Clinical trial NCT06131580: Canadian Continuation of Roll-over Study in Patients With Endogenous Cushing's Syndrome
Official title: 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE4
Conditions: Endogenous Cushing Syndrome
Keywords: Endogenous Cushing syndrome
Cushing syndrome
osilodrostat
long term safety
Canada
Lead sponsor: Centre hospitalier de l'Université de Montréal (CHUM)
Sponsor class: OTHER
Collaborators: name: Recordati Rare Diseases
First posted: 2023-11-14
Enrollment: 7
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention 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.
Primary purpose: TREATMENT

Brief summary: 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: 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of participants with adverse/serious adverse events
timeFrame: up to 2 years
Secondary outcomes:
measure: Percentage of patients with clinical benefit
timeFrame: up to 2 years
Other outcomes:
Locations:
city: Edmonton
country: Canada
facility: University of Alberta Hospital - Alberta Diabetes Institute
state: Alberta
city: Halifax
country: Canada
facility: Nova Scotia Health
state: Nova Scotia
city: Sherbrooke
country: Canada
facility: CHUS Sherbrooke
state: Quebec
Study officials:
affiliation: Centre hospitalier de l'Université de Montréal (CHUM)
name: André Lacroix, MD
role: PRINCIPAL_INVESTIGATOR
References: