Cushing SyndromePhase 3OPTICSNCT03621280
Open-label Treatment in Cushing's Syndrome
A Phase 3 study testing Levoketoconazole in people with Cushing Syndrome and Cushing Disease, run by Cortendo AB at 30 sites in 11 countries.
Trial summary
This is a long-term, open-label extension study of levoketoconazole in participants with endogenous Cushing's Syndrome. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cushing Syndrome, Cushing Disease
- What is being tested?
- Levoketoconazole
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 51 participants
- Where
- 30 sitesin 11 countries
- Study dates
- 7 Jan 2019to 31 Dec 2022
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Completed the Extended Evaluation Phase of Study COR-2012-01 (i.e. M12)
- Completed the Restoration Phase of Study COR-2017-01 (i.e. RES2)
- NOTE: Participants meeting criteria 1 or 2 above who have had a break in therapy may be eligible only after discussion with the Medical Monitor. If eligible, such participants may require re-establishment of the Therapeutic Dose via titration. All participants who have had a break in therapy should be discussed with the Medical Monitor to determine the starting dose of levoketoconazole. Prior to resuming treatment with levoketoconazole, other therapies for Cushing's Syndrome must undergo an appropriate washout period, with minimum durations as follows:
- Ketoconazole or metyrapone: 2 weeks;
- Dopamine agonists: bromocriptine (2 weeks), cabergoline (8 weeks);
- Octreotide acetate LAR, lanreotide Autogel, pasireotide LAR: 12 weeks;
Show all 14Show fewer
- Lanreotide SR: 8 weeks;
- Octreotide acetate (immediate release) or short-acting pasireotide: 1 week;
- Mifepristone (RU 486, KORLYM): 4 weeks;
- Megestrol acetate or medroxyprogesterone acetate (and selected other synthetic progestins): 6 weeks.
- Currently in a named patient program or other Expanded Access Program receiving levoketoconazole
- Were levoketoconazole-naïve prior to entry and received early rescue therapy with open-label levoketoconazole in Study COR-2017-01.
- Achieved a clinically meaningful partial response (with reduction of UFC) in Study COR-2017-01 at dose level 7 or at a maximally tolerated dose of levoketoconazole but did not meet the randomization criteria for Study COR-2017-01 at the end of the Dose Titration and Maintenance Phase when randomization was open.
- Were levoketoconazole-naïve prior to entry and were enrolled in Study COR-2017-01 in the Dose Titration and Maintenance Phase when randomization was closed. (NOTE: Such subjects must receive at least 1 dose of levoketoconazole before transitioning to this study.)
Who cannot join
- Discontinued levoketoconazole while participating in Study COR-2012-01 or Study COR-2017-01 or a named patient program or other Expanded Access program, due to safety or tolerability concerns or lack of efficacy.
- Scheduled for surgery for treatment of CS or received surgery for treatment of CS within the 6 weeks prior to Screening.
- Treated with mitotane within 6 months prior to enrollment.
- History of malignancy, including adrenal or pituitary carcinomas (other than low-risk, well-differentiated carcinomas of thyroid, breast or prostate that are very unlikely to require further treatment in the opinion of the treating physician, or squamous cell or basal cell carcinoma of the skin).
Trial locations and contacts
30 sites in 11 countries.
30 sites in 11 countries
United States8 sites
University of New Mexico HSC - HSC Sponsored Projects Office
Albuquerque, New Mexico, 87131
Emory University
Atlanta, Georgia, 30322
Cleveland Clinic
Cleveland, Ohio, 44195
The Center for Diabetes and Endocrine Care
Fort Lauderdale, Florida, 33312
Columbia University Medical Center
New York, New York, 10032
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Oregon Health & Science University
Portland, Oregon, 97239
Washington University School of Medicine
St Louis, Missouri, 63110
Italy5 sites
Clinica Endocrinologia malattie del Metabolismo
Ancona, 60126
AOU Policlinico G. Martino Sezione di Endocrinologia
Messina, 98125
Via Sergio Pansini 5 Uni Naples FedericoII Dept of Molecular&Clinical Endocrinology&Oncology
Naples, 80131
Policlinico Universitario Sant'Andrea
Roma, 00189
University of Turin
Turin, 10126
Bulgaria3 sites
Sveti Georgy University Hospital Clinic of Endocrinology and Metabolic Diseases
Plovdiv, 4000
Alexandovska University Hospital
Sofia, 1431
University Specialized Hospital for Active Treatment in Endocrinology
Sofia, 1431
Israel3 sites
Bnai Zion Medical Center Endocrinology Institute
Haifa, 34802
Rabin Medical Center, Beilinson Campus
Petah Tikva, 49100
Tel Aviv-Sourasky Medical Center Institute Endocrinology Metabolism & Hypertension
Tel Aviv, 6423906
Romania3 sites
Institutul National de Endocrinologie C.I. Parhon
Cluj-Napoca, 400349
Spitalul Clinic Judetean de Urgenta Cluj-Napoca
Cluj-Napoca, 400349
Spitalul Clinic Judetean Mures
Târgu Mureş, 540139
Greece2 sites
General Hospital of Athens Evangelismos Department of Endocrinology and Diabete
Athens, 10676
Hippokration General Hospital Endocrinology and Diabetes Department
Thessaloniki, 54642
Spain2 sites
Hospital Universidad De La Ribera
Alzira, 46600
Hospital Universitario Ramón y Cajal
Madrid, 28034
About this study
Official title: An Open-label Extension Study of Levoketoconazole (2S,4R-ketoconazole) in the Treatment of Endogenous Cushing's Syndrome
This is a long-term, open-label extension study of levoketoconazole in participants with endogenous Cushing's Syndrome.
