Clinical Research Alert: Natural History Study in Individuals with Myotonic Dystrophy Type 2

Researchers at the University of Rochester are seeking adults with myotonic dystrophy type 2 (DM2) for a longitudinal natural history study (LEOPARD-DM2: Longitudinal Endpoint Optimization Providing an Assessment of Relevant Drugs in Myotonic Dystrophy Type 2). This study will collect health information through period assessments over 2.5 years. Findings from this study could help researchers better understand DM2 disease progression and identify meaningful outcome measures for future clinical trials.

The study

This is a long-term, observational study and does not involve a new intervention. Enrolled participants will be asked to complete an online survey every 6 months over a 2.5-year period. A survey preference form will be administered at baseline and the 30-month time point. Survey assessments may include a Demographics Survey, Myotonic Dystrophy Type 2 Health Index, PROMIS-57, and a Patient Global Impression of Severity measure.

Study criteria

To be eligible for the research study, individuals must meet the following inclusion criteria:

• Diagnosed with DM2
• Age 18 years and older
• Able to read and understand English
• Reside in any country

Interested in participating?

To learn more about the study or inquire about participation, contact the study coordinator Charlotte Engebrecht by email: charlotte_engebrecht@urmc.rochester.edu or phone: 585-200-7136.