Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT05382156

Non-interventional Study on Osilodrostat in Patients With 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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Endogenous Cushing's Syndrome Adrenal Gland Diseases Adrenocortical Hyperfunction Cushing Syndrome Endocrine System Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Hôpital Haut-Lévêque, Bordeaux, France

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Osilodrostat

Drug

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

Other names: Isturisa

Primary outcomes

  1. 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

  1. Short and long-term efficacy of osilodrostat

    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

    Complete response rate: proportion of enrolled patients with mean Urinary Free Cortisol (mUFC) ≤ ULN

  2. Short and long-term efficacy of osilodrostat

    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

    Partial response rate: proportion of enrolled patients with ≥ 50% reduction from baseline in mean urinary free cortisol (mUFC), (but mUFC > ULN)

  3. Short and long-term efficacy of osilodrostat

    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

    Overall response rate: proportion of enrolled patients with mean urinary free cortisol (mUFC) ≤ ULN or at least 50% reduction from baseline

  4. Changes 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

    Actual and percentage change from baseline in pituitary tumour size

  5. Incidence of Adverse Events (Safety and Tolerability)

    Time frame: 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).

  6. Change of 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

    Actual and percentage change from baseline in mean urinary free cortisol (mUFC)

  7. Change 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

    Actual and percentage change from baseline in Serum Cortisol

  8. Change 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

    Actual and percentage change from baseline in Late Salivary Cortisol

  9. Change 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

    Actual and percentage change from baseline in adrenocorticotropic hormone (ACTH)

  10. Normalization 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

    Proportion of patients achieving normalisation of Serum Cortisol

  11. Normalization 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

    Proportion of patients achieving normalisation of Late Salivary Cortisol

  12. Normalization 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

    Proportion of patients achieving normalisation of adrenocorticotropic hormone (ACTH)

  13. Change in Fasting Glucose

    Time frame: 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

  14. Change in HbA1c

    Time frame: at baseline before treatment start, then every 3 months through study completion up to three years

    Actual and percentage change from baseline in HbA1c

  15. Change in Fasting Lipid Profile

    Time frame: 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

  16. Change in Serum Insulin

    Time frame: 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

  17. Change 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

    Actual and percentage change from baseline in Blood Pressure

  18. Change 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

    Actual and percentage change from baseline in Body Weight

  19. Change 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

    Actual and percentage change from baseline in Body Mass Index (BMI)

  20. Change 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

    Actual and percentage change from baseline in Waist Circumference

  21. Change in 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 from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Facial Rubor

  22. Change in 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 from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Hirsutism

  23. Change in 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 from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Striae

  24. Change in 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 from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Supraclavicular fat pad

  25. Change in 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 from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Dorsal fat pad

  26. Change in 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 from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Proximal muscle wasting (atrophy)

  27. Change in 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 from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Central (abdominal) obesity

  28. Change in 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

    Change from baseline in incidence and grade of severity at physical examination of the Cushing's syndrome clinical feature Ecchymoses (bruises)

  29. Changes in Patient-Reported Outcome (PRO) questionnaire Cushing Quality of Life (QoL)

    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

    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

  30. Changes in Patient-Reported Outcome (PRO) questionnaire Euro Quality of Life (EQ) - 5 Dimensions (5D) - 5 Levels (5L)

    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

    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

  31. Changes in Patient-Reported Outcome (PRO) questionnaire Beck Depression Inventory II (BDI-II)

    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

    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

  32. Changes in Patient-Reported Outcome (PRO) questionnaire Patient Global Impression of Change (PGIC)

    Time frame: 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

Sponsors and collaborators

Lead sponsor

RECORDATI GROUP

Industry

Registry information

Official study title

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

Acronym: LINC6

Important dates

Study start
2022
Primary completion
2027
Study completion
2027
First posted
May 19, 2022
Registry last updated
Nov 20, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.