Open-label Treatment in Cushing's Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 22 May 2023
• Main ID: NCT03621280
• Date of registration: 18/05/2018
• Prospective Registration: Yes
• Primary sponsor: Cortendo AB
• Public title: Open-label Treatment in Cushing's Syndrome OPTICS
• Scientific title: An Open-label Extension Study of Levoketoconazole (2S,4R-ketoconazole) in the Treatment of Endogenous Cushing's Syndrome
• Date of first enrolment: January 7, 2019
• Target sample size: 51
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03621280
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Bulgaria, Denmark, France, Greece, Hungary, Israel, Italy, Netherlands, Poland, Romania, Spain, Turkey, United States

Contacts

• Name: Fredric Cohen, MD
• Affiliation: Cortendo AB

Key inclusion & exclusion 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: Subjects 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 subjects
may require re-establishment of the Therapeutic Dose via titration. All subjects 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:

1. 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.

2. Scheduled for surgery for treatment of CS or received surgery for treatment of CS
within the 6 weeks prior to Screening.

3. Treated with mitotane within 6 months prior to enrollment.

4. 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).



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cushing Disease

Cushing Syndrome

Intervention(s)

Drug: Levoketoconazole

Primary Outcome(s)

Changes from Baseline in 24-h UFC [Time Frame: From Baseline to final study visit or up to a maximum of 3 years, whichever comes first.]

Changes from Baseline in AEs/SAEs/AESI as assessed by CTCAE V4.03 [Time Frame: From Baseline to final study visit or up to a maximum of 3 years, whichever comes first.]

Changes from Baseline in Late Night Salivary Cortisol [Time Frame: From Baseline to final study visit or up to a maximum of 3 years, whichever comes first.]

Secondary ID(s)

COR-2017-OLE

Source(s) of Monetary Support

Please refer to primary and secondary sponsors