Non-interventional Study on Osilodrostat in Patients With Endogenous Cushing's Syndrome
- Registration Number
- NCT05382156
- Lead Sponsor
- RECORDATI GROUP
- 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
- Detailed Description
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. This study is observational in nature and does not impose a therapy protocol, diagnostic/therapeutic interventions or a visit schedule.
Patients with endogenous Cushing's Syndrome who are treated with osilodrostat alone or in combination with other therapies will be considered eligible for study enrolment. Each patient enrolled in the study will be followed up for 3 years from study entry. Patients who discontinue prior to the end of the 3-year period will be followed-up for 3 months after discontinuation of osilodrostat and will be included in the analysis.
The total number of patients enrolled in this study will be approximately 201. Assuming a recruitment period of 3 years, the total study duration from First Patient First Visit (FPFV) to Last Patient Last Visit (LPLV) will be 6 years. The maximum duration for the individual patient is 3 years.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 206
- 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.
- Patients with exogenous CS
- Patients with Pseudo CS
- Patients participating in an interventional clinical trial with an investigational drug.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Osilodrostat Osilodrostat Osilodrostat - tablets of 1mg, 5mg, 10mg - based on patients needs - up to 3 years
- Primary Outcome Measures
Name Time Method Incidence of osilodrostat-related adverse events and serious adverse events 3 years of treatment with osilodrostat Number of participants with Adverse Events and Serious Adverse Events
- Secondary Outcome Measures
Name Time Method Change of mean urinary free cortisol (mUFC) at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years Actual and percentage change from baseline in mean urinary free cortisol (mUFC)
Change of Serum Cortisol at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years Actual and percentage change from baseline in Serum Cortisol
Change of Late Salivary Cortisol at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years Actual and percentage change from baseline in Late Salivary Cortisol
Normalization of adrenocorticotropic hormone (ACTH) at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years Proportion of patients achieving normalisation of adrenocorticotropic hormone (ACTH)
Change in Fasting Glucose at baseline before treatment start, then every 3 months through study completion up to three years Actual and percentage change from baseline in fasting glucose
Change in Dorsal fat pad at baseline before treatment start, after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Dorsal fat pad
Changes in Patient-Reported Outcome (PRO) questionnaire Euro Quality of Life (EQ) - 5 Dimensions (5D) - 5 Levels (5L) at baseline before treatment start, after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years Actual and percentage change from baseline in score of PRO questionnaire EQ-5D-5L. The minimum and maximum values for the questions are 11111 and 55555 respectively, where higher score is a worst outcome. For the visual analogue scale minimum and maximum values are 0 and 100 respectively, where higher score means a better outcome
Normalization of Serum Cortisol at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years Proportion of patients achieving normalisation of Serum Cortisol
Change in Hirsutism at baseline before treatment start, after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Hirsutism
Change in Supraclavicular fat pad at baseline before treatment start, after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Supraclavicular fat pad
Change in Central (abdominal) obesity at baseline before treatment start, after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Central (abdominal) obesity
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 Overall response rate: proportion of enrolled patients with mean urinary free cortisol (mUFC) ≤ ULN or at least 50% reduction from baseline
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 Actual and percentage change from baseline in pituitary tumour size
Change in HbA1c at baseline before treatment start, then every 3 months through study completion up to three years Actual and percentage change from baseline in HbA1c
Change in Serum Insulin at baseline before treatment start, then every 3 months through study completion up to three years Actual and percentage change from baseline in Serum Insulin
Incidence of Adverse Events (Safety and Tolerability) 3 years of treatment with osilodrostat Incidence of adverse events and laboratory abnormalities using the National Cancer Institute-Common Toxicology Criteria (NCI-CTC) grading scale (version 5.0).
