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.
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
Erwan GASNIER, PhD
CONTACT