Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
osilodrostat · 5 trials · 10 indications
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.
To compare the complete response rate at the end of the 8-week period of randomized withdrawal between randomized patients.A primary efficacy responder is defined as a randomized patient who has mUFC ≤ ULN at Week 34 and who was neither discontinued (study or RW treatment) nor had osilodrostat dose increase above the level at Week 26 during the RW Period of the study. mUFC: mean urinary free cortisol; ULN: Upper Limit of Normal
Percent change from baseline in the mUFC at the individual patient level
To assess the influence of renal impairment on the pharmacokinetics (PK) of LCI699i in subjects with varying degrees of renal impairment compared to subjects with normal renal function.
To assess the influence of renal impairment on the pharmacokinetics (PK) of LCI699i in subjects with varying degrees of renal impairment compared to subjects with normal renal function.
To assess the influence of renal impairment on the pharmacokinetics (PK) of LCI699i in subjects with varying degrees of renal impairment compared to subjects with normal renal function.
To assess the influence of renal impairment on the pharmacokinetics (PK) of LCI699i in subjects with varying degrees of renal impairment compared to subjects with normal renal function.
To assess the influence of hepatic impairment on the pharmacokinetics (PK) of LCI699i in subjects with varying degrees of hepatic impairment compared to subjects with normal hepatic function.
To assess the influence of hepatic impairment on the pharmacokinetics (PK) of LCI699i in subjects with varying degrees of hepatic impairment compared to subjects with normal hepatic function.
To assess the influence of hepatic impairment on the pharmacokinetics (PK) of LCI699i in subjects with varying degrees of hepatic impairment compared to subjects with normal hepatic function.
To assess the influence of hepatic impairment on the pharmacokinetics (PK) of LCI699i in subjects with varying degrees of hepatic impairment compared to subjects with normal hepatic function.
To assess the influence of hepatic impairment on the pharmacokinetics (PK) of LCI699i in subjects with varying degrees of hepatic impairment compared to subjects with normal hepatic function.
To assess the influence of hepatic impairment on the pharmacokinetics (PK) of LCI699i in subjects with varying degrees of hepatic impairment compared to subjects with normal hepatic function.
| 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) |
| osilodrostat (LCI699) | EXPERIMENTAL | Consisted of a single-arm, open-label, osilodrostat dose-titration in individual patients and then osilodrostat during a double-blind, placebo controlled RW Period. |
| LCI699 Placebo | PLACEBO_COMPARATOR | Consisted of a single-arm, open-label, osilodrostat dose-titration in individual patients and then placebo during a double-blind, placebo controlled RW Period. |
| Osilodrostat | EXPERIMENTAL | Patients in this arm took the study drug, osilodrostat. |
| osilodrostat ( LCI699) | EXPERIMENTAL | Each participant will undergo a 28 day screening /baseline period (day-28 to Day -1), followed by a 4 day treatment period ( a single 30 mg dose of LCI699 (Day 1) with 4 days of PK smple collection |
| 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 |
| LCI699 matching placebo | DRUG | Osilodrostat placebo comes in the form of film-coated tablets for oral administration, in the following strengths: 1 mg, 5 mg, 10 mg, and 20 mg. The maximum dose of osilodrostat placebo was 30 mg bid. |
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...
Osilodrostat is an investigational small molecule being developed for Cushing's Disease, Cushing's Syndrome, and related conditions such as ectopic corticotropin syndrome, adrenal adenoma, adrenal carcinoma, AIMAH, and PPNAD. It has also been studied in subjects with hepatic or renal impairment.
Osilodrostat is being developed by Novartis AG, which trades on the New York Stock Exchange under the ticker symbol NVS. The company has conducted clinical trials of the drug across multiple countries including the United States, Japan, and various European nations.
Osilodrostat is in Phase 2 clinical development for Cushing's Syndrome, based on a completed Phase 2 trial. It has also completed Phase 1 studies in healthy volunteers with hepatic or renal impairment, and a Phase 3 trial in Cushing's Disease has been completed.
Osilodrostat has completed four clinical trials: NCT02372084 (Phase 1, hepatic impairment), NCT02399202 (Phase 1, renal impairment), NCT02468193 (Phase 2, Cushing's Syndrome), and NCT02697734 (Phase 3, Cushing's Disease). All trials are completed with no active trials currently listed.
Osilodrostat is an investigational drug and is not FDA approved. It remains in clinical development, with completed trials including a Phase 3 study in Cushing's Disease. No approval status has been reported for this drug.
Osilodrostat works by inhibiting cortisol synthesis, though the specific molecular target has not been disclosed. It is being studied to reduce cortisol levels in conditions like Cushing's Disease and Cushing's Syndrome, where excess cortisol causes symptoms.