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

November 22, 2024 updated by: RECORDATI GROUP

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
127

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
    • Buenos Aires
      • Caba, Buenos Aires, Argentina, C1180AAX
        • Sanatorio Güemes
  • Austria
      • Vienna, Austria, A-1090
        • Universitaetsklinik fuer Innere Medizin III
  • Belgium
      • Leuven, Belgium, 3000
        • Gasthuisberg University Hospital
  • Brazil
    • CE
      • Fortaleza, CE, Brazil, 60020-181
        • Universidade Federal do Ceara
    • RJ
      • Rio de Janeiro, RJ, Brazil, 21941-913
        • Hospital Universitario Clementino Fraga Filho
    • SP
      • Sao Paulo, SP, Brazil, 04039 004
        • Hospital do Servidor Publico Estadual de Sao Paulo
      • Sao Paulo, SP, Brazil, 05403 000
        • Hospital das Clinicas da Faculdade de Medicina da USP
  • Bulgaria
      • Sofia, Bulgaria, 1431
        • USHATE Akad Ivan Penchev
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2B7
        • University of Alberta Hospital
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H 1V7
        • QEII Health Sciences Centre
    • Quebec
      • Montreal, Quebec, Canada, H2W 1T8
        • Centre de recherche du CHUM CRCHUM
      • Sherbrooke, Quebec, Canada, J1H 5N4
        • CHUS Hopital Fleurimont
  • China
      • Beijing, China, 100730
        • Peking Union Medical College Hospital
      • Beijing, China, 100034
        • Peking University First Hospital
    • Guangdong
      • Guangzhou, Guangdong, China, 510080
        • The First Affiliated Hopsital, Sun Yat-Sun University
    • Sichuan
      • Chengdu, Sichuan, China, 610041
        • West China Hospital of Sichuan University
  • Costa Rica
    • San Jose, Costa Rica
      • San Pedro, San Jose, Costa Rica, Costa Rica, 1406 1200
        • Clinica Los Yoses
  • France
      • Le Kremlin Bicetre, France, 94275
        • Hôpital Kremlin Bicêtre
      • Lille Cedex, France, 59037
        • CHRU de Lille
      • Paris, France, 75014
        • Hopital Cochin
    • Cedex
      • Pessac, Cedex, France, 33604
        • CHU de Bordeaux
  • Germany
      • Erlangen, Germany, 91054
        • Universitaetsklinikum Erlangen Nuernberg
      • Muenchen, Germany, 81377
        • Universitaetsklinikum Muenchen LMU
  • India
      • New Delhi, India, 110029
        • All India Institute of Medical Sciences
  • Italy
      • Napoli, Italy, 80131
        • A O Universitaria Policlinico Federico II Univ Studi Fed II
    • AN
      • Ancona, AN, Italy, 60126
        • AOU Osp Riuniti Umberto I GM Lancisi G Salesi Univ Studi
    • PD
      • Padova, PD, Italy, 35128
        • Azienda Ospedaliera di Padova Università degli Studi
    • PI
      • Pisa, PI, Italy, 56124
        • Az Ospedaliero Universitaria Pisana Pres Osped di Cisanello
  • Japan
    • Aichi
      • Nagoya, Aichi, Japan, 460-0001
        • National Hospital Organization Nagoya Medical Center
    • Hyogo
      • Nishinomiya, Hyogo, Japan, 663 8501
        • Hyogo College of Medicine Hospital
  • Korea, Republic of
      • Seoul, Korea, Republic of, 03080
        • Seoul National University Hospital
      • Seoul, Korea, Republic of, 03722
        • Yonsei Univ Health System YUCM
  • Netherlands
      • Rotterdam, Netherlands, 3015 GD
        • Erasmus MC
  • Poland
      • Krakow, Poland, 31-501
        • SP ZOZ Szpital Uniwersytecki w Krakowie
      • Warszawa, Poland, 03 242
        • Mazowiecki Szpital Bródnowski
  • Russian Federation
      • Moscow, Russian Federation, 117036
        • Center for Endocrinology Russian Academy of Med Sciences
  • Spain
      • Madrid, Spain, 28009
        • Hospital Gregorio Marañon
      • Valencia, Spain, 46026
        • Hospital Universitario i Politecnico La Fe
    • Andalucia
      • Sevilla, Andalucia, Spain, 41013
        • Hospital Universitario Virgen del Rocio
    • Galicia
      • La Coruna, Galicia, Spain, 15006
        • Complejo Uni. Hosp. A Coruña ( antes Hospital Juan Canalejo)
  • Thailand
      • Bangkok, Thailand, 10330
        • King Chulalongkorn Memorial Hospital
      • Bangkok, Thailand, 10700
        • Siriraj Hospital
      • Songkla, Thailand, 90110
        • Songklanagarind Hospital Endocrinology and Metabolism
  • Turkey
      • Altunizade, Turkey, 34662
        • Marmara University Medical Faculty
      • Kocaeli, Turkey, 41380
        • Kocaeli University Medical Faculty
    • TUR
      • Istanbul, TUR, Turkey, 34098
        • Istanbul University Cerrahpasa Faculty of Medicine
  • United States
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University School of Medicine G2304 - C2301
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University SC - LCI699C2301
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan Comprehensive Cancer Center
    • New York
      • New York, New York, United States, 10065
        • Memorial Sloan Kettering Cancer Center
      • New York, New York, United States, 10032
        • Columbia University Medical Center New York Presbyterian Neuroendocrine Unit
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic Foundation
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania Medical Center Univ Penn
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin MCW 2

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Other: osilodrostat
open label, with patients receiving same dose as provided in the parent study
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.
Other Names:
  • LCI699

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of Participants With Adverse/Serious Adverse Events
Time Frame: up to 5 years
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 Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percentage of Patients With Clinical Benefit
Time Frame: 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.
up to of 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
RECORDATI GROUP

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Recordati, Recordati AG

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 5, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 16, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 16, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 20, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 27, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 30, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 25, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 22, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CLCI699C2X01B

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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