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.

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.