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

NCT03606408 | Completed Phase 2 Results FDA Drug

• 1 other identifier: CLCI699C2X01B
• Study Type: interventional
• Target: 127
• Locations: 21 countries
• Sites: 56

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.

Conditions

Cushing's Syndrome

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse/Serious Adverse Events

  To evaluate long-term safety data with osilodrostat treatment (Frequency and severity of adverse events (AEs)/serious adverse events (SAEs))

  up to 5 years

Secondary Outcomes (1)

• Percentage of Patients With Clinical Benefit

  up to of 5 years

Study Arms (1)

osilodrostat

OTHER

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

Drug: osilodrostat

Interventions

osilodrostat 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.

Also known as: LCI699

osilodrostat

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• RECORDATI GROUP: lead

Study Sites (56)

University of Colorado

Aurora, Colorado, 80045, United States

Location

Emory University School of Medicine G2304 - C2301

Atlanta, Georgia, 30322, United States

Location

Northwestern University SC - LCI699C2301

Chicago, Illinois, 60611, United States

Location

University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, 48109, United States

Location

Columbia University Medical Center New York Presbyterian Neuroendocrine Unit

New York, New York, 10032, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

University of Pennsylvania Medical Center Univ Penn

Philadelphia, Pennsylvania, 19104, United States

Location

Medical College of Wisconsin MCW 2

Milwaukee, Wisconsin, 53226, United States

Location

Sanatorio Guemes

CABA, Buenos Aires, C1180AAX, Argentina

Location

Universitaetsklinik fuer Innere Medizin III

Vienna, A-1090, Austria

Location

Gasthuisberg University Hospital

Leuven, 3000, Belgium

Location

Universidade Federal do Ceara

Fortaleza, Ceará, 60020-181, Brazil

Location

Hospital Universitario Clementino Fraga Filho

Rio de Janeiro, Rio de Janeiro, 21941-913, Brazil

Location

Hospital do Servidor Publico Estadual de Sao Paulo

São Paulo, São Paulo, 04039 004, Brazil

Location

Hospital das Clinicas da Faculdade de Medicina da USP

São Paulo, São Paulo, 05403 000, Brazil

Location

USHATE Akad Ivan Penchev

Sofia, 1431, Bulgaria

Location

University of Alberta Hospital

Edmonton, Alberta, T6G 2B7, Canada

Location

QEII Health Sciences Centre

Halifax, Nova Scotia, B3H 1V7, Canada

Location

Centre de recherche du CHUM CRCHUM

Montreal, Quebec, H2W 1T8, Canada

Location

CHUS Hopital Fleurimont

Sherbrooke, Quebec, J1H 5N4, Canada

Location

The First Affiliated Hopsital, Sun Yat-Sun University

Guangzhou, Guangdong, 510080, China

Location

West China Hospital of Sichuan University

Chengdu, Sichuan, 610041, China

Location

Peking University First Hospital

Beijing, 100034, China

Location

Peking Union Medical College Hospital

Beijing, 100730, China

Location

Clinica Los Yoses

San Pedro, San Jose, Costa Rica, 1406 1200, Costa Rica

Location

CHU de Bordeaux

Pessac, Cedex, 33604, France

Location

Hopital Kremlin Bicetre

Le Kremlin-Bicêtre, 94275, France

Location

CHRU de Lille

Lille, 59037, France

Location

Hopital Cochin

Paris, 75014, France

Location

Universitaetsklinikum Erlangen Nuernberg

Erlangen, 91054, Germany

Location

Universitaetsklinikum Muenchen LMU

München, 81377, Germany

Location

All India Institute of Medical Sciences

New Delhi, 110029, India

Location

AOU Osp Riuniti Umberto I GM Lancisi G Salesi Univ Studi

Ancona, AN, 60126, Italy

Location

Azienda Ospedaliera di Padova Università degli Studi

Padua, PD, 35128, Italy

Location

Az Ospedaliero Universitaria Pisana Pres Osped di Cisanello

Pisa, PI, 56124, Italy

Location

A O Universitaria Policlinico Federico II Univ Studi Fed II

Napoli, 80131, Italy

Location

National Hospital Organization Nagoya Medical Center

Nagoya, Aichi-ken, 460-0001, Japan

Location

Hyogo College of Medicine Hospital

Nishinomiya, Hyōgo, 663 8501, Japan

Location

Erasmus MC

Rotterdam, 3015 GD, Netherlands

Location

SP ZOZ Szpital Uniwersytecki w Krakowie

Krakow, 31-501, Poland

Location

Mazowiecki Szpital Brodnowski

Warsaw, 03 242, Poland

Location

Center for Endocrinology Russian Academy of Med Sciences

Moscow, 117036, Russia

Location

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Yonsei Univ Health System YUCM

Seoul, 03722, South Korea

Location

Hospital Universitario Virgen del Rocio

Seville, Andalusia, 41013, Spain

Location

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

A Coruña, Galicia, 15006, Spain

Location

Hospital Gregorio Maranon

Madrid, 28009, Spain

Location

Hospital Universitario i Politecnico La Fe

Valencia, 46026, Spain

Location

King Chulalongkorn Memorial Hospital

Bangkok, 10330, Thailand

Location

Siriraj Hospital

Bangkok, 10700, Thailand

Location

Songklanagarind Hospital Endocrinology and Metabolism

Songkhla, 90110, Thailand

Location

Istanbul University Cerrahpasa Faculty of Medicine

Istanbul, TUR, 34098, Turkey (Türkiye)

Location

Marmara University Medical Faculty

Altunizade, 34662, Turkey (Türkiye)

Location

Kocaeli University Medical Faculty

Kocaeli, 41380, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Cushing Syndrome

Interventions

Osilodrostat

Condition Hierarchy (Ancestors)

Adrenocortical Hyperfunction; Adrenal Gland Diseases; Endocrine System Diseases

Results Point of Contact

• Title: Mario Maldonado
• Organization: Recordati AG

maldonado.m@recordati.com

+41798030344

Study Officials

• Recordati

  Recordati AG

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 20, 2018
• First Posted: July 30, 2018
• Study Start: October 5, 2018
• Primary Completion: November 16, 2023
• Study Completion: November 16, 2023
• Last Updated: December 18, 2024
• Results First Posted: December 18, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will share

Locations

TU (3); BE (1); AU (1); CO (1); DE (2); ES (4); BR (4); PL (2); AR (1); US (10); IN (1); CA (4); CN (4); TH (3); BU (1); NL (1); KR (2); IT (4); RU (1); FR (4); JP (2)