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

NCT05633953 | Completed Results FDA Drug

• 1 other identifier: LCI699-RECAG-NI-0596
• Study Type: observational
• Target: 103
• Locations: 1 country
• Sites: 1

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.

Conditions

Cushing's Syndrome

Keywords

Endogenous Cushing's Syndrome; Cushing's Syndrome; Osilodrostat

Outcome Measures

Primary Outcomes (1)

• Effectiveness of Osilodrostat

  Number and proportion of patients with Mean Urinary Free Cortisol (mUFC) ≤ upper limit of normal (ULN)

  at week 12

Secondary Outcomes (3)

• Long-term Effects of Osilodrostat on Mean Urinary Free Cortisol (mUFC)

  at weeks 18, 24, 36, 48, 60, and 72

• Long Term Effects of Osilodrostat on Morning Serum Cortisol

  At baseline and Weeks 4, 8, 12, 18, 24, 36, 48, 60 and 72

• Long-term Effects of Osilodrostat on Composite Cortisol Measure

  At baseline and Weeks 4, 8, 12, 18, 24, 36, 48, 60 and 72

Interventions

Osilodrostat DRUG

oral administration

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.
• Patients treated with osilodrostat between April 2019 and study start date as part of ATU programme or commercialisation.

You may not qualify if:

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

Sponsors & Collaborators

• RECORDATI GROUP: lead

Study Sites (1)

Hôpital Haut-Lévèque

Pessac, 33604, France

Location

MeSH Terms

Conditions

Cushing Syndrome

Interventions

Osilodrostat

Condition Hierarchy (Ancestors)

Adrenocortical Hyperfunction; Adrenal Gland Diseases; Endocrine System Diseases

Results Point of Contact

• Title: Mario Maldonado
• Organization: Recordati AG

maldonado.m@recordati.com

+41798030344

Study Officials

• Mario M MALDONADO, MD

  RECORDATI GROUP

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2022
• First Posted: December 1, 2022
• Study Start: January 16, 2023
• Primary Completion: September 30, 2023
• Study Completion: October 30, 2023
• Last Updated: February 10, 2025
• Results First Posted: February 10, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share

Locations

FR (1)