Clinical trial NCT05382156: Non-interventional Study on Osilodrostat in Patients With Endogenous Cushing's Syndrome
Official title: A Non-interventional Study to Assess the Long-term Safety and Efficacy of Osilodrostat in Patients With Endogenous Cushing's Syndrome
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Endogenous Cushing's Syndrome
Keywords: Cushing's syndrome
Osilodrostat
Lead sponsor: RECORDATI GROUP
Sponsor class: INDUSTRY
First posted: 2022-05-19
Enrollment: 206
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
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.

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.

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.

Primary outcomes:
measure: Incidence of osilodrostat-related adverse events and serious adverse events
timeFrame: 3 years of treatment with osilodrostat
Secondary outcomes:
measure: Short and long-term efficacy of osilodrostat
timeFrame: 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
measure: Short and long-term efficacy of osilodrostat
timeFrame: 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
measure: Short and long-term efficacy of osilodrostat
timeFrame: 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
measure: Changes in pituitary tumour size
timeFrame: at baseline before treatment start, after 6 months of treatment, then every 12 months through study completion up to three years
measure: Incidence of Adverse Events (Safety and Tolerability)
timeFrame: 3 years of treatment with osilodrostat
measure: Change of mean urinary free cortisol (mUFC)
timeFrame: at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
measure: Change of Serum Cortisol
timeFrame: at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
measure: Change of Late Salivary Cortisol
timeFrame: at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
measure: Change of adrenocorticotropic hormone (ACTH)
timeFrame: at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
measure: Normalization of Serum Cortisol
timeFrame: at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
measure: Normalization of Late Salivary Cortisol
timeFrame: at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
measure: Normalization of adrenocorticotropic hormone (ACTH)
timeFrame: at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
measure: Change in Fasting Glucose
timeFrame: at baseline before treatment start, then every 3 months through study completion up to three years
measure: Change in HbA1c
timeFrame: at baseline before treatment start, then every 3 months through study completion up to three years
measure: Change in Fasting Lipid Profile
timeFrame: at baseline before treatment start, then every 3 months through study completion up to three years
measure: Change in Serum Insulin
timeFrame: at baseline before treatment start, then every 3 months through study completion up to three years
measure: Change in Blood Pressure
timeFrame: at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
measure: Change in Body Weight
timeFrame: at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
measure: Change in Body Mass Index (BMI)
timeFrame: at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
measure: Change in Waist Circumference
timeFrame: at baseline before treatment start, after 1 month of treatment, then every 3 months through study completion up to three years
measure: Change in Facial Rubor
timeFrame: 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
measure: Change in Hirsutism
timeFrame: 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
measure: Change in Striae
timeFrame: 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
measure: Change in Supraclavicular fat pad
timeFrame: 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
measure: Change in Dorsal fat pad
timeFrame: 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
measure: Change in Proximal muscle wasting (atrophy)
timeFrame: 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
measure: Change in Central (abdominal) obesity
timeFrame: 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
measure: Change in Ecchymoses (bruises)
timeFrame: 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
measure: Changes in Patient-Reported Outcome (PRO) questionnaire Cushing Quality of Life (QoL)
timeFrame: 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
measure: Changes in Patient-Reported Outcome (PRO) questionnaire Euro Quality of Life (EQ) - 5 Dimensions (5D) - 5 Levels (5L)
timeFrame: 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
measure: Changes in Patient-Reported Outcome (PRO) questionnaire Beck Depression Inventory II (BDI-II)
timeFrame: 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
measure: Changes in Patient-Reported Outcome (PRO) questionnaire Patient Global Impression of Change (PGIC)
timeFrame: after 3 months of treatment, after 6 months of treatment, then every 6 months through study completion up to three years
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Barrow Neurological Institute
state: Arizona
city: Atlanta
country: United States
facility: Emory University School
state: Georgia
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University Schl-med
state: Indiana
city: Covington
country: United States
facility: St Elizabeth Physicians
state: Kentucky
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic - Rochester
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: New York
country: United States
facility: NYU Grossman School of Medicine
state: New York
city: New York
country: United States
facility: Memorial Sloan-Kettering Cancer Center (MSKCC) - New York
state: New York
city: Columbus
country: United States
facility: Endocrinology Research Associates, Inc.
state: Ohio
city: Portland
country: United States
facility: Oregon Health And Science University
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania Medical Center
state: Pennsylvania
city: Milwaukee
country: United States
facility: Froedtert Hospital
state: Wisconsin
city: Bordeaux
country: France
facility: Hôpital Haut-Lévêque
city: Bron cedex
country: France
facility: Hospices Civiles de Lyon
city: Grenoble
country: France
facility: CHU de Grenoble site Nord
city: Le Kremlin-Bicêtre
country: France
facility: Groupement Hospitalier Sud - Hôpital Bicêtre
city: Lille
country: France
facility: Hopital Claude Huriez - CHRU Lille
city: Marseille
country: France
facility: Hopital de la Conception - APHM
city: Nancy
country: France
facility: Hôpital de Brabois
city: Nantes
country: France
facility: CHU de Nantes-Hopital Laennec
city: Paris
country: France
facility: Hôpital Cochin
city: Toulouse
country: France
facility: Hopital Larrey
city: Berlin
country: Germany
facility: Charité Universitaetsmedizin Berlin
city: Berlin
country: Germany
facility: Medicover Berlin-Mitte MVZ
city: Duesseldorf
country: Germany
facility: Universitaet Bielefeld - Klinikum Bielefeld - Mitte
city: Frankfurt
country: Germany
facility: Universitaetsklinikum Frankfurt Goethe-Universitaet
city: Frankfurt
country: Germany
facility: Endokrinologikum Frankfurt
city: Hamburg
country: Germany
facility: Amedes Experts
city: Köln
country: Germany
facility: Medicover Köln
city: Munich
country: Germany
facility: Medicover Neuroendokrinologie
city: Munich
country: Germany
facility: Ludwig-Maximilians University of Munich
city: Oldenburg
country: Germany
facility: Medicover MVZ Oldenburg
city: Würzburg
country: Germany
facility: Universitaetsklinikum Wuerzburg
city: Ancona
country: Italy
facility: Azienda Ospedaliero Universitaria Ospedali Riuniti
city: Milan
country: Italy
facility: Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria "Federico II"
city: Roma
country: Italy
facility: Policlinico Umberto I
city: Roma
country: Italy
facility: Azienda Ospedaliera Sant'Andrea-Università di Roma La Sapienza
city: Nijmegen
country: Netherlands
facility: Radboud University Nijmegen
city: Rotterdam
country: Netherlands
facility: Erasmus MC
Study officials:
affiliation: Recordati AG - Head of Clinical Development
name: Mario Maldonado, MD
role: STUDY_CHAIR
References: