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

Endogenous Cushing's Syndrome Clinical Trial

— LINC6  

Official title:

A Non-interventional Study to Assess the Long-term Safety and Efficacy of Osilodrostat in Patients With Endogenous Cushing's Syndrome

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05382156
• Other study ID #: LCI699-RECAG-PASS-0572
• Status: Recruiting
• Start date: June 13, 2022
• Est. completion date: February 2029

Study information

• Verified date: August 2023
• Source: RECORDATI GROUP
• Contact: Julia Stermenska
• Phone: +41 79 440 58 97
• Email: stermenska.j[@]recordati.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

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 at least 100. 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 information / eligibility

• Status: Recruiting
• Enrollment: 100
• Est. completion date: February 2029
• Est. primary completion date: June 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• 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.

Exclusion Criteria:

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

Related Conditions & MeSH terms

• Cushing Syndrome
• Endogenous Cushing's Syndrome
• Syndrome

Intervention

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

Locations

Country	Name	City	State
France	Hôpital Haut-Lévêque	Bordeaux
France	Hospices Civiles de Lyon	Bron cedex
France	CHU de Grenoble site Nord	Grenoble
France	Groupement Hospitalier Sud - Hôpital Bicêtre	Le Kremlin-Bicêtre
France	Hopital Claude Huriez - CHRU Lille	Lille
France	Hopital de la Conception - APHM	Marseille
France	Hôpital de Brabois	Nancy
France	CHU de Nantes-Hopital Laennec	Nantes
France	Hôpital Cochin	Paris
France	Hopital Larrey	Toulouse
Germany	Charité Universitaetsmedizin Berlin	Berlin
Germany	Medicover Berlin-Mitte MVZ	Berlin
Germany	Endokrinologikum Frankfurt	Frankfurt
Germany	Universitaetsklinikum Frankfurt Goethe-Universitaet	Frankfurt
Germany	Amedes Experts	Hamburg
Germany	Medicover Köln	Köln
Germany	Medicover Neuroendokrinologie	Munich
Germany	Medicover MVZ Oldenburg	Oldenburg
Germany	Universitaetsklinikum Wuerzburg	Würzburg
United States	University of Michigan	Ann Arbor	Michigan
United States	Emory University School	Atlanta	Georgia
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Endocrinology Research Associates, Inc.	Columbus	Ohio
United States	Indiana University Schl-med	Indianapolis	Indiana
United States	Memorial Sloan-Kettering Cancer Center (MSKCC) - New York	New York	New York
United States	NYU Grossman School of Medicine	New York	New York
United States	University of Pennsylvania Medical Center	Philadelphia	Pennsylvania
United States	Oregon Health And Science University	Portland	Oregon
United States	Mayo Clinic - Rochester	Rochester	Minnesota
United States	Washington University School of Medicine	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
RECORDATI GROUP

Countries where clinical trial is conducted

United States,  France,  Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Short and long-term efficacy of osilodrostat	Complete response rate: proportion of enrolled patients with mean Urinary Free Cortisol (mUFC) = ULN	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
Secondary	Short and long-term efficacy of osilodrostat	Partial response rate: proportion of enrolled patients with = 50% reduction from baseline in mean urinary free cortisol (mUFC), (but mUFC > ULN)	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
Secondary	Short and long-term efficacy of osilodrostat	Overall response rate: proportion of enrolled patients with mean urinary free cortisol (mUFC) = ULN or at least 50% reduction from baseline	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
Secondary	Changes in pituitary tumour size	Actual and percentage change from baseline 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
Secondary	Incidence of Adverse Events (Safety and Tolerability)	Incidence of adverse events and laboratory abnormalities using the National Cancer Institute-Common Toxicology Criteria (NCI-CTC) grading scale (version 5.0).	3 years of treatment with osilodrostat
Secondary	Change of mean urinary free cortisol (mUFC)	Actual and percentage change from baseline in 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
Secondary	Change of Serum Cortisol	Actual and percentage change from baseline in Serum Cortisol	at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
Secondary	Change of Late Salivary Cortisol	Actual and percentage change from baseline in Late Salivary Cortisol	at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
Secondary	Change of adrenocorticotropic hormone (ACTH)	Actual and percentage change from baseline in adrenocorticotropic hormone (ACTH)	at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
Secondary	Normalization of Serum Cortisol	Proportion of patients achieving normalisation of Serum Cortisol	at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
Secondary	Normalization of Late Salivary Cortisol	Proportion of patients achieving normalisation 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
Secondary	Normalization of adrenocorticotropic hormone (ACTH)	Proportion of patients achieving normalisation 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
Secondary	Change in Fasting Glucose	Actual and percentage change from baseline in fasting glucose	at baseline before treatment start, then every 3 months through study completion up to three years
Secondary	Change in HbA1c	Actual and percentage change from baseline in HbA1c	at baseline before treatment start, then every 3 months through study completion up to three years
Secondary	Change in Fasting Lipid Profile	Actual and percentage change from baseline in Fasting Lipid Profile	at baseline before treatment start, then every 3 months through study completion up to three years
Secondary	Change in Serum Insulin	Actual and percentage change from baseline in Serum Insulin	at baseline before treatment start, then every 3 months through study completion up to three years
Secondary	Change in Blood Pressure	Actual and percentage change from baseline in Blood Pressure	at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
Secondary	Change in Body Weight	Actual and percentage change from baseline in Body Weight	at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
Secondary	Change in Body Mass Index (BMI)	Actual and percentage change from baseline 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
Secondary	Change in Waist Circumference	Actual and percentage change from baseline in Waist Circumference	at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
Secondary	Change in Facial Rubor	Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature 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
Secondary	Change in Hirsutism	Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature 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
Secondary	Change in Striae	Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature 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
Secondary	Change in Supraclavicular fat pad	Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature 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
Secondary	Change in Dorsal fat pad	Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature 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
Secondary	Change in Proximal muscle wasting (atrophy)	Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature 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
Secondary	Change in Central (abdominal) obesity	Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature 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
Secondary	Change in Ecchymoses (bruises)	Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature 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
Secondary	Changes in Patient-Reported Outcome (PRO) questionnaire Cushing Quality of Life (QoL)	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	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
Secondary	Changes in Patient-Reported Outcome (PRO) questionnaire Euro Quality of Life (EQ) - 5 Dimensions (5D) - 5 Levels (5L)	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	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
Secondary	Changes in Patient-Reported Outcome (PRO) questionnaire Beck Depression Inventory II (BDI-II)	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	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
Secondary	Changes in Patient-Reported Outcome (PRO) questionnaire Patient Global Impression of Change (PGIC)	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	after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years