NCT06503367 Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy
| NCT ID | NCT06503367 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Nationwide Children's Hospital |
| Condition | LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\) |
| Study Type | OBSERVATIONAL |
| Enrollment | 44 participants |
| Start Date | 2025-05-12 |
| Primary Completion | 2028-09 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 44 participants in total. It began in 2025-05-12 with a primary completion date of 2028-09.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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. Participants will undergo: * Neuromuscular assessments * Blood collections * Swallowing and breathing assessments * Questionnaires
Eligibility Criteria
Inclusion Criteria: * 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 Criteria: * 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.
Contact & Investigator
Anne M Connolly, MD
PRINCIPAL INVESTIGATOR
Nationwide Childrens Hospital
Frequently Asked Questions
Who can join the NCT06503367 clinical trial?
This trial is open to participants of all sexes, up to 5 Years, studying LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06503367 currently recruiting?
Yes, NCT06503367 is actively recruiting participants. Contact the research team at ReadyCMDLAMA2@nationwidechildrens.org for enrollment information.
Where is the NCT06503367 trial being conducted?
This trial is being conducted at Los Angeles, United States, Stanford, United States, Chicago, United States, Iowa City, United States and 10 additional locations.
Who is sponsoring the NCT06503367 clinical trial?
NCT06503367 is sponsored by Nationwide Children's Hospital. The principal investigator is Anne M Connolly, MD at Nationwide Childrens Hospital. The trial plans to enroll 44 participants.