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

Completed

• Conditions: Cushing's Syndrome

• Registration Number: NCT05633953
• Lead Sponsor: RECORDATI GROUP

Brief Summary

This is a multi-centre, observational, non-comparative, retrospective cohort study designed to evaluate the long-term safety and effectiveness of osilodrostat in non-CD CS patients. Patients treated with oral osilodrostat regardless of the duration of their treatment will be followed retrospectively for up to 36 months after initiating osilodrostat.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-11-16
• Study First Submitted QC: 2022-11-30
• Study First Posted: 2022-12-01
• Study Start: 2023-01-16
• Primary Completion: 2023-09-30
• Study Completion: 2023-10-30
• Results First Submitted: 2024-09-26
• Status Verified: 2025-01
• Results First Submitted QC: 2025-01-16
• Last Update Submitted: 2025-01-16
• Results First Posted: 2025-02-10
• Last Update Posted: 2025-02-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 103

Inclusion Criteria

1. Male and female patients ≥18 years old with diagnosis of CS, except for CD (i.e., an aetiology of adrenal adenoma, adrenocortical carcinoma, adrenal hyperplasia, or ectopic adrenocorticotropic hormone secretion). Patients should have a contemporaneously documented diagnosis of CS as per effective guidelines.
2. Patients treated with osilodrostat between April 2019 and study start date as part of ATU programme or commercialisation.

Exclusion Criteria

1. Patients who participated in a clinical trial anytime during the study period.
2. Patients with Pseudo-Cushing's syndrome, cyclic CS, or iatrogenic CS.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Effectiveness of Osilodrostat	at week 12	Number and proportion of patients with Mean Urinary Free Cortisol (mUFC) ≤ upper limit of normal (ULN)

Secondary Outcome Measures

Name	Time	Method
Long-term Effects of Osilodrostat on Composite Cortisol Measure	At baseline and Weeks 4, 8, 12, 18, 24, 36, 48, 60 and 72	Proportion of patients with normal response of composite cortisol (salivary cortisol, urinary cortisol and morning serum cortisol) \[≤ upper normal limit (ULN)\] by visit until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.
Long-term Effects of Osilodrostat on Mean Urinary Free Cortisol (mUFC)	at weeks 18, 24, 36, 48, 60, and 72	Proportion of patients with Mean Urinary Free Cortisol (mUFC) ≤ upper limit of normal (ULN) by visit until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.
Long Term Effects of Osilodrostat on Morning Serum Cortisol	At baseline and Weeks 4, 8, 12, 18, 24, 36, 48, 60 and 72	Proportion of patients with normal measures of morning serum cortisol \[≤ upper normal limit (ULN)\] by visit until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.

Trial Locations

Locations (1)

Hôpital Haut-Lévèque; 🇫🇷 Pessac, France