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Muscular Dystrophy Trial, Recruiting
NCT01403402
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- People diagnosed with a muscle condition related to Alpha 7 or Alpha 9 Integrin proteins.
- People diagnosed with a Collagen VI related muscle condition (ranging from Ullrich to Bethlem types).
- People diagnosed with an Alpha-Dystroglycan related muscular dystrophy (including Dystroglycanopathy, Walker-Warburg Syndrome, Muscle-Eye-Brain disease, Fukuyama, FKRP, LGMD2I, LGMD2K, LGMD2M, LGMD2N, or LGMD2O).
- People diagnosed with Choline Kinase B related muscle disease.
- People diagnosed with Emery-Dreifuss Muscular Dystrophy or related forms (including LGMD1B, and those caused by LMNA, Emerin, FHL1, SYNE1, SYNE2, or TMEM43 gene changes).
- People diagnosed with LAMA2 related muscular dystrophy (also known as Merosin-deficient muscular dystrophy or MDC1A).
- People diagnosed with LMNA related muscular dystrophy (including Laminopathy and L-CMD).
- People diagnosed with RYR1 related myopathy with a muscular dystrophy-like presentation, including those with Malignant Hyperthermia or exercise-related muscle pain with or without muscle breakdown.
- People diagnosed with SEPN1 related myopathy (including Rigid Spine Muscular Dystrophy, Congenital Fiber Type Disproportion, Mallory Weiss Body Desmin, or Multi-minicore Myopathy caused by this gene).
- People diagnosed with SYNE1 (Nesprin) related muscular dystrophy.
- People diagnosed with Telethonin related muscular dystrophy (TCAP/Titin-Cap).
- People diagnosed with Congenital Muscular Dystrophy that does not fit a more specific category (including Merosin Positive forms).
- People diagnosed with Titin related LGMD or congenital muscular dystrophy (LGMD2J).
- People diagnosed with Actin Aggregation Myopathy.
- People diagnosed with Cap Disease.
- People diagnosed with Central Core Disease, including those with Malignant Hyperthermia or exercise-related muscle pain with or without muscle breakdown.
- People diagnosed with Centronuclear Myopathy, including those with Malignant Hyperthermia or exercise-related muscle pain with or without muscle breakdown.
- People diagnosed with Congenital Fiber Type Disproportion, including those with Malignant Hyperthermia or exercise-related muscle pain with or without muscle breakdown.
- People diagnosed with Core Rod Myopathy.
- People diagnosed with Hyaline Body Myopathy.
- People diagnosed with Multiminicore Myopathy.
- People diagnosed with Myotubular Myopathy.
- People diagnosed with Nemaline Myopathy.
- People diagnosed with Reducing Body Myopathy.
- People diagnosed with RYR1 related myopathy, including those with Malignant Hyperthermia or exercise-related muscle pain with or without muscle breakdown.
- People diagnosed with Spheroid Body Myopathy.
- People diagnosed with Titin related myopathy, Titin related dilated cardiomyopathy, or LGMD2J.
- People diagnosed with Tubular Aggregate Myopathy.
- People diagnosed with Zebra Body Disease Myopathy.
- People diagnosed with Congenital Myopathy that does not fit a more specific category.
- People diagnosed with Congenital Myasthenic Syndrome.
- People diagnosed with Escobar Syndrome.
- People diagnosed with Myofibrillar Myopathy.
Who may not be able to join:
- People diagnosed with Charcot Marie Tooth disease.
- People diagnosed with Duchenne or Becker Muscular Dystrophy.
- People diagnosed with Facioscapulohumeral Dystrophy (FSHD).
- People diagnosed with Kennedy's Disease.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
This study is not part of the standard drug-approval phase pathway (for example an observational, device, behavioural, or registry study), so a phase success rate does not apply.
GP referral letter
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Trial details
Status
Recruiting
Phase
Not Applicable
Sponsor
Cure CMD
Registry
ClinicalTrials.gov
Start date
1 September 2009
Est. completion
1 September 2029
Where this trial is recruiting
🇺🇸 United States
Primary endpoints
Congenital Muscle Disease Patient and Proxy Reported Outcomes
Can't join this trial?
Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.
Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.