Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06926621 | A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I | PHASE2 | ENROLLING BY_INVITATION | 44 | — | — | Apr 28, 2025 | Jan 30, 2029 | Jun 1, 2026 | 12 | Australia, Belgium +4 |
| NCT06185764 | A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) | PHASE1 | RECRUITING | 52 | — | — | Feb 20, 2024 | Feb 2, 2027 | Jun 8, 2026 | 26 | United States, Australia +8 |
| Arm | Type | Description |
|---|---|---|
| VX-670 | EXPERIMENTAL | Participants will receive multiple doses of VX-670. |
| Part A: Single Ascending Dose | EXPERIMENTAL | Participants will be randomized to receive a single dose of different dose levels of VX-670. |
| Part A: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive single dose of placebo matched to VX-670. |
| Part B: Single and Multiple Ascending Dose | EXPERIMENTAL | Participants will be randomized to receive single and multiple doses of different dose levels of VX-670. The dose levels will be determined based on the data from Part A. |
| Part B: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive single or multiple doses of placebo matched to VX-670. |
| Name | Type | Description |
|---|---|---|
| VX-670 | DRUG | Solution for intravenous administration. |
| Placebo | DRUG | Solution for intravenous administration. |
Key Inclusion Criteria: • Completed study drug treatment in parent study VX23-670-001 (NCT06185764) Key Exclusion Criteria: • History of any illness or any clinical condition as pre-specified in the protocol Other protocol defined Inclusion/Exclusion criteria may apply.