Myotonic Dystrophy
Conditions
Brief summary
Parts A and B: Safety and tolerability of VX-670 based on the assessment of adverse events (AEs), laboratory test results, standard 12-lead electrocardiograms (ECGs), vital signs, Columbia Suicide Severity Rating Scale (C-SSRS)
Detailed description
Parts A: PK parameter estimates of VX-670 and PMO-0221a derived from plasma concentration time data, Part B: - PK parameter estimates of VX-670 and PMO-0221a derived from plasma concentration time data - PK parameter estimates of VX-670 and PMO-0221a derived from muscle concentration data - Change from baseline in splicing index in tibialis anterior muscle biopsy
Interventions
DRUG0.9% saline solution (w/v)
Sponsors
Vertex Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Age
18 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Parts A and B: Safety and tolerability of VX-670 based on the assessment of adverse events (AEs), laboratory test results, standard 12-lead electrocardiograms (ECGs), vital signs, Columbia Suicide Severity Rating Scale (C-SSRS) | — |
Secondary
| Measure | Time frame |
|---|---|
| Parts A: PK parameter estimates of VX-670 and PMO-0221a derived from plasma concentration time data, Part B: - PK parameter estimates of VX-670 and PMO-0221a derived from plasma concentration time data - PK parameter estimates of VX-670 and PMO-0221a derived from muscle concentration data - Change from baseline in splicing index in tibialis anterior muscle biopsy | — |
Countries
Belgium, France, Germany, Italy, Netherlands, Spain
Outcome results
None listed