Myotonic Dystrophy Type 1
Conditions
Brief summary
Change from baseline to Week 30 in video Hand Opening Time (vHOT).
Detailed description
1. Change from baseline to Week 30 in: • Hand grip strength. • QMT total composite score. • DM1-ActivC., 2. Change from baseline to Week 30 in: • QMT upper extremity composite score. • QMT lower extremity composite score. • QMT individual muscle group scores. • 10MWRT. • PGI-S. • EQ-5D-5L., 3. Change from baseline to Week 54 in: • vHOT. • QMT composite and individual muscle group scores. • DM1-ActivC. • 10MWRT. • EQ-5D-5L. • PGI-S. PGI-C at Week 30 and Week 54.
Interventions
Sponsors
Avidity Biosciences Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline to Week 30 in video Hand Opening Time (vHOT). | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Change from baseline to Week 30 in: • Hand grip strength. • QMT total composite score. • DM1-ActivC., 2. Change from baseline to Week 30 in: • QMT upper extremity composite score. • QMT lower extremity composite score. • QMT individual muscle group scores. • 10MWRT. • PGI-S. • EQ-5D-5L., 3. Change from baseline to Week 54 in: • vHOT. • QMT composite and individual muscle group scores. • DM1-ActivC. • 10MWRT. • EQ-5D-5L. • PGI-S. PGI-C at Week 30 and Week 54. | — |
Countries
Denmark, France, Germany, Ireland, Italy, Netherlands, Spain
Outcome results
None listed