Cushing's Disease, Cushing's Syndrome
Conditions
Keywords
Cushing's Disease, Cushing's Syndrome, Cushings, Pituitary, Ectopic ACTH secretion
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.
Interventions
DRUGMifepristone
mifepristone at doses from 300mg/day up to 1200mg/day
Sponsors
Corcept Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events | 6 months | Safety was assessed at all visits and adverse events were recorded. |
Countries
United States
Participant flow
Recruitment details
Eligible patients were men or non-pregnant women 18 yrs or older requiring medical treatment for symptoms of endogenous Cushing's syndrome due to ectopic ACTH, adrenal tumors/adrenal hyperplasia, or Cushing's disease if not candidates for pituitary surgery. Pts w/de novo Cushing's disease who were candidates for surgery were not enrolled.
Pre-assignment details
All patients received active drug (no placebo).
Participants by arm
| Arm | Count |
|---|---|
| Mifepristone At doses from 300mg/day up to 1200mg/day | 4 |
| Total | 4 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Death | 1 |
| Overall Study | Resection of neuroendocrine tumor | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Mifepristone |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 1 Participants |
| Age, Categorical Between 18 and 65 years | 3 Participants |
| Age, Continuous | 44.8 years STANDARD_DEVIATION 17.9 |
| Region of Enrollment United States | 4 participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 4 / 4 |
| serious Total, serious adverse events | 1 / 4 |
Outcome results
Primary
Number of Participants With Adverse Events
Safety was assessed at all visits and adverse events were recorded.
Time frame: 6 months
Population: Due to the small number of patients enrolled (N=4), no formal assessments were planned. All patients who received study drug were included in the safety review.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Mifepristone | Number of Participants With Adverse Events | 4 participants |