Observational study on muscular dystrophy outcomes
Long-Term Development of Muscular Dystrophy Outcome Assessments (GRASP-01-005)
Virginia Commonwealth University · NCT05989620
This study is tracking up to 1000 people with different types of muscular dystrophy to see how their conditions progress and help develop better treatments in the future.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 1000 (estimated) |
| Ages | 6 Years to 50 Years |
| Sex | All |
| Sponsor | Virginia Commonwealth University (other) |
| Locations | 1 site (Richmond, Virginia) |
| Trial ID | NCT05989620 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals aged 6-50 years who have a confirmed diagnosis of LGMD, DM2, or LOPD and exhibit clinical symptoms consistent with these conditions.
Not a fit: Patients with other illnesses that could interfere with testing or those currently participating in other clinical trials may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved understanding and management of muscular dystrophies, potentially accelerating the development of effective therapies.
How similar studies have performed: Previous observational studies within the GRASP LGMD network have laid the groundwork for this research, indicating a potential for success in similar approaches.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Richmond, Virginia
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: LGMD1B, LGMD1C, LGMD1D, LGMD1E, LGMD1F, LGMD1G, LGMD1H, LGMD2A