Observational study on muscular dystrophy outcomes

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study will follow up to 1000 participants with various forms of Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD) over a 24-month period. The study aims to define key phenotypes and standard clinical outcome assessments (COAs) to facilitate therapeutic development for these rare muscular dystrophies. Participants will be evaluated based on their clinical symptoms and genetic confirmations of their conditions. The findings will help in understanding the progression of these diseases and may inform future treatment strategies.

Why it matters

Who can join: the full rules

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

Where it's running

• Virginia Commonwealth University
  Richmond, Virginia, United States Recruiting

Study contacts

• Principal investigator: Nicholas Johnson, MD — Virginia Commonwealth University
• Study coordinator: Jennifer Raymond
• Email: Jennifer.Raymond@vcuhealth.org
• Phone: 804-828-6318

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.