- 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
Search Results
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Showing 1–12
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matching trials from the live ClinicalTrials.gov search.
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- 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
- 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