Canadian Continuation of Roll-over Study in Patients With Endogenous Cushing's Syndrome (ISS-CA-2023)

Continuation of an Open-label, Multi-center Study to Assess Long Term Safety in Canadian Patients With Endogenous Cushing's Syndrome Who Have Completed Prior Recordati-sponsored Osilodrostat (LCI699) Study LCI699C2X01B and Are Judged by the Investigator to Benefit From Continued Treatment With Osilodrostat

The purpose of this study is to continue the evaluation of long-term safety of osilodrostat in 7 Canadian patients who have already received osilodrostat treatment in a previous Global Recordati-sponsored roll-over study and who, based on investigators' judgement, will continue benefiting with its administration.

Study Overview

• Status: Active, not recruiting
• Conditions: Endogenous Cushing Syndrome
• Intervention / Treatment: Drug: Intervention/Treatment

Detailed Description

There will be no screening period for this study. Eligible subjects can start their treatment with osilodrostat as soon as they are enrolled in the study. The first study visit will be scheduled at the time of the last study visit for the parent study. Subjects must return to the study center at least on a quarterly basis (every 12 weeks ± 2 weeks) for safety and clinical benefit assessments, and resupply of study medication. Drug dispensing and administration information and adverse events will be collected. The subject may return to the clinic at any given time as per standard of care or treating physician recommendation; however, only the quarterly study visits will be recorded in the Case Report Form (CRF). Study medication dispensed will be recorded in the CRF dose administration page.

All adverse events and serious adverse events, including pregnancy, will be collected throughout the study. Subjects will continue to be treated in this roll-over study until they are no longer benefiting from their osilodrostat treatment as judged by the Investigator or until osilodrostat is commercially available or until one of other discontinuation criteria is met.

• Study Type: Interventional
• Enrollment (Actual): 7
• Phase: Phase 4

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 2E1
      • University of Alberta Hospital - Alberta Diabetes Institute
  • Nova Scotia
    • Halifax, Nova Scotia, Canada, B3H 1V7
      • Nova Scotia Health
  • Quebec
    • Sherbrooke, Quebec, Canada, J1H 5N4
      • CHUS Sherbrooke

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Patient is currently participating in a Global Recordati-sponsored roll-over study receiving osilodrostat for any type of endogenous CS and has fulfilled all their requirements in the parent study.
2. The patient is currently benefiting from treatment with osilodrostat, as determined by the Investigator.
3. Patient has demonstrated compliance, as assessed by the Investigator, with the parent roll-over study protocol requirements.
4. Willingness and ability to comply with scheduled visits and treatment plans.
5. Written informed consent obtained prior to enrolling into the study

Exclusion Criteria:

1. Patient has been permanently discontinued from osilodrostat study treatment in the parent roll-over Recordati-sponsored study.
2. New patients not previously enrolled in parent roll-over Recordatisponsored study.
3. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory evaluation.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Participant Group/Arm; Other: osilodrostat open label, with patients receiving same dose as provided in the parent study	Drug: Intervention/Treatment; Drug: osilodrostat - osilodrostat, in the form of film coated tablets for oral administration, in the following tablet strengths: 1mg, 5mg, 10mg. Each strength has unique tablet size, colour and imprint.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with adverse/serious adverse events	To evaluate long term safety	up to 2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of patients with clinical benefit	clinical benefit as assessed by the investigator	up to 2 years

Collaborators and Investigators

• Sponsor: Centre hospitalier de l'Université de Montréal (CHUM)
• Collaborators: Recordati Rare Diseases
• Investigators: Principal Investigator: André Lacroix, MD, Centre Hospitalier de l'Universite de Montreal (CHUM)

Study record dates

Study Major Dates

• Study Start (Actual): October 24, 2023
• Primary Completion (Estimated): November 24, 2025
• Study Completion (Estimated): November 24, 2025

Study Registration Dates

• First Submitted: November 9, 2023
• First Submitted That Met QC Criteria: November 9, 2023
• First Posted (Actual): November 14, 2023

Study Record Updates

• Last Update Posted (Actual): November 14, 2023
• Last Update Submitted That Met QC Criteria: November 9, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: osilodrostat; Canada; Cushing syndrome; long term safety; Endogenous Cushing syndrome
• Additional Relevant MeSH Terms: Pathologic Processes; Endocrine System Diseases; Disease; Adrenocortical Hyperfunction; Adrenal Gland Diseases; Syndrome; Cushing Syndrome
• Other Study ID Numbers: MP-02-2024-11788; 278454 (Other Identifier: Health Canada)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No