NCT06089018

Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 125 (estimated)
• Sponsor: Stanford University · Academic / Other
• Sex: All
• Age: 12 Years – 90 Years
• Healthy volunteers: Accepted

Summary

The goal of this observational study is to assess movement in individuals with Myotonic Dystrophy Type 1 (DM1) and Type 2 (DM2) using digital biomarker tools. The long-term aim of this study is to incorporate these outcomes into clinical trials of DM1 and DM2 therapies. Participants will complete a series of assessments that allow for researchers to measure hand myotonia and walking quality, including a Video Hand Opening Test (VHOT), grip strength, 10 meter walk/run test, 6 minute walk test, Timed Up and Go (TUG), Motor Function Measures-32 (MFM-32) test, and more. These assessments may be recorded to detect and map participants motion and walking patterns. Several patient reported outcome (PROs) questionnaires will also be recorded. Participants may also be asked to monitor exercise and sleep activity at home using an Actigraph wearable device. This study is divided into 2 parts: Part A consists of a single visit. Part B consists of a 1-year longitudinal study with 3 clinical follow-up visits.

Conditions

• Myotonic Dystrophy 1

Timeline

• Start date: 2022-09-10
• Primary completion: 2024-09-10
• Completion: 2024-09-10
• First posted: 2023-10-18
• Last updated: 2023-10-18

Locations

1 site across 1 country: United States