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ClinicalTrials.gov public records Last synced May 22, 2026, 12:49 AM EDT

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

Showing 1–12 of 12 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Myotubular Myopathy Clear all
Completed No phase listed Observational
View directory record Official record
Conditions
Myotubular Myopathy
Interventions
Genetic Testing
Other
Lead sponsor
Cure CMD
Other
Eligibility
30 Days and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Mar 6, 2018 · Synced May 22, 2026, 12:49 AM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
X-Linked Myotubular Myopathy
Interventions
ASP2957, Methylprednisolone, Prednisolone, Sirolimus
Genetic · Drug
Lead sponsor
Astellas Gene Therapies
Industry
Eligibility
Up to 36 Months · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
3
States / cities
Chicago, Illinois • Boston, Massachusetts • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced May 22, 2026, 12:49 AM EDT
Terminated Phase 1Phase 2 Interventional
View directory record Official record
Conditions
X Linked Myotubular Myopathy
Interventions
ApoTamox 10mg, Placebo
Drug
Lead sponsor
James Dowling
Other
Eligibility
6 Months and older · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
2
States / cities
Chicago, Illinois • Rockville, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced May 22, 2026, 12:49 AM EDT
Completed No phase listed Observational
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 6, 2018 · Synced May 22, 2026, 12:49 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Males With X-linked Myotubular Myopathy (XLMTM)
Interventions
Non-interventional, retrospective medical chart review
Other
Lead sponsor
Astellas Gene Therapies
Industry
Eligibility
Male only
Enrollment
161 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
6
States / cities
San Pedro, California • Aurora, Colorado • Gainesville, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2022 · Synced May 22, 2026, 12:49 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional Results available
View directory record Official record
Conditions
X-Linked Myotubular Myopathy
Interventions
Resamirigene bilparvovec
Genetic
Lead sponsor
Astellas Gene Therapies
Industry
Eligibility
Up to 5 Years · Male only
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2030
U.S. locations
3
States / cities
Los Angeles, California • Chicago, Illinois • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced May 22, 2026, 12:49 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
X-Linked Myotubular Myopathy
Interventions
No Intervention
Other
Lead sponsor
Astellas Gene Therapies
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
6
States / cities
Chicago, Illinois • Boston, Massachusetts • Cincinnati, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced May 22, 2026, 12:49 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Central Core Disease, Centronuclear Myopathy, Congenital Fiber Type Disproportion, Multiminicore Disease, Myotubular Myopathy, Nemaline Myopathy, Rigid Spine Muscular Dystrophy, Undefined Congenital Myopathy
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
4,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2050
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced May 22, 2026, 12:49 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
X-linked Myotubular Myopathy
Interventions
Not listed
Lead sponsor
Cure CMD
Other
Eligibility
Male only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Mar 6, 2018 · Synced May 22, 2026, 12:49 AM EDT
Recruiting Not applicable Interventional
View directory record Official record
Conditions
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
Interventions
Whole-body Electrical Muscle Stimulation Exercise
Device
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced May 22, 2026, 12:49 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
X-linked Myotubular Myopathy
Interventions
Not listed
Lead sponsor
Astellas Gene Therapies
Industry
Eligibility
Up to 3 Years · Male only
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
4
States / cities
Los Angeles, California • Gainesville, Florida • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2022 · Synced May 22, 2026, 12:49 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
X-linked Myotubular Myopathy
Interventions
History and physical, Tidal breathing, Maximal respiratory pressures, Peak cough flow, Pediatric Evaluation of Disability Inventory, PedsQL Multidimensional Fatigue Scale, Review of ventilation requirements
Other
Lead sponsor
University of Florida
Other
Eligibility
Up to 14 Years · Male only
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Feb 14, 2021 · Synced May 22, 2026, 12:49 AM EDT