← Back to Clinical Trials
Recruiting NCT05989620
Long-Term Development of Muscular Dystrophy Outcome Assessments
Trial Parameters
Condition LGMD1B
Sponsor Virginia Commonwealth University
Study Type OBSERVATIONAL
Phase N/A
Enrollment 1,000
Sex ALL
Min Age 6 Years
Max Age 50 Years
Start Date 2023-10-18
Completion 2028-10-01
Brief Summary
This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD).
Eligibility Criteria
Inclusion Criteria: 1. Age between 6-50 years at enrollment 2. Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with proximal weakness) 3. Genetic confirmation of a LGMD, DM2, or LOPD 4. FVC above 30% of predicted Exclusion Criteria: 1. Any other illness that would interfere with the ability to undergo safe testing or would interfere with interpretation of the results in the opinion of the site investigator 2. Participation in a clinical trial receiving an investigational product