Osilodrostat for the Treatment of Non-Cushing's Disease Cushing's Syndrome (LINC7)

A Retrospective Observational Study to Evaluate the Safety and Effectiveness of Osilodrostat for the Treatment of Non-Cushing's Disease Cushing's Syndrome (LINC7)

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.

Study Overview

• Status: Completed
• Conditions: Cushing's Syndrome
• Intervention / Treatment: Drug: Osilodrostat

• Study Type: Observational
• Enrollment (Actual): 103

Contacts and Locations

Study Locations

• France
  • Pessac, France, 33604
    • Hôpital Haut-Lévêque

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study population will consist of patients with non-CD CS. Patients meeting eligibility criteria will be retrospectively identified and included in the study by site investigators based on a review of medical records at site. Site policies and local regulations regarding patient consent (NOL) will be followed. All eligible patients identified at a site between April 2019 and study start date and consenting to be part of the study will be included.

Description

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 Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Long-term Effects of Osilodrostat on Mean Urinary Free Cortisol (mUFC)	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.	at weeks 18, 24, 36, 48, 60, and 72
Long Term Effects of Osilodrostat on Morning Serum Cortisol	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.	At baseline and Weeks 4, 8, 12, 18, 24, 36, 48, 60 and 72
Long-term Effects of Osilodrostat on Composite Cortisol Measure	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.	At baseline and Weeks 4, 8, 12, 18, 24, 36, 48, 60 and 72

Collaborators and Investigators

• Sponsor: RECORDATI GROUP
• Investigators: Study Director: Mario M MALDONADO, MD, RECORDATI GROUP

Study record dates

Study Major Dates

• Study Start (Actual): January 16, 2023
• Primary Completion (Actual): September 30, 2023
• Study Completion (Actual): October 30, 2023

Study Registration Dates

• First Submitted: November 16, 2022
• First Submitted That Met QC Criteria: November 30, 2022
• First Posted (Actual): December 1, 2022

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 16, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Keywords: Endogenous Cushing's Syndrome; Cushing's Syndrome; Osilodrostat
• Additional Relevant MeSH Terms: Endocrine System Diseases; Pathologic Processes; Disease; Adrenal Gland Diseases; Adrenocortical Hyperfunction; Syndrome; Cushing Syndrome
• Other Study ID Numbers: LCI699-RECAG-NI-0596

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No