Read the detailed description
This is a long-term, open-label extension (OLE) study of levoketoconazole in participants with endogenous Cushing's Syndrome (CS) who have completed one or both parent studies or otherwise potentially qualify for this study, as defined in the eligibility criteria.
- Conditions
- Cushing Syndrome, Cushing Disease
- Being tested
- Levoketoconazole
- Medical subject headings
- Cushing Syndrome, Pituitary ACTH Hypersecretion
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LevoketoconazoleDrug
Also called COR-003
Levoketoconazole up to 1200 mg daily
| Study group | Role | What they receive |
|---|---|---|
| Levoketoconazole | Experimental | Levoketoconazole taken twice daily up to 1200 mg daily Receives: Drug: Levoketoconazole |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportions of Participants With Mean Urinary Free Cortisol (mUFC) Categorization Based on Upper Limit of Normal (ULN)
Proportions of participants with mUFC: 1) Less or equal to the ULN of the reference range, 2) Above the ULN to 1.5x the ULN, and 3) Above 1.5x the ULN.
Measured: From parent study Baseline to final study visit or up to a maximum of 3 years, whichever comes first.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2018
- Study start7 Jan 2019
- Primary completion31 Dec 2022
- Study completion31 Dec 2022
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 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-Label Extension Study
- Masking
- None (open label)
- Enrolment
- 51 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cortendo AB
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Fredric Cohen, MD, Study chair, Cortendo AB
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03621280
- Sponsor's protocol ID
- COR-2017-OLE
- Acronym
- OPTICS
- Status last verified
- Jun 2025
- First submitted
- 18 May 2018
- Last updated
- 19 Jun 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Extension Study of Levoketoconazole (2S,4R-ketoconazole) in the Treatment of Endogenous Cushing's Syndrome
- ClinicalTrials.gov ID
- NCT03621280
- Acronym / sponsor ID
- OPTICS / COR-2017-OLE
- Conditions
- Cushing Syndrome, Cushing Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 51 participants (actual)
- Lead sponsor
- Cortendo ABIndustry (pharma, biotech, devices)
- Study start
- 7 Jan 2019 (actual)
- Primary completion
- 31 Dec 2022 (actual)
- Study completion
- 31 Dec 2022 (actual)
- First posted
- 8 Aug 2018 (actual)
- Results
- Posted 19 Jun 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a long-term, open-label extension study of levoketoconazole in participants with endogenous Cushing's Syndrome.
Detailed description
This is a long-term, open-label extension (OLE) study of levoketoconazole in participants with endogenous Cushing's Syndrome (CS) who have completed one or both parent studies or otherwise potentially qualify for this study, as defined in the eligibility criteria.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-Label Extension Study
| Arm | Type | Receives |
|---|---|---|
| Levoketoconazole | Experimental | Drug: LevoketoconazoleLevoketoconazole taken twice daily up to 1200 mg daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Completed the Extended Evaluation Phase of Study COR-2012-01 (i.e. M12)
- Completed the Restoration Phase of Study COR-2017-01 (i.e. RES2)
- NOTE: Participants meeting criteria 1 or 2 above who have had a break in therapy may be eligible only after discussion with the Medical Monitor. If eligible, such participants may require re-establishment of the Therapeutic Dose via titration. All participants who have had a break in therapy should be discussed with the Medical Monitor to determine the starting dose of levoketoconazole. Prior to resuming treatment with levoketoconazole, other therapies for Cushing's Syndrome must undergo an appropriate washout period, with minimum durations as follows:
- Currently in a named patient program or other Expanded Access Program receiving levoketoconazole
- Were levoketoconazole-naïve prior to entry and received early rescue therapy with open-label levoketoconazole in Study COR-2017-01.
Key exclusion criteria (4 listed)
- Discontinued levoketoconazole while participating in Study COR-2012-01 or Study COR-2017-01 or a named patient program or other Expanded Access program, due to safety or tolerability concerns or lack of efficacy.
- Scheduled for surgery for treatment of CS or received surgery for treatment of CS within the 6 weeks prior to Screening.
- Treated with mitotane within 6 months prior to enrollment.
- History of malignancy, including adrenal or pituitary carcinomas (other than low-risk, well-differentiated carcinomas of thyroid, breast or prostate that are very unlikely to require further treatment in the opinion of the treating physician, or squamous cell or basal cell carcinoma of the skin).
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 |
|---|---|---|
| LevoketoconazoleAlso: COR-003Levoketoconazole up to 1200 mg daily | Drug | Levoketoconazole |
Primary outcome
Proportions of Participants With Mean Urinary Free Cortisol (mUFC) Categorization Based on Upper Limit of Normal (ULN)
Time frame: From parent study Baseline to final study visit or up to a maximum of 3 years, whichever comes first.
Proportions of participants with mUFC: 1) Less or equal to the ULN of the reference range, 2) Above the ULN to 1.5x the ULN, and 3) Above 1.5x the ULN.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jun 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Fredric Cohen, MD study chair
Cortendo AB
30 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Levoketoconazole in people with Cushing Syndrome and Cushing Disease, run by Cortendo AB at 30 sites in 11 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 14 inclusion and 4 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 19 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in United States, Italy, Bulgaria, Israel, Romania and 6 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 19 Jun 2025). 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?
Proportions of Participants With Mean Urinary Free Cortisol (mUFC) Categorization Based on Upper Limit of Normal (ULN) (time frame: From parent study Baseline to final study visit or up to a maximum of 3 years, whichever comes first.). 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 19 Jun 2025.