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 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.
Study Type
OBSERVATIONAL
Enrollment
206
oral administration of Osilodrostat tablets at different doses according to patient's need
Incidence of osilodrostat-related adverse events and serious adverse events
Number of participants with Adverse Events and Serious Adverse Events
Time frame: 3 years of treatment with osilodrostat
Short and long-term efficacy of osilodrostat
Complete response rate: proportion of enrolled patients with mean Urinary Free Cortisol (mUFC) ≤ ULN
Time frame: 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
Partial response rate: proportion of enrolled patients with ≥ 50% reduction from baseline in mean urinary free cortisol (mUFC), (but mUFC \> ULN)
Time frame: 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
Overall response rate: proportion of enrolled patients with mean urinary free cortisol (mUFC) ≤ ULN or at least 50% reduction from baseline
Time frame: 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
Actual and percentage change from baseline in pituitary tumour size
Time frame: 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)
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Barrow Neurological Institute
Phoenix, Arizona, United States
Emory University School
Atlanta, Georgia, United States
Northwestern University
Chicago, Illinois, United States
Indiana University Schl-med
Indianapolis, Indiana, United States
St Elizabeth Physicians
Covington, Kentucky, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Mayo Clinic - Rochester
Rochester, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
NYU Grossman School of Medicine
New York, New York, United States
...and 33 more locations
Incidence of adverse events and laboratory abnormalities using the National Cancer Institute-Common Toxicology Criteria (NCI-CTC) grading scale (version 5.0).
Time frame: 3 years of treatment with osilodrostat
Change of mean urinary free cortisol (mUFC)
Actual and percentage change from baseline in mean urinary free cortisol (mUFC)
Time frame: 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
Actual and percentage change from baseline in Serum Cortisol
Time frame: 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
Actual and percentage change from baseline in Late Salivary Cortisol
Time frame: at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
Change of adrenocorticotropic hormone (ACTH)
Actual and percentage change from baseline in adrenocorticotropic hormone (ACTH)
Time frame: at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
Normalization of Serum Cortisol
Proportion of patients achieving normalisation of Serum Cortisol
Time frame: 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
Proportion of patients achieving normalisation of Late Salivary Cortisol
Time frame: 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)
Proportion of patients achieving normalisation of adrenocorticotropic hormone (ACTH)
Time frame: 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
Actual and percentage change from baseline in fasting glucose
Time frame: at baseline before treatment start, then every 3 months through study completion up to three years
Change in HbA1c
Actual and percentage change from baseline in HbA1c
Time frame: at baseline before treatment start, then every 3 months through study completion up to three years
Change in Fasting Lipid Profile
Actual and percentage change from baseline in Fasting Lipid Profile
Time frame: at baseline before treatment start, then every 3 months through study completion up to three years
Change in Serum Insulin
Actual and percentage change from baseline in Serum Insulin
Time frame: at baseline before treatment start, then every 3 months through study completion up to three years
Change in Blood Pressure
Actual and percentage change from baseline in Blood Pressure
Time frame: 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
Actual and percentage change from baseline in Body Weight
Time frame: 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)
Actual and percentage change from baseline in Body Mass Index (BMI)
Time frame: 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
Actual and percentage change from baseline in Waist Circumference
Time frame: 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
Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Facial Rubor
Time frame: 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 Hirsutism
Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Hirsutism
Time frame: 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 Striae
Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Striae
Time frame: 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
Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Supraclavicular fat pad
Time frame: 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 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
Time frame: 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)
Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Proximal muscle wasting (atrophy)
Time frame: 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
Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Central (abdominal) obesity
Time frame: 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)
Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Ecchymoses (bruises)
Time frame: 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)
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
Time frame: 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)
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
Time frame: 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 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
Time frame: 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)
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
Time frame: after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years