Trial Outcomes & Findings for Compassionate Use of CORLUX® (Mifepristone) in the Treatment of Signs and Symptoms of Endogenous Cushing's Syndrome (NCT NCT01371565)

NCT ID: NCT01371565

Last Updated: 2014-03-18

Results Overview

Safety was assessed at all visits and adverse events were recorded.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 4 participants
• Primary outcome timeframe: 6 months
• Results posted on: 2014-03-18

Participant Flow

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.

All patients received active drug (no placebo).

Participant milestones

Measure	Mifepristone At doses from 300mg/day up to 1200mg/day
Overall Study STARTED	4
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Measure	Mifepristone At doses from 300mg/day up to 1200mg/day
Overall Study Withdrawal by Subject	1
Overall Study Death	1
Overall Study Resection of neuroendocrine tumor	1

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Mifepristone n=4 Participants At doses from 300mg/day up to 1200mg/day
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Age, Continuous	44.8 years STANDARD_DEVIATION 17.9 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

Safety was assessed at all visits and adverse events were recorded.

Outcome measures

Measure	Mifepristone n=4 Participants At doses from 300mg/day up to 1200mg/day
Number of Participants With Adverse Events	4 participants

Adverse Events

Mifepristone

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Measure	Mifepristone n=4 participants at risk At doses from 300mg/day up to 1200mg/day
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Adrenal carcinoma	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Psychiatric disorders Mental status changes	25.0% 1/4 • Number of events 2 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Renal and urinary disorders Haematuria	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.

Other adverse events

Measure	Mifepristone n=4 participants at risk At doses from 300mg/day up to 1200mg/day
Blood and lymphatic system disorders Pancytopenia	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Eye disorders Eye irritation	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Gastrointestinal disorders Dysphagia	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Gastrointestinal disorders Dyspepsia	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Gastrointestinal disorders Vomiting	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
General disorders Influenza like illness	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
General disorders Swelling	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
General disorders Thirst	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
General disorders Asthenia	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
General disorders Fatigue	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Hepatobiliary disorders Gallbladder polyp	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Infections and infestations Influenza	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Infections and infestations Upper respiratory tract infection	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Investigations Blood potassium decreased	50.0% 2/4 • Number of events 2 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Investigations Blood potassium increased	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Metabolism and nutrition disorders Hypoglycaemia	50.0% 2/4 • Number of events 2 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Metabolism and nutrition disorders Decreased appetite	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Metabolism and nutrition disorders Food craving	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Metabolism and nutrition disorders Metabolic alkalosis	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Musculoskeletal and connective tissue disorders Arthropathy	25.0% 1/4 • Number of events 2 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Musculoskeletal and connective tissue disorders Back pain	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Musculoskeletal and connective tissue disorders Muscle twitching	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Musculoskeletal and connective tissue disorders Pain in extremity	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Nervous system disorders Dysarthria	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Nervous system disorders Headache	25.0% 1/4 • Number of events 2 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Nervous system disorders Hypoaesthesia	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Nervous system disorders Restless legs syndrome	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Nervous system disorders Restless	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Nervous system disorders Somnolence	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Psychiatric disorders Anxiety	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Psychiatric disorders Insomnia	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Renal and urinary disorders Haematuria	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Renal and urinary disorders Nocturia	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Respiratory, thoracic and mediastinal disorders Acute respiratory failure	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Respiratory, thoracic and mediastinal disorders Cough	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Respiratory, thoracic and mediastinal disorders Pleural effusion	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Respiratory, thoracic and mediastinal disorders Pneumonia aspiration	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Skin and subcutaneous tissue disorders Alopecia	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Skin and subcutaneous tissue disorders Drug eruption	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Skin and subcutaneous tissue disorders Skin erosion	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Vascular disorders Epistaxis	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Vascular disorders Haemorrhage	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Vascular disorders Hypertension	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Vascular disorders Hypotension	25.0% 1/4 • Number of events 2 • From date of consent to final visit. All patients who received at least one dose of study drug are included.
Vascular disorders Jugular vein thrombosis	25.0% 1/4 • Number of events 1 • From date of consent to final visit. All patients who received at least one dose of study drug are included.

Additional Information

Medical Director

Corcept Therapeutics

Phone: 650-327-3270

Email: info@corcept.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: GT60