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

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

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.

Study Overview

• Status: Completed
• Conditions: Cushing's Syndrome; Cushing's Disease
• Intervention / Treatment: Drug: Mifepristone

• Study Type: Interventional
• Enrollment (Actual): 4
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Florida
    • Hollywood, Florida, United States, 33021
      • The Center for Diabetes and Endocrine Care
  • Maryland
    • Baltimore, Maryland, United States, 21215
      • Sinai Hospital of Baltimore
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Medical Center
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic Foundation
    • Columbus, Ohio, United States, 43210
      • The Ohio State University, Division of Endocrinology Diabetes and Metabolism

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: mifepristone	Drug: Mifepristone; mifepristone at doses from 300mg/day up to 1200mg/day; Other Names: CORLUX®

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Adverse Events	Safety was assessed at all visits and adverse events were recorded.	6 months

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start: November 1, 2010
• Primary Completion (ACTUAL): June 1, 2012
• Study Completion (ACTUAL): September 1, 2012

Study Registration Dates

• First Submitted: June 7, 2011
• First Submitted That Met QC Criteria: June 9, 2011
• First Posted (ESTIMATE): June 13, 2011

Study Record Updates

• Last Update Posted (ESTIMATE): March 18, 2014
• Last Update Submitted That Met QC Criteria: February 19, 2014
• Last Verified: February 1, 2014

More Information

Terms related to this study

• Keywords: Pituitary; Cushing's Syndrome; Cushing's Disease; Cushings; Ectopic ACTH secretion
• Additional Relevant MeSH Terms: Pathologic Processes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Neoplasms, Glandular and Epithelial; Endocrine System Diseases; Disease; Endocrine Gland Neoplasms; Hypothalamic Diseases; Hyperpituitarism; Pituitary Diseases; Adenoma; Adrenocortical Hyperfunction; Adrenal Gland Diseases; Pituitary Neoplasms; Syndrome; Cushing Syndrome; ACTH-Secreting Pituitary Adenoma; Pituitary ACTH Hypersecretion; Physiological Effects of Drugs; Hormones, Hormone Substitutes, and Hormone Antagonists; Hormone Antagonists; Contraceptive Agents, Hormonal; Contraceptive Agents; Reproductive Control Agents; Contraceptives, Oral; Contraceptive Agents, Female; Contraceptives, Oral, Synthetic; Abortifacient Agents; Luteolytic Agents; Abortifacient Agents, Steroidal; Contraceptives, Postcoital, Synthetic; Contraceptives, Postcoital; Menstruation-Inducing Agents; Mifepristone
• Other Study ID Numbers: C1073-405