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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02312011 | A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1 | PHASE1 | COMPLETED | 48 | — | — | Dec 1, 2014 | Aug 1, 2016 | Dec 6, 2022 | 8 | United States |
The number of participants with adverse events
The number of participants with adverse events
| Arm | Type | Description |
|---|---|---|
| IONIS-DMPKRx | EXPERIMENTAL | IONIS DMPKRx is administered subcutaneously over the course of 6 weeks for dose levels 1, 2, 3, 4, and 5. IONIS DMPKRx is administered subcutaneously over the course of 12 weeks for dose levels 4 or 5. |
| Placebo | PLACEBO_COMPARATOR | A placebo is administered subcutaneously over the course of 6 weeks. A placebo is administered subcutaneously over the course of 12 weeks. |
| Name | Type | Description |
|---|---|---|
| IONIS-DMPKRx | DRUG | Drug |
| Placebo | DRUG | Placebo |
Inclusion Criteria: 1. Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements 2. Males or females aged 20 to 55 years old at the time of informed consent 3. Satisfy the following: 1. Females: non...