Canadian Continuation of Roll-over Study in Patients With Endogenous Cushing's Syndrome

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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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

Conditions
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Endogenous Cushing Syndrome

Intervention / Treatment
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* Drug: Intervention/Treatment


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. The patient is currently benefiting from treatment with osilodrostat, as determined by the Investigator. Patient has demonstrated compliance, as assessed by the Investigator, with the parent roll-over study protocol requirements. Willingness and ability to comply with scheduled visits and treatment plans. Written informed consent obtained prior to enrolling into the study Exclusion Criteria: Patient has been permanently discontinued from osilodrostat study treatment in the parent roll-over Recordati-sponsored study. New patients not previously enrolled in parent roll-over Recordatisponsored study. 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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: This is a multi-center (four sites in Canada), open-label study to evaluate the long term safety of osilodrostat in 7 patients currently being treated in a Global Recordati-sponsored study (LCI699C2X01B) and who are judged by their doctor as benefiting from the current study treatment. The study is expected to remain open for approximately 2 years from EOT (end of trial) visit for each patient in roll-over study. Patients will continue to be treated in this study until they are no longer benefiting from their osilodrostat treatment as judged by the Investigator, until one of the protocoldefined discontinuation criteria is met. Additionally, Company (RRD) agrees to provide osilodrostat for investigational use through the study until patients can transition to commercially available and reimbursed drug in patients' province, as long as there is a path, as determine by RRD Canada Inc., to reimbursement.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Participant Group/Arm<br>Other: osilodrostat open label, with patients receiving same dose as provided in the parent study | Drug: Intervention/Treatment<br>* 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.<br>|

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Canada, Endogenous Cushing syndrome, Cushing syndrome, osilodrostat, long term safety