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Open-label Treatment in Cushing's Syndrome (OPTICS)

Primary Purpose

Cushing Syndrome, Cushing Disease

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Levoketoconazole
Sponsored by
Cortendo AB
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cushing Syndrome

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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).

Sites / Locations

  • The Center for Diabetes and Endocrine Care
  • Emory University
  • Washington University School of Medicine
  • University of New Mexico HSC - HSC Sponsored Projects Office
  • Columbia University Medical Center
  • Memorial Sloan Kettering Cancer Center
  • Cleveland Clinic
  • Oregon Health & Science University
  • Sveti Georgy University Hospital Clinic of Endocrinology and Metabolic Diseases
  • Alexandovska University Hospital
  • University Specialized Hospital for Active Treatment in Endocrinology
  • APHM Hôpital de la Conception
  • General Hospital of Athens Evangelismos Department of Endocrinology and Diabete
  • Hippokration General Hospital Endocrinology and Diabetes Department
  • Semmelweis Egyetem II. Belgyógyászati Klinika
  • Bnai Zion Medical Center Endocrinology Institute
  • Rabin Medical Center, Beilinson Campus
  • Tel Aviv-Sourasky Medical Center Institute Endocrinology Metabolism & Hypertension
  • Clinica Endocrinologia malattie del Metabolismo
  • AOU Policlinico G. Martino Sezione di Endocrinologia
  • Via Sergio Pansini 5 Uni Naples FedericoII Dept of Molecular&Clinical Endocrinology&Oncology
  • Policlinico Universitario Sant'Andrea
  • University of Turin
  • Erasmus Medical Center
  • Instytut Centrum Zdrowia Matki Polki
  • Institutul National de Endocrinologie C.I. Parhon
  • Spitalul Clinic Judetean de Urgenta Cluj-Napoca
  • Spitalul Clinic Judetean Mures
  • Hospital Universidad De La Ribera
  • Hospital Universitario Ramón y Cajal

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Levoketoconazole

Arm Description

Levoketoconazole taken twice daily up to 1200 mg daily

Outcomes

Primary Outcome Measures

Changes from Baseline in 24-h UFC
Changes or shifts from baseline in mUFC
Changes from Baseline in Late Night Salivary Cortisol
Changes or shifts from baseline in late night salivary cortisol levels
Changes from Baseline in AEs/SAEs/AESI as assessed by CTCAE V4.03
Changes from Baseline in the incidence and severity of AEs, SAEs, AESIs

Secondary Outcome Measures

Full Information

First Posted
May 18, 2018
Last Updated
May 9, 2023
Sponsor
Cortendo AB
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1. Study Identification

Unique Protocol Identification Number
NCT03621280
Brief Title
Open-label Treatment in Cushing's Syndrome
Acronym
OPTICS
Official Title
An Open-label Extension Study of Levoketoconazole (2S,4R-ketoconazole) in the Treatment of Endogenous Cushing's Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Completed
Study Start Date
January 7, 2019 (Actual)
Primary Completion Date
December 31, 2022 (Actual)
Study Completion Date
December 31, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cortendo AB

