Non-interventional Study on Osilodrostat in Patients With Endogenous Cushing's Syndrome

NCT05382156 | Active Not Recruiting FDA Drug

• 1 other identifier: LCI699-RECAG-PASS-0572
• Study Type: observational
• Target: 206
• Locations: 5 countries
• Sites: 43

Brief Summary

This is a non-interventional, multinational, multi-centre study with primary data collection, to further document the safety and efficacy of osilodrostat administered in routine clinical practice in patients treated with osilodrostat for endogenous Cushing's Syndrome

Conditions

Endogenous Cushing's Syndrome

Keywords

Cushing's syndrome; Osilodrostat

Outcome Measures

Primary Outcomes (1)

• Incidence of osilodrostat-related adverse events and serious adverse events

  Number of participants with Adverse Events and Serious Adverse Events

  3 years of treatment with osilodrostat

Secondary Outcomes (32)

• Short and long-term efficacy of osilodrostat

  at baseline before treatment start, after 1 month of treatment, then every 3 months in the first year and every 6 months thereafter through study completion up to three years

• Short and long-term efficacy of osilodrostat

  at baseline before treatment start, after 1 month of treatment, then every 3 months in the first year and every 6 months thereafter through study completion up to three years

• Short and long-term efficacy of osilodrostat

  at baseline before treatment start, after 1 month of treatment, then every 3 months in the first year and every 6 months thereafter through study completion up to three years

• Changes in pituitary tumour size

  at baseline before treatment start, after 6 months of treatment, then every 12 months through study completion up to three years

• Incidence of Adverse Events (Safety and Tolerability)

  3 years of treatment with osilodrostat

Study Arms (1)

Osilodrostat

Osilodrostat - tablets of 1mg, 5mg, 10mg - based on patients needs - up to 3 years

Drug: Osilodrostat

Interventions

Osilodrostat DRUG

oral administration of Osilodrostat tablets at different doses according to patient's need

Also known as: Isturisa

Osilodrostat

Eligibility Criteria

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

Study Population

The patient population will consist of adult male and female patients with endogenous Cushing's Syndrome. Eligible patients for the study must be treated with osilodrostat. Investigators need to ensure that patients enrolled in this study meet the study inclusion and exclusion criteria listed in the protocol.

You may qualify if:

• Written informed consent obtained prior to registration of any patient data
• Male or female patients aged 18 years or older with endogenous CS treated with osilodrostat. Treatment with osilodrostat can either be initiated at the first visit of the study or can have been initiated before screening.

You may not qualify if:

• Patients with exogenous CS
• Patients with Pseudo CS
• Patients participating in an interventional clinical trial with an investigational drug.

Sponsors & Collaborators

• RECORDATI GROUP: lead

Study Sites (43)

Barrow Neurological Institute

Phoenix, Arizona, 85013, United States

Location

Emory University School

Atlanta, Georgia, 30322, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Indiana University Schl-med

Indianapolis, Indiana, 46202, United States

Location

St Elizabeth Physicians

Covington, Kentucky, 41011, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114-2696, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

NYU Grossman School of Medicine

New York, New York, 10017, United States

Location

Memorial Sloan-Kettering Cancer Center (MSKCC) - New York

New York, New York, 10021, United States

Location

Endocrinology Research Associates, Inc.

Columbus, Ohio, 43201, United States

Location

Oregon Health And Science University

Portland, Oregon, 97239, United States

Location

University of Pennsylvania Medical Center

Philadelphia, Pennsylvania, 19104, United States

Location

Froedtert Hospital

Milwaukee, Wisconsin, 53226, United States

Location

Hôpital Haut-Lévêque

Bordeaux, 33604, France

Location

Hospices Civiles de Lyon

Bron, 69677, France

Location

CHU de Grenoble site Nord

Grenoble, 38043, France

Location

Groupement Hospitalier Sud - Hôpital Bicêtre

Le Kremlin-Bicêtre, 94275, France

Location

Hopital Claude Huriez - CHRU Lille

Lille, 59037, France

Location

Hopital de la Conception - APHM

Marseille, 13005, France

Location

Hôpital de Brabois

Nancy, 54500, France

Location

CHU de Nantes-Hopital Laennec

Nantes, 44800, France

Location

Hôpital Cochin

Paris, 75679, France

Location

Hopital Larrey

Toulouse, 31000, France

Location

Charité Universitaetsmedizin Berlin

Berlin, 10117, Germany

Location

Medicover Berlin-Mitte MVZ

Berlin, 10117, Germany

Location

Medicover Köln

Cologne, 50939, Germany

Location

Universitaet Bielefeld - Klinikum Bielefeld - Mitte

Düsseldorf, Germany

Location

Universitaetsklinikum Frankfurt Goethe-Universitaet

Frankfurt, 60590, Germany

Location

Endokrinologikum Frankfurt

Frankfurt, 60596, Germany

Location

Amedes Experts

Hamburg, 20095, Germany

Location

Medicover Neuroendokrinologie

Munich, 81667, Germany

Location

Ludwig-Maximilians University of Munich

Munich, Germany

Location

Medicover MVZ Oldenburg

Oldenburg, 26122, Germany

Location

Universitaetsklinikum Wuerzburg

Würzburg, 97080, Germany

Location

Azienda Ospedaliero Universitaria Ospedali Riuniti

Ancona, 60126, Italy

Location

Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Azienda Ospedaliera Universitaria "Federico II"

Napoli, 80131, Italy

Location

Policlinico Umberto I

Roma, 00186, Italy

Location

Azienda Ospedaliera Sant'Andrea-Università di Roma La Sapienza

Roma, 00189, Italy

Location

Radboud University Nijmegen

Nijmegen, 6500, Netherlands

Location

Erasmus MC

Rotterdam, 3015 GD, Netherlands

Location

MeSH Terms

Conditions

Cushing Syndrome

Interventions

Osilodrostat

Condition Hierarchy (Ancestors)

Adrenocortical Hyperfunction; Adrenal Gland Diseases; Endocrine System Diseases

Study Officials

• Mario Maldonado, MD

  Recordati AG - Head of Clinical Development

  STUDY CHAIR

Study Design

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

Study Record Dates

• First Submitted: May 3, 2022
• First Posted: May 19, 2022
• Study Start: June 13, 2022
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): August 1, 2027
• Last Updated: November 20, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share

Locations

DE (11); FR (10); US (15); IT (5); NL (2)