Open-label Treatment in Cushing's Syndrome

NCT03621280 | Completed Phase 3 Results FDA Drug

• 1 other identifier: COR-2017-OLE
• Study Type: interventional
• Target: 51
• Locations: 11 countries
• Sites: 30

Brief Summary

This is a long-term, open-label extension study of levoketoconazole in participants with endogenous Cushing's Syndrome.

Conditions

Cushing Syndrome; Cushing Disease

Outcome Measures

Primary Outcomes (1)

• 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.

  From parent study Baseline to final study visit or up to a maximum of 3 years, whichever comes first.

Study Arms (1)

Levoketoconazole

EXPERIMENTAL

Levoketoconazole taken twice daily up to 1200 mg daily

Drug: Levoketoconazole

Interventions

Levoketoconazole DRUG

Levoketoconazole up to 1200 mg daily

Also known as: COR-003

Levoketoconazole

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;
• 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.)

You may not qualify if:

• 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).

Sponsors & Collaborators

• Cortendo AB: lead

Study Sites (30)

The Center for Diabetes and Endocrine Care

Fort Lauderdale, Florida, 33312, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

University of New Mexico HSC - HSC Sponsored Projects Office

Albuquerque, New Mexico, 87131, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Sveti Georgy University Hospital Clinic of Endocrinology and Metabolic Diseases

Plovdiv, 4000, Bulgaria

Location

Alexandovska University Hospital

Sofia, 1431, Bulgaria

Location

University Specialized Hospital for Active Treatment in Endocrinology

Sofia, 1431, Bulgaria

Location

APHM Hôpital de la Conception

Marseille, 13385, France

Location

General Hospital of Athens Evangelismos Department of Endocrinology and Diabete

Athens, 10676, Greece

Location

Hippokration General Hospital Endocrinology and Diabetes Department

Thessaloniki, 54642, Greece

Location

Semmelweis Egyetem II. Belgyógyászati Klinika

Budapest, 1083, Hungary

Location

Bnai Zion Medical Center Endocrinology Institute

Haifa, 34802, Israel

Location

Rabin Medical Center, Beilinson Campus

Petah Tikva, 49100, Israel

Location

Tel Aviv-Sourasky Medical Center Institute Endocrinology Metabolism & Hypertension

Tel Aviv, 6423906, Israel

Location

Clinica Endocrinologia malattie del Metabolismo

Ancona, 60126, Italy

Location

AOU Policlinico G. Martino Sezione di Endocrinologia

Messina, 98125, Italy

Location

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

Naples, 80131, Italy

Location

Policlinico Universitario Sant'Andrea

Roma, 00189, Italy

Location

University of Turin

Turin, 10126, Italy

Location

Erasmus Medical Center

Rotterdam, 3015 CE, Netherlands

Location

Instytut Centrum Zdrowia Matki Polki

Lodz, 93-338, Poland

Location

Institutul National de Endocrinologie C.I. Parhon

Cluj-Napoca, 400349, Romania

Location

Spitalul Clinic Judetean de Urgenta Cluj-Napoca

Cluj-Napoca, 400349, Romania

Location

Spitalul Clinic Judetean Mures

Târgu Mureş, 540139, Romania

Location

Hospital Universidad De La Ribera

Alzira, 46600, Spain

Location

Hospital Universitario Ramón y Cajal

Madrid, 28034, Spain

Location

MeSH Terms

Conditions

Cushing Syndrome; Pituitary ACTH Hypersecretion

Condition Hierarchy (Ancestors)

Adrenocortical Hyperfunction; Adrenal Gland Diseases; Endocrine System Diseases; Hyperpituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Results Point of Contact

• Title: Valentina Conoscenti, MD
• Organization: Cortendo AB

OPTICS@cortendo.com

1-877-937-4737

Study Officials

• Fredric Cohen, MD

  Cortendo AB

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open-Label Extension Study

Study Record Dates

• First Submitted: May 18, 2018
• First Posted: August 8, 2018
• Study Start: January 7, 2019
• Primary Completion: December 31, 2022
• Study Completion: December 31, 2022
• Last Updated: June 19, 2025
• Results First Posted: June 19, 2025

Record last verified: 2025-06

Locations

FR (1); US (8); BU (3); HU (1); ES (2); RO (3); IL (3); IT (5); GR (2); NL (1); PL (1)