Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy

Recruiting now

Conditions studied: LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)

In brief

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. Participants will undergo: * Neuromuscular assessments * Blood collections * Swallowing and breathing assessments * Questionnaires

Key facts

Study ID

NCT06503367

Run by

Nationwide Children's Hospital

People needed

44

Starts

2025-05-12

Expected to finish

2028-09-01

Last updated by the study team

2026-04-08

Who can join

Age: any, up to 5. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• University of California, Los Angeles — Los Angeles, California, United States (enrolling)
• Stanford University — Stanford, California, United States (enrolling)
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
• University of Iowa Stead Family Children's Hospital — Iowa City, Iowa, United States (enrolling)
• National Institute of Neurological Disorders and Stroke, NIH — Bethesda, Maryland, United States (enrolling)
• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
• University of Minnesota — Saint Paul, Minnesota, United States (enrolling)
• Washington University in St. Louis — St Louis, Missouri, United States (enrolling)
• University of Rochester — Rochester, New York, United States (enrolling)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
• Nationwide Children's Hospital — Columbus, Ohio, United States (enrolling)
• Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States (enrolling)
• University of Texas Southwestern — Dallas, Texas, United States (enrolling)
• University of Utah — Salt Lake City, Utah, United States

Full record on ClinicalTrials.gov

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