Osilodrostat
- Generic Name
- Osilodrostat
- Brand Names
- Isturisa
- Drug Type
- Small Molecule
- Chemical Formula
- C13H10FN3
- CAS Number
- 928134-65-0
- Unique Ingredient Identifier
- 5YL4IQ1078
- Background
Osilodrostat is an inhibitor of 11β-hydroxylase (also referred to as CYP11B1), the enzyme that catalyzes the final step in the biosynthesis of endogenous cortisol. It is used to lower circulating cortisol levels in the treatment of Cushing's disease, a disorder in which cortisol levels are chronically and supraphysiologically elevated. Cushing's disease is often the result of ACTH hypersecretion secondary to a pituitary tumor, and surgical resection of the tumour is generally the treatment of choice. As an orally bioavailable drug therapy, osilodrostat provides a novel treatment option for patients in whom removal of the causative tumor is not an option or for whom previous pituitary surgery has not been curative.
Osilodrostat is manufactured by Novartis under the brand name Isturisa. It has undergone phase II clinical trials for the treatment of solid tumours, hypertension, and heart failure, but development for these indications was discontinued by Novartis in January 2013. Osilodrostat was approved for use in the EU in January 2020 for the treatment of endogenous Cushing's syndrome (i.e. Cushing's disease), and was granted FDA approval and Orphan Drug designation in the US in March 2020 for the same indication.
- Indication
Osilodrostat is indicated for the treatment of adult patients with Cushing's disease for whom pituitary surgery is not an option or has not been curative.
- Associated Conditions
- Cushing's Disease
- Associated Therapies
- -
A Block-and-Replace Therapy With Osilodrostat and Concomitant Glucocorticoid Replacement
- Conditions
- Endogenous Cushing SyndromeAdrenal InsufficiencyHypercortisolism
- Interventions
- First Posted Date
- 2024-05-28
- Last Posted Date
- 2024-08-09
- Lead Sponsor
- University of Michigan
- Target Recruit Count
- 12
- Registration Number
- NCT06430528
- Locations
- 🇺🇸
University of Michigan, Ann Arbor, Michigan, United States
Osilodrostat for the Treatment of Non-Cushing's Disease Cushing's Syndrome
- Conditions
- Cushing's Syndrome
- First Posted Date
- 2022-12-01
- Last Posted Date
- 2024-04-26
- Lead Sponsor
- RECORDATI GROUP
- Target Recruit Count
- 104
- Registration Number
- NCT05633953
- Locations
- 🇫🇷
Hôpital Haut-Lévèque, Pessac, France
Non-interventional Study on Osilodrostat in Patients With Endogenous Cushing's Syndrome
- First Posted Date
- 2022-05-19
- Last Posted Date
- 2024-07-16
- Lead Sponsor
- RECORDATI GROUP
- Target Recruit Count
- 206
- Registration Number
- NCT05382156
- Locations
- 🇺🇸
Northwestern University, Chicago, Illinois, United States
🇳🇱Erasmus MC, Rotterdam, Netherlands
🇫🇷CHU de Grenoble site Nord, Grenoble, France
Roll-over Study in Patients With Endogenous Cushing's Syndrome for LCI699
- First Posted Date
- 2018-07-30
- Last Posted Date
- 2024-12-18
- Lead Sponsor
- RECORDATI GROUP
- Target Recruit Count
- 127
- Registration Number
- NCT03606408
- Locations
- 🇺🇸
Emory University School of Medicine G2304 - C2301, Atlanta, Georgia, United States
🇺🇸Memorial Sloan Kettering Cancer Center, New York, New York, United States
🇺🇸Columbia University Medical Center New York Presbyterian Neuroendocrine Unit, New York, New York, United States
Efficacy and Safety Evaluation of Osilodrostat in Cushing's Disease
- Conditions
- Cushing's Disease
- Interventions
- First Posted Date
- 2016-03-03
- Last Posted Date
- 2021-11-01
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 73
- Registration Number
- NCT02697734
- Locations
- 🇹🇷
Novartis Investigative Site, Kocaeli, Turkey
🇺🇸Columbia University Medical Center New York Presbyterian Neuroendocrine Unit, New York, New York, United States
🇺🇸University of Colorado Endocrinology Clinical Trials Unit, Aurora, Colorado, United States
Study of Efficacy and Safety of Osilodrostat in Cushing's Syndrome
- Conditions
- Cushing's SyndromeEctopic Corticotropin SyndromeAdrenal AdenomaAIMAHPPNADAdrenal Carcinoma
- Interventions
- First Posted Date
- 2015-06-10
- Last Posted Date
- 2020-05-06
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 9
- Registration Number
- NCT02468193
- Locations
- 🇯🇵
Novartis Investigative Site, Chiba, Japan
A Phase I Study of Osilodrostat (LCI699) in Healthy Volunteers and Subjects With Impaired Renal Function
- First Posted Date
- 2015-03-26
- Last Posted Date
- 2020-12-19
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 15
- Registration Number
- NCT02399202
- Locations
- 🇩🇪
Novartis Investigative Site, Berlin, Germany
A Phase 1 Study of Osilodrostat (LCI699) in Healthy Volunteers and Subjects With Impaired Hepatic Function
- First Posted Date
- 2015-02-26
- Last Posted Date
- 2020-12-19
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 33
- Registration Number
- NCT02372084
- Locations
- 🇺🇸
University of Miami / Clinical Research Services, Inc. Boynton Beach, Miami, Florida, United States
🇺🇸Orlando Clinical Research Center, Orlando, Florida, United States
🇺🇸DaVita Clinical Research, Minneapolis, Minnesota, United States
Safety and Efficacy of LCI699 for the Treatment of Patients With Cushing's Disease
- Conditions
- Cushings Disease
- Interventions
- Drug: LCI699 matching placeboDrug: osilodrostat
- First Posted Date
- 2014-07-02
- Last Posted Date
- 2021-01-06
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 137
- Registration Number
- NCT02180217
- Locations
- 🇺🇸
University of Colorado Hospital SC - LCI699C2301, Aurora, Colorado, United States
🇺🇸Emory University School of Medicine G2304 - C2301, Atlanta, Georgia, United States
🇺🇸Northwestern University SC - LCI699C2301, Chicago, Illinois, United States
Drug Development Updates
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