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
This is a long-term, open-label extension study of levoketoconazole in subjects with endogenous Cushing's Syndrome.
Detailed Description
This is a long-term, OLE study of levoketoconazole in subjects with endogenous CS who have completed one or both parent studies or otherwise potentially qualify for this study, as defined in the eligibility criteria.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cushing Syndrome, Cushing Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
Open-Label Extension Study
Masking
None (Open Label)
Allocation
N/A
Enrollment
51 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Levoketoconazole
Arm Type
Experimental
Arm Description
Levoketoconazole taken twice daily up to 1200 mg daily
Intervention Type
Drug
Intervention Name(s)
Levoketoconazole
Other Intervention Name(s)
COR-003
Intervention Description
Levoketoconazole up to 1200 mg daily
Primary Outcome Measure Information:
Title
Changes from Baseline in 24-h UFC
Description
Changes or shifts from baseline in mUFC
Time Frame
From Baseline to final study visit or up to a maximum of 3 years, whichever comes first.
Title
Changes from Baseline in Late Night Salivary Cortisol
Description
Changes or shifts from baseline in late night salivary cortisol levels
Time Frame
From Baseline to final study visit or up to a maximum of 3 years, whichever comes first.
Title
Changes from Baseline in AEs/SAEs/AESI as assessed by CTCAE V4.03
Description
Changes from Baseline in the incidence and severity of AEs, SAEs, AESIs
Time Frame
From Baseline to final study visit or up to a maximum of 3 years, whichever comes first.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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: 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. 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.) 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).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Fredric Cohen, MD
Organizational Affiliation
Cortendo AB
Official's Role
Study Chair
Facility Information:
Facility Name
The Center for Diabetes and Endocrine Care
City
Fort Lauderdale
State/Province
Florida
ZIP/Postal Code
33312
Country
United States
Facility Name
Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
University of New Mexico HSC - HSC Sponsored Projects Office
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87131
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Memorial Sloan Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Cleveland Clinic
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Sveti Georgy University Hospital Clinic of Endocrinology and Metabolic Diseases
City
Plovdiv
ZIP/Postal Code
4000
Country
Bulgaria
Facility Name
Alexandovska University Hospital
City
Sofia
ZIP/Postal Code
1431
Country
Bulgaria
Facility Name
University Specialized Hospital for Active Treatment in Endocrinology
City
Sofia
ZIP/Postal Code
1431
Country
Bulgaria
Facility Name
APHM Hôpital de la Conception
City
Marseille
ZIP/Postal Code
13385
Country
France
Facility Name
General Hospital of Athens Evangelismos Department of Endocrinology and Diabete
City
Athens
ZIP/Postal Code
10676
Country
Greece
Facility Name
Hippokration General Hospital Endocrinology and Diabetes Department
City
Thessaloníki
ZIP/Postal Code
54642
Country
Greece
Facility Name
Semmelweis Egyetem II. Belgyógyászati Klinika
City
Budapest
ZIP/Postal Code
1083
Country
Hungary
Facility Name
Bnai Zion Medical Center Endocrinology Institute
City
Haifa
ZIP/Postal Code
34802
Country
Israel
Facility Name
Rabin Medical Center, Beilinson Campus
City
Petah tikva
ZIP/Postal Code
49100
Country
Israel
Facility Name
Tel Aviv-Sourasky Medical Center Institute Endocrinology Metabolism & Hypertension
City
Tel Aviv
ZIP/Postal Code
6423906
Country
Israel
Facility Name
Clinica Endocrinologia malattie del Metabolismo
City
Ancona
ZIP/Postal Code
60126
Country
Italy
Facility Name
AOU Policlinico G. Martino Sezione di Endocrinologia
City
Messina
ZIP/Postal Code
98125
Country
Italy
Facility Name
Via Sergio Pansini 5 Uni Naples FedericoII Dept of Molecular&Clinical Endocrinology&Oncology
City
Naples
ZIP/Postal Code
80131
Country
Italy
Facility Name
Policlinico Universitario Sant'Andrea
City
Roma
ZIP/Postal Code
00189
Country
Italy
Facility Name
University of Turin
City
Turin
ZIP/Postal Code
10126
Country
Italy
Facility Name
Erasmus Medical Center
City
Rotterdam
ZIP/Postal Code
3015 CE
Country
Netherlands
Facility Name
Instytut Centrum Zdrowia Matki Polki
City
Łódź
ZIP/Postal Code
93-338
Country
Poland
Facility Name
Institutul National de Endocrinologie C.I. Parhon
City
Cluj-Napoca
ZIP/Postal Code
400349
Country
Romania
Facility Name
Spitalul Clinic Judetean de Urgenta Cluj-Napoca
City
Cluj-Napoca
ZIP/Postal Code
400349
Country
Romania
Facility Name
Spitalul Clinic Judetean Mures
City
Târgu-Mureş
ZIP/Postal Code
540139
Country
Romania
Facility Name
Hospital Universidad De La Ribera
City
Alzira
ZIP/Postal Code
46600
Country
Spain
Facility Name
Hospital Universitario Ramón y Cajal
City
Madrid
ZIP/Postal Code
28034
Country
Spain

12. IPD Sharing Statement

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Open-label Treatment in Cushing's Syndrome

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