NCT02297945

Effects of Metyrapone in Patients With Endogenous Cushing's Syndrome

Prospective, Single Arm, Open-label, Multicenter, International Study to Assess the Effects of Metyrapone in Patients With Endogenous Cushing's Syndrome During a 12-week Treatment Period Followed by an Extension Period of 24 Weeks

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 50 (actual)
• Sponsor: HRA Pharma · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this prospective, international phase III/IV study is to assess the efficacy and safety of metyrapone in patients with endogenous Cushing's syndrome during up to 36 weeks of treatment. The ability of metyrapone (250 mg capsules) to normalize urinary free cortisol (UFC) levels will be assessed during up to 36 weeks (9 months) of treatment.

Detailed description

This study will include Cushing disease patients with persistent or recurrent disease (after pituitary surgery) or who are newly diagnosed but are unsuitable for early surgery or wish to defer surgery. It will also include patients with ectopic ACTH syndrome (either occult, after surgery failure, or inoperable or metastatic) and patients with Cushing's syndrome from adrenal causes. The ability of metyrapone to normalize urinary free cortisol levels will be assessed during up to 36 weeks (9 months) of treatment. Patients participating in this study and who are controlled or close to the target at the end of a 3-months period may continue with an optional extension period of 6 months in which the long-term efficacy and safety profiles of metyrapone will be assessed. This extension study is intended to provide new findings to consolidate existing efficacy and safety data on metyrapone in the treatment of Cushing's syndrome.

Conditions

• Cushing's Syndrome

Interventions

Type	Name	Description
DRUG	metyrapone	Single arm study

Timeline

• Start date: 2015-04-01
• Primary completion: 2019-11-01
• Completion: 2020-04-29
• First posted: 2014-11-21
• Last updated: 2020-09-25

Locations

36 sites across 8 countries: Belgium, Germany, Hungary, Italy, Poland, Romania, Spain, Turkey (Türkiye)