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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07220603 | An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE) | PHASE2 | RECRUITING | 48 | — | — | Dec 23, 2025 | Jan 1, 2029 | Mar 30, 2026 | 3 | Canada |
| NCT06667453 | A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 | PHASE2 | RECRUITING | 24 | — | — | Dec 10, 2024 | Mar 1, 2027 | Apr 28, 2026 | 8 | Canada, New Zealand +1 |
| Arm | Type | Description |
|---|---|---|
| PGN-EDODM1 | EXPERIMENTAL | Participants will receive doses of PGN-EDODM1 once every 4 weeks (Q4W) |
| Placebo | PLACEBO_COMPARATOR | Participants randomized to the placebo arm will receive doses of saline (0.9% NaCl), once every 4 weeks (Q4W) for 12 weeks |
| Name | Type | Description |
|---|---|---|
| PGN-EDODM1 | DRUG | Administered by intravenous (IV) infusion |
| Placebo | OTHER | Administered by intravenous (IV) infusion |
Inclusion Criteria: * Participant has completed a prior study with PGN-EDODM1 Exclusion Criteria: * Abnormal laboratory tests at screening considered clinically significant by the Investigator * Use of an investigational drug (other than PGN-EDODM1), device, or product, within 30 days or 5 half-l...