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

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: Active, not recruiting
• Conditions: Cushing's Syndrome
• Intervention / Treatment: Drug: osilodrostat

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: Interventional
• Enrollment (Anticipated): 180
• Phase: Phase 2

Contacts and Locations

Study Locations

• Argentina
  • Buenos Aires
    • Caba, Buenos Aires, Argentina, C1180AAX
      • Recordati Investigative Site
• Austria
  • Vienna, Austria, A-1090
    • Recordati Investigative Site
• Belgium
  • Leuven, Belgium, 3000
    • Recordati Investigative Site
• Brazil
  • CE
    • Fortaleza, CE, Brazil, 60020-181
      • Recordati Investigative Site
  • RJ
    • Rio de Janeiro, RJ, Brazil, 21941-913
      • Recordati Investigative Site
  • SP
    • Sao Paulo, SP, Brazil, 04039 004
      • Recordati Investigative Site
    • Sao Paulo, SP, Brazil, 05403 000
      • Recordati Investigative Site
• Bulgaria
  • Sofia, Bulgaria, 1431
    • Recordati Investigative Site
• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 2B7
      • Recordati Investigative Site
  • Nova Scotia
    • Halifax, Nova Scotia, Canada, B3H 1V7
      • Recordati Investigative Site
  • Quebec
    • Montreal, Quebec, Canada, H2W 1T8
      • Recordati Investigative Site
    • Sherbrooke, Quebec, Canada, J1H 5N4
      • Recordati Investigative Site
• China
  • Beijing, China, 100034
    • Recordati Investigative Site
  • Beijing, China, 100730
    • Recordati Investigative Site
  • Sichuan
    • Chengdu, Sichuan, China, 610041
      • Recordati Investigative Site
• Costa Rica
  • San Jose, Costa Rica
    • San Pedro, San Jose, Costa Rica, Costa Rica, 1406 1200
      • Recordati Investigative Site
• France
  • Le Kremlin Bicetre, France, 94275
    • Recordati Investigative Site
  • Lille Cedex, France, 59037
    • Recordati Investigative Site
  • Paris, France, 75014
    • Recordati Investigative Site
  • Cedex
    • Pessac, Cedex, France, 33604
      • Recordati Investigative Site
• Germany
  • Erlangen, Germany, 91054
    • Recordati Investigative Site
  • Muenchen, Germany, 81377
    • Recordati Investigative Site
• India
  • New Delhi, India, 110029
    • Recordati Investigative Site
• Italy
  • Napoli, Italy, 80131
    • Recordati Investigative Site
  • AN
    • Ancona, AN, Italy, 60126
      • Recordati Investigative Site
  • PD
    • Padova, PD, Italy, 35128
      • Recordati Investigative Site
  • PI
    • Pisa, PI, Italy, 56124
      • Recordati Investigative Site
• Japan
  • Aichi
    • Nagoya, Aichi, Japan, 460-0001
      • Recordati Investigative Site
  • Hyogo
    • Nishinomiya, Hyogo, Japan, 663 8501
      • Recordati Investigative Site
• Korea, Republic of
  • Seoul, Korea, Republic of, 03080
    • Recordati Investigative Site
  • Seoul, Korea, Republic of, 03722
    • Recordati Investigative Site
• Poland
  • Krakow, Poland, 31-501
    • Recordati Investigative Site
  • Warszawa, Poland, 03 242
    • Recordati Investigative Site
• Russian Federation
  • Moscow, Russian Federation, 117036
    • Recordati Investigative Site
• Spain
  • Madrid, Spain, 28009
    • Recordati Investigative Site
  • Valencia, Spain, 46026
    • Recordati Investigative Site
  • Andalucia
    • Sevilla, Andalucia, Spain, 41013
      • Recordati Investigative Site
  • Galicia
    • La Coruna, Galicia, Spain, 15006
      • Recordati Investigative Site
• Thailand
  • Bangkok, Thailand, 10330
    • Recordati Investigative Site
  • Bangkok, Thailand, 10700
    • Recordati Investigative Site
  • Songkla, Thailand, 90110
    • Recordati Investigative Site
• Turkey
  • Altunizade, Turkey, 34662
    • Recordati Investigative Site
  • Kocaeli, Turkey, 41380
    • Recordati Investigative Site
  • TUR
    • Istanbul, TUR, Turkey, 34098
      • Recordati Investigative Site
• 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

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

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

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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

Outcome Measure	Measure Description	Time Frame
Number of participants with adverse/serious adverse events	To evaluate long term safety	up to 5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of patients with clinical benefit	clinical benefit as assessed by the investigator	up to of 5 years

Collaborators and Investigators

• Sponsor: RECORDATI GROUP
• Investigators: Study Director: Recordati, Recordati AG

Study record dates

Study Major Dates

• Study Start (Actual): October 5, 2018
• Primary Completion (Anticipated): February 28, 2023
• Study Completion (Anticipated): September 27, 2023

Study Registration Dates

• First Submitted: July 20, 2018
• First Submitted That Met QC Criteria: July 27, 2018
• First Posted (Actual): July 30, 2018

Study Record Updates

• Last Update Posted (Actual): May 6, 2022
• Last Update Submitted That Met QC Criteria: May 2, 2022
• Last Verified: May 1, 2022

More Information

Terms related to this study

• Keywords: osilodrostat; LCI699; Cushing's syndrome; endogenous Cushing's syndrome
• Additional Relevant MeSH Terms: Pathologic Processes; Endocrine System Diseases; Disease; Adrenocortical Hyperfunction; Adrenal Gland Diseases; Syndrome; Cushing Syndrome
• Other Study ID Numbers: CLCI699C2X01B

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No