Cushing's SyndromePhase 2NCT03606408
Roll-over Study in Patients With Endogenous Cushing's Syndrome for LCI699
A Phase 2 study testing osilodrostat in people with Cushing's Syndrome, run by RECORDATI GROUP at 56 sites in 21 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Cushing's Syndrome
- What is being tested?
- osilodrostat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 127 participants
- Where
- 56 sitesin 21 countries
- Study dates
- 5 Oct 2018to 16 Nov 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
56 sites in 21 countries.
56 sites in 21 countries
United States10 sites
University of Michigan Comprehensive Cancer Center
Ann Arbor, Michigan, 48109
Emory University School of Medicine G2304 - C2301
Atlanta, Georgia, 30322
University of Colorado
Aurora, Colorado, 80045
Northwestern University SC - LCI699C2301
Chicago, Illinois, 60611
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Medical College of Wisconsin MCW 2
Milwaukee, Wisconsin, 53226
Columbia University Medical Center New York Presbyterian Neuroendocrine Unit
New York, New York, 10032
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
University of Pennsylvania Medical Center Univ Penn
Philadelphia, Pennsylvania, 19104
Oregon Health and Science University
Portland, Oregon, 97239
Brazil4 sites
Universidade Federal do Ceara
Fortaleza, Ceará, 60020-181
Hospital Universitario Clementino Fraga Filho
Rio de Janeiro, Rio de Janeiro, 21941-913
Hospital do Servidor Publico Estadual de Sao Paulo
São Paulo, São Paulo, 04039 004
Hospital das Clinicas da Faculdade de Medicina da USP
São Paulo, São Paulo, 05403 000
Canada4 sites
University of Alberta Hospital
Edmonton, Alberta, T6G 2B7
QEII Health Sciences Centre
Halifax, Nova Scotia, B3H 1V7
Centre de recherche du CHUM CRCHUM
Montreal, Quebec, H2W 1T8
CHUS Hopital Fleurimont
Sherbrooke, Quebec, J1H 5N4
China4 sites
Peking University First Hospital
Beijing, 100034
Peking Union Medical College Hospital
Beijing, 100730
West China Hospital of Sichuan University
Chengdu, Sichuan, 610041
The First Affiliated Hopsital, Sun Yat-Sun University
Guangzhou, Guangdong, 510080
France4 sites
Hopital Kremlin Bicetre
Le Kremlin-Bicêtre, 94275
CHRU de Lille
Lille, 59037
Hopital Cochin
Paris, 75014
CHU de Bordeaux
Pessac, Cedex, 33604
Italy4 sites
AOU Osp Riuniti Umberto I GM Lancisi G Salesi Univ Studi
Ancona, AN, 60126
A O Universitaria Policlinico Federico II Univ Studi Fed II
Naples, 80131
Azienda Ospedaliera di Padova Università degli Studi
Padova, PD, 35128
Az Ospedaliero Universitaria Pisana Pres Osped di Cisanello
Pisa, PI, 56124
Spain4 sites
Complejo Uni. Hosp. A Coruña ( antes Hospital Juan Canalejo)
A Coruña, Galicia, 15006
Hospital Gregorio Maranon
Madrid, 28009
Hospital Universitario Virgen del Rocio
Seville, Andalusia, 41013
Hospital Universitario i Politecnico La Fe
Valencia, 46026
Thailand3 sites
King Chulalongkorn Memorial Hospital
Bangkok, 10330
Siriraj Hospital
Bangkok, 10700
Songklanagarind Hospital Endocrinology and Metabolism
Songkhla, 90110
Turkey (Türkiye)3 sites
Marmara University Medical Faculty
Altunizade, 34662
Istanbul University Cerrahpasa Faculty of Medicine
Istanbul, TUR, 34098
Kocaeli University Medical Faculty
Kocaeli, 41380
Germany2 sites
Universitaetsklinikum Erlangen Nuernberg
Erlangen, 91054
Universitaetsklinikum Muenchen LMU
München, 81377
Japan2 sites
National Hospital Organization Nagoya Medical Center
Nagoya, Aichi-ken, 460-0001
Hyogo College of Medicine Hospital
Nishinomiya, Hyōgo, 663 8501
Poland2 sites
SP ZOZ Szpital Uniwersytecki w Krakowie
Krakow, 31-501
Mazowiecki Szpital Brodnowski
Warsaw, 03 242
South Korea2 sites
Seoul National University Hospital
Seoul, 03080
Yonsei Univ Health System YUCM
Seoul, 03722
Costa Rica1 site
Clinica Los Yoses
San Pedro, San Jose, Costa Rica, 1406 1200
Russia1 site
Center for Endocrinology Russian Academy of Med Sciences
Moscow, 117036
About this study
Official title: 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.
Read the 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
- Being tested
- osilodrostat
- Keywords
- Cushing's syndrome, osilodrostat, LCI699, endogenous Cushing's syndrome
- Medical subject headings
- Cushing Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
osilodrostatDrug
Also called LCI699
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.
| Study group | Role | What they receive |
|---|---|---|
| osilodrostat | Other | open label, with patients receiving same dose as provided in the parent study Receives: Drug: osilodrostat |
What is being measured
The primary outcome is the main question the study is designed to answer.
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))
Measured: up to 5 years
Secondary outcomes (1)
Percentage of Patients With Clinical Benefit
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.
Measured: up to of 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2018
- Study start5 Oct 2018
- Primary completion16 Nov 2023
- Study completion16 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cushing's SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 127 participants (actual)
Sponsor and investigators
- Lead sponsor
- RECORDATI GROUP
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Recordati, Study director, Recordati AG
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03606408
- Sponsor's protocol ID
- CLCI699C2X01B
- Status last verified
- Nov 2024
- First submitted
- 20 Jul 2018
- Last updated
- 18 Dec 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT03606408
- Acronym / sponsor ID
- CLCI699C2X01B
- Conditions
- Cushing's Syndrome
- Keywords
- Cushing's syndrome, osilodrostat, LCI699, endogenous Cushing's syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 127 participants (actual)
- Lead sponsor
- RECORDATI GROUPIndustry (pharma, biotech, devices)
- Study start
- 5 Oct 2018 (actual)
- Primary completion
- 16 Nov 2023 (actual)
- Study completion
- 16 Nov 2023 (actual)
- First posted
- 30 Jul 2018 (actual)
- Results
- Posted 18 Dec 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| osilodrostat | Other | Drug: osilodrostatopen label, with patients receiving same dose as provided in the parent study |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (8 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| osilodrostatAlso: LCI699osilodrostat, 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. | Drug | osilodrostat |
Primary outcome
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))
1 secondary outcome
Percentage of Patients With Clinical Benefit
Time frame: up to of 5 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes
Related trials for Cushing's Syndrome
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Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Recordati study director
Recordati AG
56 sites in 21 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing osilodrostat in people with Cushing's Syndrome, run by RECORDATI GROUP at 56 sites in 21 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 56 sites in United States, Brazil, Canada, China, France and 16 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Dec 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse/Serious Adverse Events (time frame: up to 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Dec 2024.