Clinical Trial

Natural History Study of Children With LAMA2-related Dystrophies

Study acronym: LAMA2
Recruiting
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Summary
The goal of this natural history study is to characterize the disease course, characteristics in paediatric population of LAMA2-RD (related dystrophies) patients. The aim of the study is to establish a well-described cohort of patients in France with LAMA2-RD for prospective follow-up and recruitment for future clinical trials. Participants will be follow up during a two years period regarding exhaustive aspects of the pathology: * Muscular function * Respiratory function * Cognitive phenotyping * Quality of life * Growth parameters * Biomarkers
Protocol Amendment History 2 amendments
This ClinicalTrials.gov record has been amended 2 times since 2024-04-04; most recent amendment 2024-12-09.
Status change: Not Yet Recruiting → Recruiting 2024-12-09
Trial Details
NCT Number NCT06354790
Lead Sponsor Institut de Myologie, France
Collaborators: Association Française contre les Myopathies (AFM), Paris
Conditions Merosin Deficient Congenital Muscular Dystrophy
Enrollment 40 participants
Start Date 2024-12-05
Primary Completion 2027-12-04 (estimated)
Study Completion 2027-12-31 (estimated)
Updated on ClinicalTrials.gov 2024-12-12