Status:
RECRUITING
Long-Term Development of Muscular Dystrophy Outcome Assessments
Lead Sponsor:
Virginia Commonwealth University
Collaborating Sponsors:
Muscular Dystrophy Association
Conditions:
LGMD1B
LGMD1C
Eligibility:
All Genders
6-50 years
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).
Detailed Description
Limb Girdle Muscular Dystrophy (LGMD) comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles. While th...
Eligibility Criteria
Inclusion
- Age between 6-50 years at enrollment
- Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with proximal weakness)
- Genetic confirmation of a LGMD, DM2, or LOPD
- FVC above 30% of predicted
Exclusion
- 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
- Participation in a clinical trial receiving an investigational product
Key Trial Info
Start Date :
October 18 2023
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
May 1 2029
Estimated Enrollment :
1000 Patients enrolled
Trial Details
Trial ID
NCT05989620
Start Date
October 18 2023
End Date
May 1 2029
Last Update
November 18 2025
Active Locations (1)
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1
Virginia Commonwealth University
Richmond, Virginia, United States, 23298