Roll-over Study in Patients With Endogenous Cushing's Syndrome for LCI699
- Registration Number
- NCT03606408
- Lead Sponsor
- RECORDATI GROUP
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 127
- 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.
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Patient has been permanently discontinued from osilodrostat study treatment in a parent Novartis-sponsor study.
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Patients who are receiving osilodrostat in combination with unapproved or experimental treatments for any type of endogenous CS.
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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
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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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description osilodrostat osilodrostat open label, with patients receiving same dose as provided in the parent study
- Primary Outcome Measures
Name Time Method Number of Participants With Adverse/Serious Adverse Events up to 5 years To evaluate long-term safety data with osilodrostat treatment (Frequency and severity of adverse events (AEs)/serious adverse events (SAEs))
- Secondary Outcome Measures
Name Time Method Percentage of Patients With Clinical Benefit up to of 5 years Proportion of patients with clinical benefit as assessed by the Investigator at scheduled visits based on medical check-up and lab values such as Urine Free Cortisol.
Trial Locations
- Locations (56)
Emory University School of Medicine G2304 - C2301
🇺🇸Atlanta, Georgia, United States
Memorial Sloan Kettering Cancer Center
🇺🇸New York, New York, United States
Columbia University Medical Center New York Presbyterian Neuroendocrine Unit
🇺🇸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
Gasthuisberg University Hospital
🇧🇪Leuven, Belgium
Hospital Universitario Clementino Fraga Filho
🇧🇷Rio de Janeiro, RJ, Brazil
Hospital do Servidor Publico Estadual de Sao Paulo
🇧🇷Sao Paulo, SP, Brazil
Hospital das Clinicas da Faculdade de Medicina da USP
🇧🇷Sao Paulo, SP, Brazil
QEII Health Sciences Centre
🇨🇦Halifax, Nova Scotia, Canada
Centre de recherche du CHUM CRCHUM
🇨🇦Montreal, Quebec, Canada
Hopital Kremlin Bicetre
🇫🇷Le Kremlin Bicetre, France
Universitaetsklinikum Muenchen LMU
🇩🇪Muenchen, Germany
Hyogo College of Medicine Hospital
🇯🇵Nishinomiya, Hyogo, Japan
Seoul National University Hospital
🇰🇷Seoul, Korea, Republic of
Hospital Universitario i Politecnico La Fe
🇪🇸Valencia, Spain
West China Hospital of Sichuan University
🇨🇳Chengdu, Sichuan, China
The First Affiliated Hopsital, Sun Yat-Sun University
🇨🇳Guangzhou, Guangdong, China
Peking Union Medical College Hospital
🇨🇳Beijing, China
Sanatorio Guemes
🇦🇷Caba, Buenos Aires, Argentina
Universitaetsklinik fuer Innere Medizin III
🇦🇹Vienna, Austria
USHATE Akad Ivan Penchev
🇧🇬Sofia, Bulgaria
Erasmus MC
🇳🇱Rotterdam, Netherlands
Center for Endocrinology Russian Academy of Med Sciences
🇷🇺Moscow, Russian Federation
Clinica Los Yoses
🇨🇷San Pedro, San Jose, Costa Rica, Costa Rica
All India Institute of Medical Sciences
🇮🇳New Delhi, India
National Hospital Organization Nagoya Medical Center
🇯🇵Nagoya, Aichi, Japan
SP ZOZ Szpital Uniwersytecki w Krakowie
🇵🇱Krakow, Poland
University of Alberta Hospital
🇨🇦Edmonton, Alberta, Canada
Northwestern University SC - LCI699C2301
🇺🇸Chicago, Illinois, United States
University of Colorado
🇺🇸Aurora, Colorado, United States
Universidade Federal do Ceara
🇧🇷Fortaleza, CE, Brazil
CHUS Hopital Fleurimont
🇨🇦Sherbrooke, Quebec, Canada
CHRU de Lille
🇫🇷Lille Cedex, France
Hopital Cochin
🇫🇷Paris, France
CHU de Bordeaux
🇫🇷Pessac, Cedex, France
AOU Osp Riuniti Umberto I GM Lancisi G Salesi Univ Studi
🇮🇹Ancona, AN, Italy
Az Ospedaliero Universitaria Pisana Pres Osped di Cisanello
🇮🇹Pisa, PI, Italy
Universitaetsklinikum Erlangen Nuernberg
🇩🇪Erlangen, Germany
Azienda Ospedaliera di Padova Università degli Studi
🇮🇹Padova, PD, Italy
A O Universitaria Policlinico Federico II Univ Studi Fed II
🇮🇹Napoli, Italy
Yonsei Univ Health System YUCM
🇰🇷Seoul, Korea, Republic of
Mazowiecki Szpital Brodnowski
🇵🇱Warszawa, Poland
Complejo Uni. Hosp. A Coruña ( antes Hospital Juan Canalejo)
🇪🇸La Coruna, Galicia, Spain
King Chulalongkorn Memorial Hospital
🇹🇭Bangkok, Thailand
Hospital Universitario Virgen del Rocio
🇪🇸Sevilla, Andalucia, Spain
Siriraj Hospital
🇹🇭Bangkok, Thailand
Istanbul University Cerrahpasa Faculty of Medicine
🇹🇷Istanbul, TUR, Turkey
Hospital Gregorio Maranon
🇪🇸Madrid, Spain
Songklanagarind Hospital Endocrinology and Metabolism
🇹🇭Songkla, Thailand
Marmara University Medical Faculty
🇹🇷Altunizade, Turkey
Kocaeli University Medical Faculty
🇹🇷Kocaeli, Turkey
University of Michigan Comprehensive Cancer Center
🇺🇸Ann Arbor, Michigan, United States
Peking University First Hospital
🇨🇳Beijing, China