Status:
RECRUITING
Spanish Natural History Study for LAMA2 Muscular Dystrophy
Lead Sponsor:
Hospital Universitari Vall d'Hebron Research Institute
Collaborating Sponsors:
ASOCIACIÓN IMPÚLSATE PARA LA CURA DE LOS NIÑOS CON DÉFICIT DE MEROSINA
Conditions:
LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)
Merosin Deficient CMD (Full or Partial)
Eligibility:
All Genders
Up to 100 years
Brief Summary
The objective of this natural history study is to comprehensively characterize the disease progression and clinical features of LAMA2-related dystrophies (LAMA2-RD) in the pediatric population. The st...
Eligibility Criteria
Inclusion
- 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)
Exclusion
Key Trial Info
Start Date :
July 27 2021
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
July 1 2030
Estimated Enrollment :
100 Patients enrolled
Trial Details
Trial ID
NCT06924125
Start Date
July 27 2021
End Date
July 1 2030
Last Update
April 11 2025
Active Locations (1)
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1
University Hospital Vall d'Hebron
Barcelona, Barcelona, Spain, 08035