Natural History Study of Children With LAMA2-related Dystrophies

Recruiting Observational Study
Merosin Deficient Congenital Muscular Dystrophy
No Placebo Group Every participant receives an active treatment — no one gets a placebo. No Study Drug Researchers observe your health over time — no experimental treatment is given.
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At a Glance
Age
2 – 15
Sex
Any
Study type
Observational
Participants needed
40 (estimated)
Sponsor
Institut de Myologie, France · Other
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About This Trial
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 e…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Signed informed consent by the Legal Authority Responsible and/or assent by the subject (starting from 6 years old) * Subject must be * Supportive clinical phenotype and diagnosis of LAMA2-RD, confirmed by: * Two pathogenic variants in the LAMA2 gene (via a diagnostic labor…
Contacts

Andreea SEFERIAN, Dr

+33 (0)1 71 73 80 50

a.seferian@institut-myologie.org

CONTACT

Erwan GASNIER, PhD

CONTACT