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

RECORDATI GROUP43 sites in 5 countries206 target enrollmentJune 13, 2022

ConditionsEndogenous Cushing's Syndrome

InterventionsOsilodrostat

DrugsOsilodrostat

Overview

Phase: Not Applicable
Intervention: Osilodrostat
Conditions: Endogenous Cushing's Syndrome
Sponsor: RECORDATI GROUP
Enrollment: 206
Locations: 43
Primary Endpoint: Incidence of osilodrostat-related adverse events and serious adverse events
Status: Active, not recruiting
Last Updated: 5 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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.

Registry

clinicaltrials.gov

Start Date

June 13, 2022

End Date

August 1, 2027

Last Updated

5 months ago

Study Type

Observational

Sex

All

Investigators

Sponsor

RECORDATI GROUP

Responsible Party

Sponsor

Eligibility Criteria

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.

Arms & Interventions

Osilodrostat

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

Intervention: Osilodrostat

Outcomes

Primary Outcomes

Incidence of osilodrostat-related adverse events and serious adverse events

Time Frame: 3 years of treatment with osilodrostat

Number of participants with Adverse Events and Serious Adverse Events

Secondary Outcomes

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)
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)
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)
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)
Change in Fasting Glucose(at baseline before treatment start, then every 3 months through study completion up to three years)
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)
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)
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)
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 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 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)
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)
Change in HbA1c(at baseline before treatment start, then every 3 months through study completion up to three years)
Change in Serum Insulin(at baseline before treatment start, then every 3 months through study completion up to three years)
Incidence of Adverse Events (Safety and Tolerability)(3 years of treatment with osilodrostat)
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)
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)
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)
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 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 in Fasting Lipid Profile(at baseline before treatment start, then every 3 months through study completion up to three years)
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)
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)
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)
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 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)
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)
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)
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)