- 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
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Showing 1–10
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- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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