Status:
RECRUITING
Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy
Lead Sponsor:
Nationwide Children's Hospital
Collaborating Sponsors:
National Institute of Neurological Disorders and Stroke (NINDS)
Ann & Robert H Lurie Children's Hospital of Chicago
Conditions:
LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)
Eligibility:
All Genders
Up to 5 years
Brief Summary
The goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time. We will also learn about how this condition impacts other body systems....
Detailed Description
READY CMD LAMA2 is a Pre-Phase 1, single-arm, multicenter, prospective natural history clinical study to evaluate the natural history and potential early outcome measures in subjects with laminin α2-r...
Eligibility Criteria
Inclusion
- Signed informed consent by the subject, parent(s) or legally authorized representative (LAR) and/or assent by the subject (when applicable).
- Subject must be aged birth to less than 5.0 years of age at time of consent.
- A confirmed diagnosis of LAMA2-RD confirmed via:
- a: Two pathogenic variants in the LAMA2 gene (via a CLIA-approved laboratory) or: b. muscle biopsy with absence of merosin (laminin-211) and at least one pathogenic variant in the LAMA2 gene
- Absence of another confirmed genetic disease.
- Willingness to maintain current exercise and/or physical therapy regimen for the duration of the clinical study.
- Willingness to comply with the study protocol, including but not limited to, all study procedures and visits.
Exclusion
- Acute medical illness or hospitalization within 30 days prior to informed consent.
- Participation in a previous trial of any investigational agent for LAMA2-RD within 1 month prior to informed consent, or use of any other investigational therapy (including off-label use of Losartan) within 30 days prior to informed consent, or participation in other clinical studies, within 30 days (or 3 half-lives, whichever is longer) prior to informed consent, which in the opinion of the PI, may potentially confound results from this study.
- Other significant medical condition, which in the opinion of the site Principal Investigator may confound interpretation of the clinical course of LAMA2- RD.
Key Trial Info
Start Date :
May 12 2025
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
September 1 2028
Estimated Enrollment :
44 Patients enrolled
Trial Details
Trial ID
NCT06503367
Start Date
May 12 2025
End Date
September 1 2028
Last Update
August 12 2025
Active Locations (14)
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1
University of California, Los Angeles
Los Angeles, California, United States, 90095
2
Stanford University
Stanford, California, United States, 94305
3
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
4
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, United States, 52242