Compassionate Use of CORLUX® (Mifepristone) in the Treatment of Signs and Symptoms of Endogenous Cushing's Syndrome

• ClinicalTrials.gov Identifier: NCT01371565
• Recruitment Status: Completed
• First Posted: June 13, 2011
• Results First Posted: November 20, 2013
• Last Update Posted: March 18, 2014
• Sponsor: Corcept Therapeutics
• Information provided by (Responsible Party): Corcept Therapeutics

Study Description

Brief Summary:

This is a compassionate use study. In addition to providing compassionate use access to mifepristone, objectives of the study will be to evaluate the safety and utility of mifepristone in the treatment of the signs and symptoms of endogenous Cushing's syndrome when given on a compassionate use basis. The study will only enroll subjects whose physicians have determined that medical treatment is needed to control the symptoms or signs of hypercortisolemia.

Condition or disease	Intervention/treatment	Phase
Cushing's Disease; Cushing's Syndrome	Drug: Mifepristone	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 4 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Compassionate Use Protocol for the Administration of CORLUX® (Mifepristone) in the Treatment of the Signs and Symptoms of Endogenous Cushing's Syndrome
• Study Start Date: November 2010
• Actual Primary Completion Date: June 2012
• Actual Study Completion Date: September 2012

Arms and Interventions

Arm	Intervention/treatment
Experimental: mifepristone	Drug: Mifepristone; mifepristone at doses from 300mg/day up to 1200mg/day; Other Name: CORLUX®

Outcome Measures

Primary Outcome Measures :

1. Number of Participants With Adverse Events [ Time Frame: 6 months ]
   Safety was assessed at all visits and adverse events were recorded.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

1. Have a confirmed diagnosis of endogenous hypercortisolemia caused by ACTH dependent or ACTH independent etiologies including:

   • Cushing's Disease that (more than one may apply)

     • has recurred after primary pituitary surgery
     • has persisted despite pituitary surgery (failed pituitary surgery)
     • has been treated with radiation therapy to the pituitary
     • is not treatable with surgery
     • exists in subjects who are not candidates for or who refuse surgery
   • Ectopic ACTH
   • Ectopic CRF secretion
   • Adrenal adenoma
   • Adrenal carcinoma
   • Adrenal autonomy
2. Have documented biochemical evidence of endogenous hypercortisolemia which includes elevated urinary free cortisol.
3. Require medical treatment of hypercortisolemia.

Exclusion Criteria:

Individuals not eligible to be enrolled into the study are those who:

• Have de novo Cushing's disease and are surgical candidates for pituitary surgery.
• Have an acute or unstable medical problem, which could be aggravated by mifepristone treatment.
• Taking medications within 14 days of the baseline visit (Day 1) that a) have a large first pass metabolism largely mediated by CYP3A4 and a narrow therapeutic margin and/or b) are strong CYP3A4 inhibitors.
• Female patients of reproductive potential, who are pregnant or who are unable or unwilling to use medically acceptable, non-hormonal methods of contraception during the study.
• Have received investigational treatment (drug, biological agent or device) within 30 days of Screening
• Have a history of an allergic reaction or intolerance to CORLUX (mifepristone)
• Have a non-endogenous source of hypercortisolemia such as factious hypercortisolemia (exogenous source of glucocorticoid, iatrogenic Cushing's syndrome), factious or therapeutic use of ACTH
• Have Pseudo-Cushing's syndrome.
• Postmenopausal women with an intact uterus who have experienced unexplained vaginal bleeding within 12 months of Screening are excluded.

Contacts and Locations

Locations

United States, Florida

The Center for Diabetes and Endocrine Care

Hollywood, Florida, United States, 33021

United States, Maryland

Sinai Hospital of Baltimore

Baltimore, Maryland, United States, 21215

United States, Michigan

University of Michigan Medical Center

Ann Arbor, Michigan, United States, 48109

United States, Ohio

Cleveland Clinic Foundation

Cleveland, Ohio, United States, 44195

The Ohio State University, Division of Endocrinology Diabetes and Metabolism

Columbus, Ohio, United States, 43210

Sponsors and Collaborators

Corcept Therapeutics

Investigators

• Study Director: Coleman Gross, M.D.; Corcept Therapeutics

More Information

• Responsible Party: Corcept Therapeutics
• ClinicalTrials.gov Identifier: NCT01371565
• Other Study ID Numbers: C1073-405
• First Posted: June 13, 2011
• Results First Posted: November 20, 2013
• Last Update Posted: March 18, 2014
• Last Verified: February 2014

Keywords provided by Corcept Therapeutics:

Cushing's Disease; Cushing's Syndrome; Cushings; Pituitary; Ectopic ACTH secretion

Additional relevant MeSH terms:

ACTH-Secreting Pituitary Adenoma; Pituitary ACTH Hypersecretion; Cushing Syndrome; Syndrome; Disease; Pathologic Processes; Adrenocortical Hyperfunction; Adrenal Gland Diseases; Endocrine System Diseases; Hyperpituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Adenoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Pituitary Neoplasms; Endocrine Gland Neoplasms; Neoplasms by Site; Mifepristone; Abortifacient Agents, Steroidal; Abortifacient Agents; Reproductive Control Agents; Physiological Effects of Drugs; Contraceptives, Oral, Synthetic; Contraceptives, Oral; Contraceptive Agents, Female