Muscular Dystrophy Trial, Recruiting
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:
- A parent or legal guardian must provide signed consent, and children aged 6 and older must also provide their own agreement to take part
- People who have a clinical diagnosis of LAMA2-related muscular dystrophy (LAMA2-RD), confirmed either by two faulty copies of the LAMA2 gene (identified by an approved genetic testing laboratory), or by a muscle biopsy showing absence of the merosin protein plus at least one faulty copy of the LAMA2 gene
- People who do not have any other confirmed genetic neurological condition
- People who are willing to keep their current exercise and/or physiotherapy routine the same throughout the study
- People who are willing to follow all study requirements, including attending all required visits and completing all required procedures
- People who are covered by a French social security scheme, or one recognised in France
Who may not be able to join:
- People with a developmental score below 70 and/or a behavioural condition that would require a general anaesthetic to complete an MRI scan
- People who have had an acute illness or been hospitalised within the 30 days before signing consent
- People who have previously taken part in a clinical trial for LAMA2-RD, or who have used any experimental treatment within 30 days before signing consent, or who are currently in another clinical study that the trial doctor believes could affect the results
- People with another significant medical condition or overall fragile health that, in the trial doctor's opinion, could make it difficult to interpret results
- People who are pregnant or breastfeeding
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Phone: +33 (0)1 71 73 80 50
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Change in Motor function Measurement (MFM32) score; Change in Motor Milestone Checklist; Change in Revised Upper Limb Module (RULM) score; Change in grip strength measured by dynamometer tool; Change in pinch strength measured by dynamometer tool; Change in arm flexion/extension strength measured by dynamometer tool; Change in 6 Minutes Walking Test; Change in 4 Stairs Climbing Test (4SCT); Change in 10m Walking Test; Change in Rise from Floor Test; Change in patient's Forced Vital Capacity (FVC) results; Change in patient's Peak Cough Flow (PCF) results; Change in patient's Maximum Expiratory...
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.