Natural history study of LAMA2 muscular dystrophy in children

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study aims to comprehensively characterize the disease progression and clinical features of LAMA2-related dystrophies in the pediatric population. It will establish a well-defined cohort of patients in Spain, enabling long-term follow-up and facilitating recruitment for future clinical trials. The study will utilize various assessments, including motor function scales, muscle ultrasound, and physical examinations to gather detailed data on the condition.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* All patients with compatible clinical presentation and identification of 2 pathogenic variants in LAMA2, or muscle biopsy with decreased laminin alpha2 protein and at least one pathogenic variant
* Signed informed consent by the Legal Authority Responsible and/or assent by the subject (starting from 6 years old)

Where it's running

• University Hospital Vall d'Hebron
  Barcelona, Barcelona, Spain Recruiting

Study contacts

• Study coordinator: David Gómez-Andrés
• Email: david.gomezandres@vallhebron.cat
• Phone: +34934893156

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.