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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02697734 | Efficacy and Safety Evaluation of Osilodrostat in Cushing's Disease | PHASE3 | COMPLETED | 73 | — | — | Oct 3, 2016 | Dec 31, 2020 | Nov 1, 2021 | 36 | United States, Belgium +12 |
A complete responder at week 12 is defined as a participant who had a mean urine free cortisol ≤ upper limit of normal (mUFC ≤ ULN) at Week 12. Participants who had a missing mUFC assessment at Week 12 were counted as non-responders for the primary endpoint.
| Arm | Type | Description |
|---|---|---|
| osilodrostat Group | EXPERIMENTAL | Participants in this arm were randomized to receive the study drug, osilodrostat followed after Week 12 by open-label osilodrostat at the starting dose (with a second dose titration) |
| osilodrostat Placebo Group | PLACEBO_COMPARATOR | Participants in this arm were randomized to receive osilodrostat placebo followed after Week 12 by open-label osilodrostat at the starting dose (with a dose titration) |
| Name | Type | Description |
|---|---|---|
| osilodrostat | DRUG | In the form of filmcoated tablets for oral administration, in the following dose strengths: 1 mg, 5 mg, 10 mg, and 20 mg. |
| osilodrostat Placebo | DRUG | Matching Placebo in the form of filmcoated tablets for oral administration |
Key inclusion criteria: * Confirmed Cushing's Disease (CD) that is persistent or recurrent as evidenced by all of the following criteria being met (i.e., a, b and c): 1. mUFC \> 1.3 x ULN (Mean of three 24-hour urine samples collected preferably on 3 consecutive days, during screening after wash...