Compassionate Use of CORLUX® (Mifepristone) in the Treatment of Signs and Symptoms of Endogenous Cushing's Syndrome
This study has been completed.
Information provided by (Responsible Party):
First received: June 7, 2011
Last updated: September 19, 2013
Last verified: September 2013
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.
||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
||Compassionate Use Protocol for the Administration of CORLUX® (Mifepristone) in the Treatment of the Signs and Symptoms of Endogenous Cushing's Syndrome
Primary Outcome Measures:
- The Long-term Safety of Mifepristone in the Treatment of the Signs and Symptoms of Endogenous Cushing's Syndrome. [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Safety was assessed at all visits, based on physical examination, vital signs, adverse events, and laboratory tests. Women with an intact uterus were examined at Screening, Week 12, Week 24 and 6 Week Follow-up for changes in endometrial thickness. Cushing's disease patients were monitored by pituitary MRI at Screening, Week 10 and Week 24.
Due to the small number of enrolled patients, a formal assessment of study efficacy was not planned. Clinical efficacy was examined using various approaches, including changes in HbA1c, fasting glucose and reduction in anti-diabetic medications and change in blood pressure and reduction in anti-hypertensive medications. Additional measures of clinical utility reviewed were changes in weight and BMI, quality of life (SF36 ver. 2, CushingQoL) and disease severity scores as assessed by the Physicians Global Assessment of Disease Severity and Subject Rated Disease Severity questionnaire.
| Study Start Date:
| Study Completion Date:
| Primary Completion Date:
||June 2012 (Final data collection date for primary outcome measure)
mifepristone at doses from 300mg/day up to 1200mg/day
Other Name: CORLUX®
|Ages Eligible for Study:
||18 Years and older
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
Have a confirmed diagnosis of endogenous hypercortisolemia caused by ACTH dependent or ACTH independent etiologies including:
- Have documented biochemical evidence of endogenous hypercortisolemia which includes elevated urinary free cortisol.
- Require medical treatment of hypercortisolemia.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01371565
|The Center for Diabetes and Endocrine Care
|Hollywood, Florida, United States, 33021 |
|Sinai Hospital of Baltimore
|Baltimore, Maryland, United States, 21215 |
|University of Michigan Medical Center
|Ann Arbor, Michigan, United States, 48109 |
|Cleveland Clinic Foundation
|Cleveland, Ohio, United States, 44195 |
|The Ohio State University, Division of Endocrinology Diabetes and Metabolism
|Columbus, Ohio, United States, 43210 |
||Coleman Gross, M.D.
No publications provided
History of Changes
|Other Study ID Numbers:
|Study First Received:
||June 7, 2011
|Results First Received:
||September 19, 2013
||September 19, 2013
||United States: Food and Drug Administration
Keywords provided by Corcept Therapeutics:
Ectopic ACTH secretion
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on December 11, 2013
Pituitary ACTH Hypersecretion
Signs and Symptoms
Adrenal Gland Diseases
Endocrine System Diseases
Central Nervous System Diseases
Nervous System Diseases
Contraceptives, Oral, Synthetic
Contraceptive Agents, Female
Reproductive Control Agents
Physiological Effects of Drugs
Contraceptives, Postcoital, Synthetic
Hormones, Hormone Substitutes, and Hormone Antagonists
Abortifacient Agents, Steroidal