Osilodrostat

Treatment for Cushings Syndrome

Typical Dosage: 2-30 mg twice daily

Effectiveness
55%
Safety Score
55%
Clinical Trials
9
Participants
400

Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
55
DangerousModerateSafe
Treatment Details
Dosage Range
2-30 mg twice daily
Time to Effect
2-4 weeks
Treatment Duration
lifetime
Evidence Quality
HIGH
Number Needed to Treat (NNT)
2.5(Treat 2.5 patients to see 1 additional successful outcome)
Number Needed to Harm (NNH)
3.5(Treat 3.5 patients to see 1 additional serious adverse event)
Confidence Score
90%confidence in effectiveness data
Health Economics
Annual Cost of Care
Drug Cost:$140,000
Monitoring:$2,000
Side Effect Mgmt:$1,500
Total Annual:$143,500
Cost-Effectiveness Analysis
Cost-Effectiveness Rating
POOR
ICER
$580,000/QALY
QALYs Gained
0.5
Outcome-Based Costs
Cost per Responder
$260,909
Cost per Remission
$260,909
Osilodrostat Outcomes

for Cushings Syndrome

Efficacy Outcomes
Overall Effectiveness
+55%
Response Rate
+55%
Remission Rate
+55%
Common Side Effects
Adrenal insufficiency
+35%
Nausea/Fatigue
+25%
Hypokalemia
+25%
Peripheral edema
+15%
QT prolongation
+8%

WARNING: LIMITED TRIALS AVAILABLE

You can search for trials, but you probably can't join any because the 1% Treaty hasn't passed yet. Most trials are severely limited by lack of funding and bureaucratic barriers. Help change this!

Active Clinical Trials
7 active trials recruiting for Osilodrostat in Cushings Syndrome

Non-interventional Study on Osilodrostat in Patients With Endogenous Cushing's Syndrome

NCT05382156ACTIVE NOT RECRUITING
View Study
206 participants
OBSERVATIONAL
Phoenix, United States +42 more
Started: Jun 13, 2022

A Block-and-Replace Therapy With Osilodrostat and Concomitant Glucocorticoid Replacement

NCT06430528RECRUITING
View Study
12 participants
OBSERVATIONAL
Ann Arbor, United States
Started: Jul 19, 2024

Pharmacokinetic (PK), Pharmacodynamic (PD) and Tolerability of Osilodrostat in Pediatric Patients With Cushing's Syndrome

NCT03708900RECRUITINGPHASE2
View Study
12 participants
INTERVENTIONAL
San Francisco, United States +11 more
Started: Apr 28, 2021

Osilodrostat in Patients With Hypertension Caused by Hypercortisolaemia Due to Cushing's Syndrome

NCT07247162NOT YET RECRUITINGPHASE4
View Study
63 participants
INTERVENTIONAL
Started: Aug 1, 2026

Impact of 1 mg Osilodrostat Therapy on Mild Autonomous Cortisol Secretion (MACS)

NCT07104812NOT YET RECRUITINGPHASE2
View Study
15 participants
INTERVENTIONAL
Rochester, United States
Started: Jan 1, 2026

Osilodrostat Therapy and 11C-methionine PET to Improve Corticotroph Adenoma Detection

NCT06701838NOT YET RECRUITING
View Study
15 participants
OBSERVATIONAL
Started: Dec 3, 2024

Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)

NCT07247058RECRUITINGPHASE4
View Study
10 participants
INTERVENTIONAL
Baltimore, United States
Started: Jan 20, 2026
Completed Clinical Trials
3 completed trials for Osilodrostat in Cushings Syndrome

Osilodrostat for the Treatment of Non-Cushing's Disease Cushing's Syndrome

NCT05633953COMPLETED
View Study
103 participants
OBSERVATIONAL
Pessac, France
Started: Jan 16, 2023

Roll-over Study in Patients With Endogenous Cushing's Syndrome for LCI699

NCT03606408COMPLETEDPHASE2
View Study
127 participants
INTERVENTIONAL
Aurora, United States +55 more
Started: Oct 5, 2018

Study of Efficacy and Safety of Osilodrostat in Cushing's Syndrome

NCT02468193COMPLETEDPHASE2
View Study
9 participants
INTERVENTIONAL
Fukushima, Japan +3 more
Started: Sep 24, 2015