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

Cushing's Syndrome Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03606408
• Other study ID #: CLCI699C2X01B
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: October 5, 2018
• Est. completion date: September 27, 2023

Study information

• Verified date: May 2022
• Source: RECORDATI GROUP
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 180
• Est. completion date: September 27, 2023
• Est. primary completion date: February 28, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Cushing Syndrome
• Cushing's Syndrome
• 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.

Locations

Country	Name	City	State
Argentina	Recordati Investigative Site	Caba	Buenos Aires
Austria	Recordati Investigative Site	Vienna
Belgium	Recordati Investigative Site	Leuven
Brazil	Recordati Investigative Site	Fortaleza	CE
Brazil	Recordati Investigative Site	Rio de Janeiro	RJ
Brazil	Recordati Investigative Site	Sao Paulo	SP
Brazil	Recordati Investigative Site	Sao Paulo	SP
Bulgaria	Recordati Investigative Site	Sofia
Canada	Recordati Investigative Site	Edmonton	Alberta
Canada	Recordati Investigative Site	Halifax	Nova Scotia
Canada	Recordati Investigative Site	Montreal	Quebec
Canada	Recordati Investigative Site	Sherbrooke	Quebec
China	Recordati Investigative Site	Beijing
China	Recordati Investigative Site	Beijing
China	Recordati Investigative Site	Chengdu	Sichuan
Costa Rica	Recordati Investigative Site	San Pedro	San Jose, Costa Rica
France	Recordati Investigative Site	Le Kremlin Bicetre
France	Recordati Investigative Site	Lille Cedex
France	Recordati Investigative Site	Paris
France	Recordati Investigative Site	Pessac	Cedex
Germany	Recordati Investigative Site	Erlangen
Germany	Recordati Investigative Site	Muenchen
India	Recordati Investigative Site	New Delhi
Italy	Recordati Investigative Site	Ancona	AN
Italy	Recordati Investigative Site	Napoli
Italy	Recordati Investigative Site	Padova	PD
Italy	Recordati Investigative Site	Pisa	PI
Japan	Recordati Investigative Site	Nagoya	Aichi
Japan	Recordati Investigative Site	Nishinomiya	Hyogo
Korea, Republic of	Recordati Investigative Site	Seoul
Korea, Republic of	Recordati Investigative Site	Seoul
Poland	Recordati Investigative Site	Krakow
Poland	Recordati Investigative Site	Warszawa
Russian Federation	Recordati Investigative Site	Moscow
Spain	Recordati Investigative Site	La Coruna	Galicia
Spain	Recordati Investigative Site	Madrid
Spain	Recordati Investigative Site	Sevilla	Andalucia
Spain	Recordati Investigative Site	Valencia
Thailand	Recordati Investigative Site	Bangkok
Thailand	Recordati Investigative Site	Bangkok
Thailand	Recordati Investigative Site	Songkla
Turkey	Recordati Investigative Site	Altunizade
Turkey	Recordati Investigative Site	Istanbul	TUR
Turkey	Recordati Investigative Site	Kocaeli
United States	University of Michigan Comprehensive Cancer Center	Ann Arbor	Michigan
United States	Emory University School of Medicine G2304 - C2301	Atlanta	Georgia
United States	University of Colorado	Aurora	Colorado
United States	Northwestern University SC - LCI699C2301	Chicago	Illinois
United States	Cleveland Clinic Foundation	Cleveland	Ohio
United States	Medical College of Wisconsin MCW 2	Milwaukee	Wisconsin
United States	Columbia University Medical Center New York Presbyterian Neuroendocrine Unit	New York	New York
United States	Memorial Sloan Kettering Cancer Center	New York	New York
United States	University of Pennsylvania Medical Center Univ Penn	Philadelphia	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
RECORDATI GROUP

Countries where clinical trial is conducted

United States,  Argentina,  Austria,  Belgium,  Brazil,  Bulgaria,  Canada,  China,  Costa Rica,  France,  Germany,  India,  Italy,  Japan,  Korea, Republic of,  Poland,  Russian Federation,  Spain,  Thailand,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with adverse/serious adverse events	To evaluate long term safety	up to 5 years
Secondary	Percentage of patients with clinical benefit	clinical benefit as assessed by the investigator	up to of 5 years