Roll-over Study in Patients With Endogenous Cushing's Syndrome for LCI699

• ClinicalTrials.gov Identifier: NCT03606408
• Recruitment Status: Unknown
• First Posted: July 30, 2018
• Last Update Posted: May 6, 2022
• Sponsor: RECORDATI GROUP
• Information provided by (Responsible Party): RECORDATI GROUP

Tracking Information

• First Submitted Date: July 20, 2018
• First Posted Date: July 30, 2018
• Last Update Posted Date: May 6, 2022
• Actual Study Start Date: October 5, 2018
• Estimated Primary Completion Date: February 28, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 27, 2018)

Number of participants with adverse/serious adverse events [ Time Frame: up to 5 years ]

To evaluate long term safety

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: July 27, 2018)

Percentage of patients with clinical benefit [ Time Frame: up to of 5 years ]

clinical benefit as assessed by the investigator

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Roll-over Study in Patients With Endogenous Cushing's Syndrome for LCI699
• Official Title: An Open-label, Multi-center, Roll-over Study to Assess Long Term Safety in Patients With Endogenous Cushing's Syndrome Who Have Completed a Prior Novartis-sponsored Osilodrostat (LCI699) Study and Are Judged by the Investigator to Benefit From Continued Treatment With Osilodrostat
• Brief Summary: The purpose of this study is the evaluation of long-term safety of osilodrostat in patients who have already received osilodrostat treatment in a previous Global Novartis-sponsored trial 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.

• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Cushing's Syndrome

Intervention

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.

Other Name: LCI699

Study Arms

osilodrostat

open label, with patients receiving same dose as provided in the parent study

Intervention: Drug: osilodrostat

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: July 27, 2018): 180
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: September 27, 2023
• Estimated Primary Completion Date: February 28, 2023 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Patient is currently participating in a Global Novartis-sponsored study receiving osilodrostat for any type of endogenous CS and has fulfilled all their requirements in the parent study.
• 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 study protocol requirements.
• Willingness and ability to comply with scheduled visits and treatment plans.
• Written informed consent obtained prior to enrolling into the roll-over study before evaluating the applicability of the subject's participating in the study. -- If consent cannot be expressed in writing, it must be formally documented and witnessed, ideally via an independent trusted witness.

Exclusion Criteria:

• Patient has been permanently discontinued from osilodrostat study treatment in a parent Novartis-sponsor study.
• Patients who are receiving osilodrostat in combination with unapproved or experimental treatments for any type of endogenous CS.
• 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 test. Pregnant or nursing (lactating) women

• Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 1 week of study after stopping medication. Highly effective contraception methods include:

  • Total abstinence (when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception
  • Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or tubal ligation. at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment
  • Male sterilization (at least 6 months prior to baseline). The vasectomized male partner should be the sole partner for that subject
  • Use of oral, (estrogen and progesterone), injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 75 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Argentina, Austria, Belgium, Brazil, Bulgaria, Canada, China, Costa Rica, France, Germany, India, Italy, Japan, Korea, Republic of, Poland, Russian Federation, Spain, Thailand, Turkey, United States

Administrative Information

• NCT Number: NCT03606408
• Other Study ID Numbers: CLCI699C2X01B
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: RECORDATI GROUP
• Original Responsible Party: Novartis Pharmaceuticals
• Current Study Sponsor: RECORDATI GROUP
• Original Study Sponsor: Novartis Pharmaceuticals
• Collaborators: Not Provided

Investigators

• Study Director: Recordati; Recordati AG

• PRS Account: RECORDATI GROUP
• Verification Date: May 2022