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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| mifepristone | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Mifepristone | Drug | mifepristone at doses from 300mg/day up to 1200mg/day |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events | Safety was assessed at all visits and adverse events were recorded. | 6 months |
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Inclusion Criteria:
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)
Ectopic ACTH
Ectopic CRF secretion
Adrenal adenoma
Adrenal carcinoma
Adrenal autonomy
Have documented biochemical evidence of endogenous hypercortisolemia which includes elevated urinary free cortisol.
Require medical treatment of hypercortisolemia.
Exclusion Criteria:
Individuals not eligible to be enrolled into the study are those who:
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| Name | Affiliation | Role |
|---|---|---|
| Coleman Gross, M.D. | Corcept Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Center for Diabetes and Endocrine Care | Hollywood | Florida | 33021 | United States | ||
| Sinai Hospital of Baltimore |
All patients received active drug (no placebo).
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | Mifepristone | At doses from 300mg/day up to 1200mg/day |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Baltimore |
| Maryland |
| 21215 |
| United States |
| University of Michigan Medical Center | Ann Arbor | Michigan | 48109 | United States |
| Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | United States |
| The Ohio State University, Division of Endocrinology Diabetes and Metabolism | Columbus | Ohio | 43210 | United States |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Subjects who received one dose of study drug were included in the safety analysis.
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| ID | Title | Description |
|---|---|---|
| BG000 | Mifepristone | At doses from 300mg/day up to 1200mg/day |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Adverse Events | Safety was assessed at all visits and adverse events were recorded. | 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. | Posted | Number | participants | 6 months |
|
|
|
From date of consent to final visit.
All patients who received at least one dose of study drug are included.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Mifepristone | At doses from 300mg/day up to 1200mg/day | 1 | 4 | 4 | 4 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Adrenal carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 10 | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA 10 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 10 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pancytopenia | Blood and lymphatic system disorders | MedDRA 10 | Systematic Assessment |
| |
| Eye irritation | Eye disorders | MedDRA 10 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 10 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 10 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 10 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 10 | Systematic Assessment |
| |
| Swelling | General disorders | MedDRA 10 | Systematic Assessment |
| |
| Thirst | General disorders | MedDRA 10 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 10 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 10 | Systematic Assessment |
| |
| Gallbladder polyp | Hepatobiliary disorders | MedDRA 10 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 10 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 10 | Systematic Assessment |
| |
| Blood potassium decreased | Investigations | MedDRA 10 | Systematic Assessment |
| |
| Blood potassium increased | Investigations | MedDRA 10 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 10 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 10 | Systematic Assessment |
| |
| Food craving | Metabolism and nutrition disorders | MedDRA 10 | Systematic Assessment |
| |
| Metabolic alkalosis | Metabolism and nutrition disorders | MedDRA 10 | Systematic Assessment |
| |
| Arthropathy | Musculoskeletal and connective tissue disorders | MedDRA 10 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 10 | Systematic Assessment |
| |
| Muscle twitching | Musculoskeletal and connective tissue disorders | MedDRA 10 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 10 | Systematic Assessment |
| |
| Dysarthria | Nervous system disorders | MedDRA 10 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 10 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 10 | Systematic Assessment |
| |
| Restless legs syndrome | Nervous system disorders | MedDRA 10 | Systematic Assessment |
| |
| Restless | Nervous system disorders | MedDRA 10 | Systematic Assessment |
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| Somnolence | Nervous system disorders | MedDRA 10 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 10 | Systematic Assessment |
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| Insomnia | Psychiatric disorders | MedDRA 10 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 10 | Systematic Assessment |
| |
| Nocturia | Renal and urinary disorders | MedDRA 10 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 10 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 10 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 10 | Systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 10 | Systematic Assessment |
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| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 10 | Systematic Assessment |
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| Drug eruption | Skin and subcutaneous tissue disorders | MedDRA 10 | Systematic Assessment |
| |
| Skin erosion | Skin and subcutaneous tissue disorders | MedDRA 10 | Systematic Assessment |
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| Epistaxis | Vascular disorders | MedDRA 10 | Systematic Assessment |
| |
| Haemorrhage | Vascular disorders | MedDRA 10 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 10 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 10 | Systematic Assessment |
| |
| Jugular vein thrombosis | Vascular disorders | MedDRA 10 | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Corcept Therapeutics | 650-327-3270 | info@corcept.com |
| ID | Term |
|---|---|
| D047748 | Pituitary ACTH Hypersecretion |
| D003480 | Cushing Syndrome |
| D010900 | Pituitary Diseases |
| D000182 | ACTH Syndrome, Ectopic |
| ID | Term |
|---|---|
| D006964 | Hyperpituitarism |
| D007027 | Hypothalamic Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D004700 | Endocrine System Diseases |
| D000308 | Adrenocortical Hyperfunction |
| D000307 | Adrenal Gland Diseases |
| D009384 | Paraneoplastic Endocrine Syndromes |
| D010257 | Paraneoplastic Syndromes |
| D009369 | Neoplasms |
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| ID | Term |
|---|---|
| D015735 | Mifepristone |
| C511997 | Corlux |
| ID | Term |
|---|---|
| D004963 | Estrenes |
| D004962 | Estranes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
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