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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04535609 | An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients | PHASE2 | COMPLETED | 213 | — | — | May 21, 2021 | Oct 5, 2023 | May 8, 2024 | 41 | United States, Australia +13 |
Distance walked in meters
| Arm | Type | Description |
|---|---|---|
| Mavodelpar | EXPERIMENTAL | Once daily |
| Matched placebo | PLACEBO_COMPARATOR | Once daily |
| Name | Type | Description |
|---|---|---|
| Mavodelpar | DRUG | Once daily |
| Placebo | DRUG | Once daily |
Inclusion Criteria: 1. Subjects age 18 years or older with PMM as defined by the International Workshop: Outcome measures and clinical trial readiness in primary mitochondrial myopathies in children and adult (Mancuso et al 2017). 2. A confirmed PMM diagnosis due to known pathogenic gene mutation o...