Myotonic Dystrophy
Conditions
Brief summary
Safety and tolerability of VX-670 based on the assessment of adverse events (AEs), vital signs, electrocardiograms (ECGs), and clinical laboratory values
Detailed description
Change from baseline in splicing in tibialis anterior muscle biopsy at Week 24 VX-670 and PMO-0221a concentration in plasma and muscle tissues
Interventions
Sponsors
Vertex Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Age
18 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability of VX-670 based on the assessment of adverse events (AEs), vital signs, electrocardiograms (ECGs), and clinical laboratory values | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in splicing in tibialis anterior muscle biopsy at Week 24 VX-670 and PMO-0221a concentration in plasma and muscle tissues | — |
Countries
Belgium, France, Germany, Italy, Netherlands, Spain
Outcome results
None listed