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: Completed

Administrative data

• NCT number: NCT03606408
• Other study ID #: CLCI699C2X01B
• Status: Completed
• Phase: Phase 2
• Start date: October 5, 2018
• Est. completion date: November 16, 2023

Study information

• Verified date: May 2024
• 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: Completed
• Enrollment: 127
• Est. completion date: November 16, 2023
• Est. primary completion date: November 16, 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	Sanatorio Guemes	Caba	Buenos Aires
Austria	Universitaetsklinik fuer Innere Medizin III	Vienna
Belgium	Gasthuisberg University Hospital	Leuven
Brazil	Universidade Federal do Ceara	Fortaleza	CE
Brazil	Hospital Universitario Clementino Fraga Filho	Rio de Janeiro	RJ
Brazil	Hospital das Clinicas da Faculdade de Medicina da USP	Sao Paulo	SP
Brazil	Hospital do Servidor Publico Estadual de Sao Paulo	Sao Paulo	SP
Bulgaria	USHATE Akad Ivan Penchev	Sofia
Canada	University of Alberta Hospital	Edmonton	Alberta
Canada	QEII Health Sciences Centre	Halifax	Nova Scotia
Canada	Centre de recherche du CHUM CRCHUM	Montreal	Quebec
Canada	CHUS Hopital Fleurimont	Sherbrooke	Quebec
China	Peking Union Medical College Hospital	Beijing
China	Peking University First Hospital	Beijing
China	West China Hospital of Sichuan University	Chengdu	Sichuan
China	The First Affiliated Hopsital, Sun Yat-Sun University	Guangzhou	Guangdong
Costa Rica	Clinica Los Yoses	San Pedro	San Jose, Costa Rica
France	Hopital Kremlin Bicetre	Le Kremlin Bicetre
France	CHRU de Lille	Lille Cedex
France	Hopital Cochin	Paris
France	CHU de Bordeaux	Pessac	Cedex
Germany	Universitaetsklinikum Erlangen Nuernberg	Erlangen
Germany	Universitaetsklinikum Muenchen LMU	Muenchen
India	All India Institute of Medical Sciences	New Delhi
Italy	AOU Osp Riuniti Umberto I GM Lancisi G Salesi Univ Studi	Ancona	AN
Italy	A O Universitaria Policlinico Federico II Univ Studi Fed II	Napoli
Italy	Azienda Ospedaliera di Padova Università degli Studi	Padova	PD
Italy	Az Ospedaliero Universitaria Pisana Pres Osped di Cisanello	Pisa	PI
Japan	National Hospital Organization Nagoya Medical Center	Nagoya	Aichi
Japan	Hyogo College of Medicine Hospital	Nishinomiya	Hyogo
Korea, Republic of	Seoul National University Hospital	Seoul
Korea, Republic of	Yonsei Univ Health System YUCM	Seoul
Netherlands	Erasmus MC	Rotterdam
Poland	SP ZOZ Szpital Uniwersytecki w Krakowie	Krakow
Poland	Mazowiecki Szpital Brodnowski	Warszawa
Russian Federation	Center for Endocrinology Russian Academy of Med Sciences	Moscow
Spain	Complejo Uni. Hosp. A Coruña ( antes Hospital Juan Canalejo)	La Coruna	Galicia
Spain	Hospital Gregorio Maranon	Madrid
Spain	Hospital Universitario Virgen del Rocio	Sevilla	Andalucia
Spain	Hospital Universitario i Politecnico La Fe	Valencia
Thailand	King Chulalongkorn Memorial Hospital	Bangkok
Thailand	Siriraj Hospital	Bangkok
Thailand	Songklanagarind Hospital Endocrinology and Metabolism	Songkla
Turkey	Marmara University Medical Faculty	Altunizade
Turkey	Istanbul University Cerrahpasa Faculty of Medicine	Istanbul	TUR
Turkey	Kocaeli University Medical Faculty	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,  Netherlands,  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