RecruitingNot Applicable

Long-Term Development of Muscular Dystrophy Outcome Assessments

United States1,000 participantsStarted 2023-10-18

Plain-language 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).

Who can participate

Age range6 Years – 50 Years

SexALL

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Inclusion criteria

✓. 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 criteria

✕. 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

What they're measuring

To explore the suitability and feasibility of the North Star Assessment for LGMD (NSAD) in the muscular dystrophies

Timeframe: Baseline to 24 months

Trial details

NCT IDNCT05989620

SponsorVirginia Commonwealth University

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2028-10-01

Contact for this trial

Jennifer Raymond

804-828-6318 Jennifer.Raymond@vcuhealth.org

View on ClinicalTrials.gov More LGMD1B trials