Change of adrenocorticotropic hormone (ACTH) at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years Actual and percentage change from baseline in adrenocorticotropic hormone (ACTH)
Normalization of Late Salivary Cortisol at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years Proportion of patients achieving normalisation of Late Salivary Cortisol
Change in Body Mass Index (BMI) at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years Actual and percentage change from baseline in Body Mass Index (BMI)
Change in Striae at baseline before treatment start, after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Striae
Change in Ecchymoses (bruises) at baseline before treatment start, after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Ecchymoses (bruises)
Change in Fasting Lipid Profile at baseline before treatment start, then every 3 months through study completion up to three years Actual and percentage change from baseline in Fasting Lipid Profile
Change in Blood Pressure at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years Actual and percentage change from baseline in Blood Pressure
Change in Body Weight at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years Actual and percentage change from baseline in Body Weight
Change in Waist Circumference at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years Actual and percentage change from baseline in Waist Circumference
Change in Facial Rubor at baseline before treatment start, after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Facial Rubor
Change in Proximal muscle wasting (atrophy) at baseline before treatment start, after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Proximal muscle wasting (atrophy)
Changes in Patient-Reported Outcome (PRO) questionnaire Cushing Quality of Life (QoL) at baseline before treatment start, after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years Actual and percentage change from baseline in score of PRO questionnaire CushingQoL. The minimum and maximum values are 12 and 60 respectively, where higher score means a better outcome
Changes in Patient-Reported Outcome (PRO) questionnaire Patient Global Impression of Change (PGIC) after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years Actual and percentage change in score of PRO questionnaire PGIC. The minimum and maximum values of the question are 1 and 7 respectively, where higher score means a better outcome. For the visual analogue scale minimum and maximum values are 0 and 10 respectively, where higher score means a worse outcome
Changes in Patient-Reported Outcome (PRO) questionnaire Beck Depression Inventory II (BDI-II) at baseline before treatment start, after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years Actual and percentage change from baseline in score of PRO questionnaire BDI-II. The minimum and maximum values are 1 and 63 respectively, where higher score means a worse outcome
Trial Locations
- Locations (43)
Northwestern University
🇺🇸Chicago, Illinois, United States
Erasmus MC
🇳🇱Rotterdam, Netherlands
CHU de Grenoble site Nord
🇫🇷Grenoble, France
Barrow Neurological Institute
🇺🇸Phoenix, Arizona, United States
Emory University School
🇺🇸Atlanta, Georgia, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Indiana University Schl-med
🇺🇸Indianapolis, Indiana, United States
St Elizabeth Physicians
🇺🇸Covington, Kentucky, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Mayo Clinic - Rochester
🇺🇸Rochester, Minnesota, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
Memorial Sloan-Kettering Cancer Center (MSKCC) - New York
🇺🇸New York, New York, United States
Endocrinology Research Associates, Inc.
🇺🇸Columbus, Ohio, United States
NYU Grossman School of Medicine
🇺🇸New York, New York, United States
University of Pennsylvania Medical Center
🇺🇸Philadelphia, Pennsylvania, United States
Hôpital Haut-Lévêque
🇫🇷Bordeaux, France
Froedtert Hospital
🇺🇸Milwaukee, Wisconsin, United States
Hospices Civiles de Lyon
🇫🇷Bron cedex, France
Groupement Hospitalier Sud - Hôpital Bicêtre
🇫🇷Le Kremlin-Bicêtre, France
Hopital de la Conception - APHM
🇫🇷Marseille, France
Hopital Claude Huriez - CHRU Lille
🇫🇷Lille, France
Hôpital de Brabois
🇫🇷Nancy, France
CHU de Nantes-Hopital Laennec
🇫🇷Nantes, France
Hôpital Cochin
🇫🇷Paris, France
Hopital Larrey
🇫🇷Toulouse, France
Charité Universitaetsmedizin Berlin
🇩🇪Berlin, Germany
Medicover Berlin-Mitte MVZ
🇩🇪Berlin, Germany
Universitaet Bielefeld - Klinikum Bielefeld - Mitte
🇩🇪Duesseldorf, Germany
Universitaetsklinikum Frankfurt Goethe-Universitaet
🇩🇪Frankfurt, Germany
Endokrinologikum Frankfurt
🇩🇪Frankfurt, Germany
Amedes Experts
🇩🇪Hamburg, Germany
Medicover Köln
🇩🇪Köln, Germany
Azienda Ospedaliero Universitaria Ospedali Riuniti
🇮🇹Ancona, Italy
Ludwig-Maximilians University of Munich
🇩🇪Munich, Germany
Medicover Neuroendokrinologie
🇩🇪Munich, Germany
Medicover MVZ Oldenburg
🇩🇪Oldenburg, Germany
Universitaetsklinikum Wuerzburg
🇩🇪Würzburg, Germany
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
🇮🇹Milan, Italy
Azienda Ospedaliera Universitaria "Federico II"
🇮🇹Napoli, Italy
Azienda Ospedaliera Sant'Andrea-Università di Roma La Sapienza
🇮🇹Roma, Italy
Policlinico Umberto I
🇮🇹Roma, Italy
Radboud University Nijmegen
🇳🇱Nijmegen, Netherlands
Oregon Health And Science University
🇺🇸Portland, Oregon, United States