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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03992430 | A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) | PHASE3 | ACTIVE NOT_RECRUITING | 160 | — | — | Jul 13, 2020 | Oct 31, 2026 | Feb 27, 2026 | 59 | United States, Colombia +24 |
| NCT02286947 | Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy | PHASE2 | COMPLETED | 24 | — | — | Nov 1, 2014 | Mar 23, 2018 | Mar 30, 2020 | 9 | United States |
An adverse event (AE) was any untoward medical occurrence in a participant that did not necessarily have a causal relationship with the study drug. A serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; Required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs were events that developed or worsened during the on-treatment period (defined as time from first dose of study drug and up to 28 days after last dose of study drug (up to 100 weeks) that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious adverse events.
| Arm | Type | Description |
|---|---|---|
| Part 1: Eteplirsen | EXPERIMENTAL | Participants will receive eteplirsen 100 mg/kg once weekly for at least 4 weeks, followed by eteplirsen 200 mg/kg once weekly for at least 4 weeks. |
| Part 2: Eteplirsen 30 mg/kg | ACTIVE_COMPARATOR | Randomized participants will receive eteplirsen 30 mg/kg once weekly for up to 144 weeks. |
| Part 2: Eteplirsen 100 mg/kg | EXPERIMENTAL | Randomized participants will receive eteplirsen 100 mg/kg once weekly for up to 144 weeks. |
| Part 2: Eteplirsen 200 mg/kg | EXPERIMENTAL | Randomized participants will receive eteplirsen 200 mg/kg once weekly for up to 144 weeks. |
| Eteplirsen 30 mg/kg | EXPERIMENTAL | Participants will receive eteplirsen 30 mg/kg/week intravenous (IV) infusions, weekly, for up to 96 weeks. |
| Name | Type | Description |
|---|---|---|
| Eteplirsen | DRUG | Solution for intravenous (IV) infusion. |
Inclusion Criteria: * Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping. * Ambulatory participant, able to perform TTRISE in 10 seconds or less at the time of screening visit. * Able to walk independently witho...