Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
IONIS-DMPKRx · 1 trial · 1 indication
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...
IONIS-DMPKRx is an investigational drug being developed for the treatment of Myotonic Dystrophy Type 1, a rare genetic disorder. It is currently in clinical development and has not been approved by regulatory authorities. The drug is being studied in adult patients with this condition.
IONIS-DMPKRx is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. The company is conducting clinical trials to evaluate the safety and tolerability of this investigational drug.
IONIS-DMPKRx is in Phase 1 clinical development. A Phase 1 study has been completed, which evaluated the safety and tolerability of multiple doses of the drug in adults with Myotonic Dystrophy Type 1. The drug remains investigational and is not yet approved.
IONIS-DMPKRx has been studied in one clinical trial, identified as NCT02312011. This was a Phase 1, randomized, double-blind, placebo-controlled study that evaluated the safety and tolerability of multiple doses in adults with Myotonic Dystrophy Type 1. The trial enrolled 48 participants in the United States and has been completed.
Yes, IONIS-DMPKRx is the same drug as ISIS-DMPKRx. The clinical trial NCT02312011 was originally titled as a study of ISIS-DMPKRx, reflecting the previous name of the compound. The drug is now referred to as IONIS-DMPKRx.