Open-label Treatment in Cushing's Syndrome

NCT ID: NCT03621280

Last Updated: 2025-06-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 51 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-01-07
• Study Completion Date: 2022-12-31

Brief Summary

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.

Conditions

Cushing Syndrome; Cushing Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Levoketoconazole

Levoketoconazole taken twice daily up to 1200 mg daily

Group Type: EXPERIMENTAL

Levoketoconazole

Intervention Type: DRUG

Levoketoconazole up to 1200 mg daily

Interventions

Levoketoconazole

Levoketoconazole up to 1200 mg daily

Intervention Type: DRUG

Other Intervention Names

COR-003

Eligibility Criteria

Inclusion Criteria

1. Completed the Extended Evaluation Phase of Study COR-2012-01 (i.e. M12)

2. 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;
• 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.

3. Currently in a named patient program or other Expanded Access Program receiving levoketoconazole

4. Were levoketoconazole-naïve prior to entry and received early rescue therapy with open-label levoketoconazole in Study COR-2017-01.

5. 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.

6. 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.)

Exclusion Criteria

• 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).

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cortendo AB

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Fredric Cohen, MD

Role: STUDY_CHAIR

Cortendo AB

Locations

The Center for Diabetes and Endocrine Care

Fort Lauderdale, Florida, United States

Emory University

Atlanta, Georgia, United States

Washington University School of Medicine

St Louis, Missouri, United States

University of New Mexico HSC - HSC Sponsored Projects Office

Albuquerque, New Mexico, United States

Columbia University Medical Center

New York, New York, United States

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Cleveland Clinic

Cleveland, Ohio, United States

Oregon Health & Science University

Portland, Oregon, United States

Sveti Georgy University Hospital Clinic of Endocrinology and Metabolic Diseases

Plovdiv, , Bulgaria

Alexandovska University Hospital

Sofia, , Bulgaria

University Specialized Hospital for Active Treatment in Endocrinology

Sofia, , Bulgaria

APHM Hôpital de la Conception

Marseille, , France

General Hospital of Athens Evangelismos Department of Endocrinology and Diabete

Athens, , Greece

Hippokration General Hospital Endocrinology and Diabetes Department

Thessaloniki, , Greece

Semmelweis Egyetem II. Belgyógyászati Klinika

Budapest, , Hungary

Bnai Zion Medical Center Endocrinology Institute

Haifa, , Israel

Rabin Medical Center, Beilinson Campus

Petah Tikva, , Israel

Tel Aviv-Sourasky Medical Center Institute Endocrinology Metabolism & Hypertension

Tel Aviv, , Israel

Clinica Endocrinologia malattie del Metabolismo

Ancona, , Italy

AOU Policlinico G. Martino Sezione di Endocrinologia

Messina, , Italy

Via Sergio Pansini 5 Uni Naples FedericoII Dept of Molecular&Clinical Endocrinology&Oncology

Naples, , Italy

Policlinico Universitario Sant'Andrea

Roma, , Italy

University of Turin

Turin, , Italy

Erasmus Medical Center

Rotterdam, , Netherlands

Instytut Centrum Zdrowia Matki Polki

Lodz, , Poland

Institutul National de Endocrinologie C.I. Parhon

Cluj-Napoca, , Romania

Spitalul Clinic Judetean de Urgenta Cluj-Napoca

Cluj-Napoca, , Romania

Spitalul Clinic Judetean Mures

Târgu Mureş, , Romania

Hospital Universidad De La Ribera

Alzira, , Spain

Hospital Universitario Ramón y Cajal

Madrid, , Spain

Countries

United States; Bulgaria; France; Greece; Hungary; Israel; Italy; Netherlands; Poland; Romania; Spain

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

COR-2017-OLE

Identifier Type: -

Identifier Source: org_study_id