Remote Assessments and Genetic Determinants of Myotonic Dystrophy

Recruiting now

Conditions studied: Myotonic Dystrophy Type 1 (DM1), DM1

In brief

The goal of this observational study, conducted in participants' homes and requiring no travel to a study site, is to better understand disease variability in people with myotonic dystrophy type 1 (DM1) and to identify effective ways to measure symptoms. Myotonic dystrophy is one of the most variable diseases. Some people develop symptoms when they are young, others when they are much older. In the same family, some people may have mild problems, while others are strongly affected. The goal of this study is to find out more about what is causing these differences. To accomplish this, investigators will study the effects of DM1 on skeletal and smooth muscles, the heart, and the nervous system. Then, investigators will evaluate genetic differences with a blood sample. * Participants will receive a toolkit in the mail which includes all necessary equipment to participate in the study, including an iPad with video conferencing software. * Then the study team will connect with participants via videoconferencing for medical interview about DM1 symptoms and functional assessments * Participants will have their blood drawn in a lab in their community or using a home draw device, and ship it to us for research genetic analysis * Participants can chose to have their research genetic test result returned to them

Key facts

Study ID
NCT07505342
Run by
University of Rochester
People needed
1000
Starts
2022-05-10
Expected to finish
2030-01-01
Last updated by the study team
2026-04-01

Who can join

Age: 18 and older, up to 88. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Where it is running

Full record on ClinicalTrials.gov

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