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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07486934 | Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 | PHASE3 | RECRUITING | 150 | — | — | Apr 1, 2026 | Jan 1, 2029 | Jun 1, 2026 | 6 | United States, Japan |
| Arm | Type | Description |
|---|---|---|
| Placebo-Controlled Period: DYNE-101 | EXPERIMENTAL | Participants will be randomized to receive DYNE-101, once every 8 weeks (Q8W) for up to 48 weeks. |
| Placebo-Controlled Period: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks. |
| Long-Term Extension Period: DYNE-101 | EXPERIMENTAL | Participants who receive DYNE-101 in Placebo-Controlled Period will continue to received DYNE-101, Q8W for up to 24 weeks. Participants who received placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks. |
| Name | Type | Description |
|---|---|---|
| zeleciment basivarsen (DYNE-101) | DRUG | Administered by IV infusion |
| Placebo | DRUG | Administered by IV infusion |
Inclusion Criteria: * Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size greater than (\>) 100. Historical results from clinical testing are acceptable. * Able to walk 10 meters and complete 5 times sit to stand independently (inserts or supports that don't go above the...