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

NCT ID: NCT03606408

Last Updated: 2024-12-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 127 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-10-05
• Study Completion Date: 2023-11-16

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.

Conditions

Cushing's Syndrome

Keywords

Cushing's syndrome; osilodrostat; LCI699; endogenous Cushing's syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

osilodrostat

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

Group Type: OTHER

osilodrostat

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Other Intervention Names

LCI699

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

RECORDATI GROUP

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Recordati

Role: STUDY_DIRECTOR

Recordati AG

Locations

University of Colorado

Aurora, Colorado, United States

Emory University School of Medicine G2304 - C2301

Atlanta, Georgia, United States

Northwestern University SC - LCI699C2301

Chicago, Illinois, United States

University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, 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

Cleveland Clinic Foundation

Cleveland, Ohio, United States

Oregon Health and Science University

Portland, Oregon, United States

University of Pennsylvania Medical Center Univ Penn

Philadelphia, Pennsylvania, United States

Medical College of Wisconsin MCW 2

Milwaukee, Wisconsin, United States

Sanatorio Guemes

CABA, Buenos Aires, Argentina

Universitaetsklinik fuer Innere Medizin III

Vienna, , Austria

Gasthuisberg University Hospital

Leuven, , Belgium

Universidade Federal do Ceara

Fortaleza, Ceará, Brazil

Hospital Universitario Clementino Fraga Filho

Rio de Janeiro, Rio de Janeiro, Brazil

Hospital do Servidor Publico Estadual de Sao Paulo

São Paulo, São Paulo, Brazil

Hospital das Clinicas da Faculdade de Medicina da USP

São Paulo, São Paulo, Brazil

USHATE Akad Ivan Penchev

Sofia, , Bulgaria

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, Canada

The First Affiliated Hopsital, Sun Yat-Sun University

Guangzhou, Guangdong, China

West China Hospital of Sichuan University

Chengdu, Sichuan, China

Peking University First Hospital

Beijing, , China

Peking Union Medical College Hospital

Beijing, , China

Clinica Los Yoses

San Pedro, San Jose, Costa Rica, Costa Rica

CHU de Bordeaux

Pessac, Cedex, France

Hopital Kremlin Bicetre

Le Kremlin-Bicêtre, , France

CHRU de Lille

Lille, , France

Hopital Cochin

Paris, , France

Universitaetsklinikum Erlangen Nuernberg

Erlangen, , Germany

Universitaetsklinikum Muenchen LMU

München, , Germany

All India Institute of Medical Sciences

New Delhi, , India

AOU Osp Riuniti Umberto I GM Lancisi G Salesi Univ Studi

Ancona, AN, Italy

Azienda Ospedaliera di Padova Università degli Studi

Padua, PD, Italy

Az Ospedaliero Universitaria Pisana Pres Osped di Cisanello

Pisa, PI, Italy

A O Universitaria Policlinico Federico II Univ Studi Fed II

Napoli, , Italy

National Hospital Organization Nagoya Medical Center

Nagoya, Aichi-ken, Japan

Hyogo College of Medicine Hospital

Nishinomiya, Hyōgo, Japan

Erasmus MC

Rotterdam, , Netherlands

SP ZOZ Szpital Uniwersytecki w Krakowie

Krakow, , Poland

Mazowiecki Szpital Brodnowski

Warsaw, , Poland

Center for Endocrinology Russian Academy of Med Sciences

Moscow, , Russia

Seoul National University Hospital

Seoul, , South Korea

Yonsei Univ Health System YUCM

Seoul, , South Korea

Hospital Universitario Virgen del Rocio

Seville, Andalusia, Spain

Complejo Uni. Hosp. A Coruña ( antes Hospital Juan Canalejo)

A Coruña, Galicia, Spain

Hospital Gregorio Maranon

Madrid, , Spain

Hospital Universitario i Politecnico La Fe

Valencia, , Spain

King Chulalongkorn Memorial Hospital

Bangkok, , Thailand

Siriraj Hospital

Bangkok, , Thailand

Songklanagarind Hospital Endocrinology and Metabolism

Songkhla, , Thailand

Istanbul University Cerrahpasa Faculty of Medicine

Istanbul, TUR, Turkey (Türkiye)

Marmara University Medical Faculty

Altunizade, , Turkey (Türkiye)

Kocaeli University Medical Faculty

Kocaeli, , Turkey (Türkiye)

Countries

United States; Argentina; Austria; Belgium; Brazil; Bulgaria; Canada; China; Costa Rica; France; Germany; India; Italy; Japan; Netherlands; Poland; Russia; South Korea; Spain; Thailand; Turkey (Türkiye)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

CLCI699C2X01B

Identifier Type: -

Identifier Source: org_study_id