne Muscular Dystrophy

NCT01865084
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Tadalafil, Placebo
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
7 Years to 14 Years · Male only
Enrollment
331 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
23
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 20 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2019 · Synced Jul 22, 2026, 2:18 AM EDT
Completed Phase 1 Interventional

A Phase 1, Open-label, Multicenter, Safety and Pharmacokinetic Study of EDI200

NCT01564225
View directory record Official record
Conditions
X-linked Hypohidrotic Ectodermal Dysplasia
Interventions
EDI200
Drug
Lead sponsor
Edimer Pharmaceuticals
Industry
Eligibility
18 Years to 40 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
2
States / cities
San Francisco, California • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 26, 2013 · Synced Jul 22, 2026, 2:18 AM EDT
Completed No phase listed Observational

Registry of Fabry Disease - A Multicenter Observational Study

NCT00055016
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
2
States / cities
Bethesda, Maryland • Cambridge, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Jul 22, 2026, 2:18 AM 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 Jun 30, 2024 · Synced Jul 22, 2026, 2:18 AM EDT
Terminated Phase 3 Interventional

An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

NCT03917719
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Edasalonexent
Drug
Lead sponsor
Catabasis Pharmaceuticals
Industry
Eligibility
4 Years to 12 Years · Male only
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
15
States / cities
Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 11 more
Source: ClinicalTrials.gov public record
Updated Nov 22, 2020 · Synced Jul 22, 2026, 2:18 AM 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 16, 2021 · Synced Jul 22, 2026, 2:18 AM EDT
Completed Phase 3 Interventional Results available

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

NCT03569891
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV5-hFIXco-Padua, Factor IX (FIX)
Genetic · Biological
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older · Male only
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
19
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 14 more
Source: ClinicalTrials.gov public record
Updated Mar 26, 2026 · Synced Jul 22, 2026, 2:18 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease

NCT03678324
View directory record Official record
Conditions
Fabry Disease
Interventions
Functional MRI and fast bound-pool fraction imaging
Other
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 25, 2022 · Synced Jul 22, 2026, 2:18 AM EDT
Completed Phase 2 Interventional

A Safety Study of NNZ-2566 in Patients With Rett Syndrome

NCT01703533
View directory record Official record
Conditions
Rett Syndrome
Interventions
NNZ-2566, Placebo
Drug
Lead sponsor
Neuren Pharmaceuticals Limited
Industry
Eligibility
16 Years to 45 Years · Female only
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
3
States / cities
Birmingham, Alabama • Saint Paul, Minnesota • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 4, 2018 · Synced Jul 22, 2026, 2:18 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease

NCT04519749
View directory record Official record
Conditions
Fabry Disease
Interventions
4D-310
Biological
Lead sponsor
4D Molecular Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
4
States / cities
La Jolla, California • Atlanta, Georgia • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2024 · Synced Jul 22, 2026, 2:18 AM EDT
Completed Phase 2 Interventional Results available

Ganaxolone Treatment in Children With Fragile X Syndrome

NCT01725152
View directory record Official record
Conditions
Fragile x Syndrome
Interventions
Ganaxolone, Placebo
Drug
Lead sponsor
Marinus Pharmaceuticals
Industry
Eligibility
6 Years to 17 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Apr 9, 2023 · Synced Jul 22, 2026, 2:18 AM EDT
Recruiting Phase 3 Interventional

Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

NCT05005975
View directory record Official record
Conditions
EPP, XLP
Interventions
MT-7117
Drug
Lead sponsor
Tanabe Pharma America, Inc.
Industry
Eligibility
12 Years to 75 Years
Enrollment
301 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
15
States / cities
Huntington Beach, California • San Francisco, California • Miami, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2025 · Synced Jul 22, 2026, 2:18 AM EDT