Long-Term Development of Muscular Dystrophy Outcome Assessments (GRASP-01-005)
Status: Recruiting
Location: See location...
Study Type: Observational
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
Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 50
View:
• 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
Locations
United States
Virginia
Virginia Commonwealth University
RECRUITING
Richmond
Contact Information
Primary
Jennifer Raymond
Jennifer.Raymond@vcuhealth.org
804-828-6318
Backup
Ruby Langeslay
Ruby.Langeslay@vcuhealth.org
Time Frame
Start Date: 2023-10-18
Estimated Completion Date: 2029-05-01
Participants
Target number of participants: 1000
Treatments
LGMD, DM2, LOPD
Related Therapeutic Areas
Sponsors
Leads: Virginia Commonwealth University
Collaborators: Muscular Dystrophy Association