Long-Term Development of Muscular Dystrophy Outcome Assessments
This is an observational study looking at how Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late-onset Pompe disease (LOPD) progress over 24 months. Researchers want to see if a specific assessment tool, the North Star Assessment for LGMD (NSAD), is a good way to measure changes in these conditions. This will help in developing new treatments faster. You might be able to join if you are between 6 and 50 years old, have weakness consistent with proximal weakness, and have a confirmed genetic diagnosis of LGMD, DM2, or LOPD, with good lung function (FVC above 30%). There are no specific interventions or drugs being tested in this study.
- Study design
- This is an observational study with a planned enrollment of up to 1000 participants. It is not testing a specific drug or treatment.
- What's involved
- This is a 24-month study. The primary goal is to explore the suitability and feasibility of the North Star Assessment for LGMD (NSAD) at baseline and 24 months.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for 24 months, with measurements taken at baseline and 24 months.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Long-Term Development of Muscular Dystrophy Outcome Assessments
At a glance
Conditions
NCT05989620
Where you'd take part
This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Virginia Commonwealth University
Richmond, Virginiastudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Study leadership
- Nicholas Johnson, MD · PRINCIPAL_INVESTIGATOR · Virginia Commonwealth University
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- To explore the suitability and feasibility of the North Star Assessment for LGMD (NSAD) in the muscular dystrophiesBaseline to 24 months
The NSAD is a functional scale specifically designed to measure motor performance in individuals with LGMD. It consists of 29 items that are considered clinically relevant items from the adapted North Star Ambulatory Assessment and the Motor Function Measure 20 with a maximum score of 54.