Actively Recruiting
Spanish Natural History Study for LAMA2 Muscular Dystrophy
Led by Hospital Universitari Vall d'Hebron Research Institute · Updated on 2025-04-11
100
Participants Needed
1
Research Sites
465 weeks
Total Duration
On this page
Sponsors
H
Hospital Universitari Vall d'Hebron Research Institute
Lead Sponsor
A
ASOCIACIÓN IMPÚLSATE PARA LA CURA DE LOS NIÑOS CON DÉFICIT DE MEROSINA
Collaborating Sponsor
AI-Summary
What this Trial Is About
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 study aims to establish a well-defined cohort of patients in Spain, enabling long-term follow-up and facilitating recruitment for future clinical trials.
CONDITIONS
Official Title
Spanish Natural History Study for LAMA2 Muscular Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with clinical signs compatible with LAMA2 muscular dystrophy and either two pathogenic variants in the LAMA2 gene or a muscle biopsy showing decreased laminin alpha2 protein with at least one pathogenic variant
- Signed informed consent by the legal guardian or assent by the patient if 6 years or older
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
University Hospital Vall d'Hebron
Barcelona, Barcelona, Spain, 08035
Actively Recruiting
Research Team
D
David Gómez-Andrés
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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