LINC7: Osilodrostat for the Treatment of Non-Cushing's Disease Cushing's Syndrome

Sponsor
RECORDATI GROUP (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05633953
Collaborator
(none)
50
5

Study Details

Study Description

Brief Summary

This is a multi-centre, observational, non-comparative, retrospective cohort study designed to evaluate the long-term safety and effectiveness of osilodrostat in non-CD CS patients. Patients treated with oral osilodrostat regardless of the duration of their treatment will be followed retrospectively for up to 36 months after initiating osilodrostat.

Condition or Disease Intervention/Treatment Phase

Study Design

Study Type:
Observational
Anticipated Enrollment :
50 participants
Observational Model:
Other
Time Perspective:
Retrospective
Official Title:
A Retrospective Observational Study to Evaluate the Safety and Effectiveness of Osilodrostat for the Treatment of Non-Cushing's Disease Cushing's Syndrome (LINC7)
Anticipated Study Start Date :
Dec 1, 2022
Anticipated Primary Completion Date :
May 1, 2023
Anticipated Study Completion Date :
May 1, 2023

Outcome Measures

Primary Outcome Measures

  1. Mean Urinary Free Cortisol (mUFC) [12 weeks]

    Number and proportion of patients with Mean Urinary Free Cortisol (mUFC) ≤ upper limit of normal (ULN)

Secondary Outcome Measures

  1. Morning serum cortisol [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Change from baseline

  2. Mean Urinary Free Cortisol (mUFC) [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Change from baseline

  3. Body Mass Index (BMI) [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  4. Electrocardiogram (ECG) [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  5. Co-morbidities [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  6. Blood Pressure [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  7. Sodium [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  8. Potassium [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  9. Calcium [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  10. CO2 [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  11. Glucose Levels [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  12. Glycated haemoglobin (HBA1c) [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  13. Plasma Adrenocorticotropic hormone (ACTH) [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  14. Serum 11-Deoxycortisol [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  15. Plasma 11-Deoxycorticosterone [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  16. Plasma Aldosterone [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  17. Plasma Renin [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  18. Total Serum Testosterone or oestradiol (per patient sex) [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  19. Serum LH [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

  20. Serum FSH [At Weeks 4, 8, 12, 20, 28, 36 of treatment and every additional 12 weeks of treatment until the earlier of loss to follow-up, treatment discontinuation, death and up to 36 months of retrospective follow-up.]

    Actual and percentage change from Baseline

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
  1. Male and female patients ≥18 years old with diagnosis of CS, except for CD (i.e., an aetiology of adrenal adenoma, adrenocortical carcinoma, adrenal hyperplasia, or ectopic adrenocorticotropic hormone secretion). Patients should have a contemporaneously documented diagnosis of CS as per effective guidelines.

  2. Patients treated with osilodrostat between April 2019 and study start date as part of ATU programme or commercialisation.

Exclusion Criteria:
  1. Patients who participated in a clinical trial anytime during the study period.

  2. Patients with Pseudo-Cushing's syndrome, cyclic CS, or iatrogenic CS.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

  • RECORDATI GROUP

Investigators

  • Study Director: Alberto M PEDRONCELLI, MD, RECORDATI GROUP

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
RECORDATI GROUP
ClinicalTrials.gov Identifier:
NCT05633953
Other Study ID Numbers:
  • LCI699-RECAG-NI-0596
First Posted:
Dec 1, 2022
Last Update Posted:
Dec 1, 2022
Last Verified:
Nov 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by RECORDATI GROUP
Additional relevant MeSH terms:

Study Results

No Results Posted as of Dec 1, 2022