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Showing 1–12 of 12 matching trials from the live ClinicalTrials.gov search.
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Active, not recruiting Phase 2Phase 3 Interventional Results available

A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)

NCT03199469
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
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
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
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
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
Completed No phase listed Observational

Myotubular Myopathy Event Study

NCT01840657
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
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
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
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
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
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
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