Extension Study to Evaluate the Safety of Long-Term Use of Relacorilant in Patients With Cushing Syndrome

This study is designed to allow continued therapy with relacorilant, a potent, selective glucocorticoid receptor (GR) antagonist in patients who have successfully completed participation of a Corcept-sponsored study of relacorilant (referred to as the "parent" study). Patients may qualify to enter this extension study if they complete their last treatment visit in their parent study and in the Investigator's opinion will benefit from continued treatment.

Once-daily dosing with relacorilant may continue for patients who receive clinical benefits (as judged by the Investigator) until relacorilant is commercially or otherwise available or the study is stopped by the Sponsor. A patient's dose may be maintained, reduced, or increased based on individual response and tolerability.

• relacorilant (Drug), also known as CORT125134: CORT125134 is supplied as capsules for oral dosing.

Primary outcomes

• Long-term safety of relacorilant [36 months]: Number of patients with treatment-emergent adverse events (TEAEs) as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v4.0 or higher

Other outcomes

• Number of patients with one or more hematology laboratory abnormality by severity grade [Up to Month 99]: The hematology laboratory abnormalities to be assessed are hemoglobin decreased, hemoglobin increased, platelet count decreased, white blood cell count decreased, leukocytosis, eosinophilia, lymphocyte count decreased, lymphocyte count increased, neutrophil count decreased. The laboratory abnormalities will be reported by worst grade of mild, moderate, severe, or life-threatening, using the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE, version 4 or higher) criteria.
• Number of patients with one or more clinical chemistry laboratory abnormality by severity grade [Up to Month 99]: The clinical chemistry laboratory abnormalities to be assessed are hypoalbuminemia, alkaline phosphatase increased, alanine aminotransferase increased, aspartate aminotransferase increased, hypocalcemia, hypercalcemia, creatine increased, hypoglycemia, hyperglycemia, blood lactate dehydrogenase increased, hypomagnesemia, hypermagnesemia, hypokalemia, hyperkalemia, hyponatremia, hypernatremia, creatine phosphokinase increased, blood bilirubin increased, and hyperuricemia. The laboratory abnormalities will be reported by worst grade of mild, moderate, severe, or life-threatening, using the CTCAE, version 4 or higher criteria.
• Change from Baseline in oral body temperature [Baseline and up to Month 99]
• Change from Baseline in respiratory rate [Baseline and up to Month 99]
• Change from Baseline in blood pressure [Baseline and up to Month 99]: Systolic and diastolic blood pressure will be assessed.
• Change from Baseline in heart rate [Baseline and up to Month 99]
• Change from Baseline in electrocardiogram (ECG) parameters [Baseline and Month 3]: 12-lead ECGs will be performed in duplicate and will be evaluated by a central reviewer. The parameters to be assessed are PR interval, QRS interval, QT interval, RR interval, QTcF interval, and QTcB interval.
• Change from Baseline in ECG ventricular rate [Baseline and Month 3]: 12-lead ECGs will be performed in duplicate and will be evaluated by a central reviewer.
• Change from Baseline in the longest vertical diameter of pituitary tumors as determined with magnetic resonance imaging (MRI) [Baseline and up to Month 72]: Pituitary MRI imaging will be used to assess changes in tumor size during dosing. A computed tomography (CT) scan can be used in patients for whom MRI is contraindicated.
• Change from baseline in the volume of pituitary tumors as determined with MRI [Baseline and up to Month 72]: Pituitary MRI imaging will be used to assess changes in tumor size during dosing. A CT scan can be used in patients for whom MRI is contraindicated.

Sex: All

Inclusion Criteria:

* Major Inclusion Criteria:

  * Have completed a Corcept-sponsored study of relacorilant in endogenous Cushing syndrome
  * According to the Investigator's opinion will benefit from continuing treatment with relacorilant.

Exclusion Criteria:

* Major Exclusion Criteria:

  * Premature discontinuation from a relacorilant parent study
  * Has uncontrolled, clinically significant hypothyroidism or hyperthyroidism.

• John McNally, Study director, Corcept Therapeutics