Study to Evaluate CORT125134 in Participants With Cushing's Syndrome
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|ClinicalTrials.gov Identifier: NCT02804750|
Recruitment Status : Completed
First Posted : June 17, 2016
Results First Posted : September 25, 2019
Last Update Posted : October 15, 2019
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Cushing's syndrome is a relatively rare disorder caused by prolonged exposure to high levels of the glucocorticoid hormone cortisol. Cushing's syndrome may result from elevated endogenous or exogenous sources of cortisol. Endogenous Cushing's syndrome resulting from cortisol overproduction by the adrenal glands is the subject of this protocol.
Patients with exogenous Cushing's syndrome, which develops as a side effect of chronic administration of high doses of glucocorticoids, were not eligible for enrollment in this study.
The purpose of this study was to evaluate the safety and efficacy of CORT125134 for treatment of endogenous Cushing's syndrome. The multicenter study was conducted in the United States and in Europe.
|Condition or disease||Intervention/treatment||Phase|
|Cushing's Syndrome||Drug: CORT125134||Phase 2|
This was a Phase 2, open-label study with two dose groups, each with a two-step dose escalation, designed to evaluate the safety and efficacy of CORT125134 for the treatment of endogenous Cushing's syndrome. CORT125134 was administered orally once daily for 16 weeks with dose escalations occurring every 4 weeks.
Pharmacokinetics (PK) profiles were generated at every dose level. A data review committee reviewed PK and safety data and recommended the final plan for dose escalation in Group 2.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||35 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase 2 Study of the Safety and Efficacy of CORT125134 in the Treatment of Endogenous Cushing's Syndrome|
|Actual Study Start Date :||June 2016|
|Actual Primary Completion Date :||September 2018|
|Actual Study Completion Date :||September 2018|
Experimental: Group 1: Low-dose Group
100 mg/day for 4 weeks in Period 1, then 150 mg/day for 4 weeks in Period 2, then 200 mg/day for 4 weeks in Period 3. There was no washout between treatment periods. Period 3 was followed by a 4-week follow-up period. Per-protocol, Group 1 did not participate in treatment Period 4.
Experimental: Group 2: High-dose Group
250 mg/day for 4 weeks in Period 1, then 300 mg/day for 4 weeks in Period 2, then 350 mg/day for 4 weeks in Period 3, then 400 mg/day for 4 weeks in Period 4. There was no washout between treatment periods. Period 4 was followed by a 4-week follow-up period.
- Percentage of Participants With One or More Adverse Events [ Time Frame: Group 1: up to Week 16; Group 2: up to Week 20 ]All treatment-emergent adverse events were recorded and summarized.
- Percentage of Participants With One or More Severe (≥Grade 3) Adverse Events [ Time Frame: Group 1: up to Week 16; Group 2: up to Week 20 ]All treatment-emergent adverse events with Common Terminology Criteria for Adverse Events (CTCAE) ≥Grade 3 (severe) were recorded and summarized.
- Percentage of Participants With Hypertension Who Experience Improvement in Blood Pressure Following Treatment With CORT125134 [ Time Frame: Group 1: Week 12 or last observation; Group 2: Week 16 or last observation ]Improvement in blood pressure was defined as a participant who experiences at least a 5 mmHg decrease in mean diastolic or systolic BP from baseline who has not taken an additional antihypertensive medication during the treatment period or increased the dosage of a concurrent antihypertensive medication.
- Percentage of Participants With IGT / T2DM Who Experienced a ≥25% Reduction in AUCglucose Following Treatment With CORT125134 [ Time Frame: Before and 0.5, 1, 1.5, and 2 hours after a glucose drink at Week 12 or last observation (Group 1) or Week 16 or last observation (Group 2) ]Improvement in glucose control was defined as a participant who experiences at least a 25% decrease from baseline in area under the concentration-time curve for blood glucose (AUCglucose) who has not taken an additional diabetes medication during the treatment period or increased the dosage of a concurrent diabetes medication.
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|Ages Eligible for Study:||18 Years to 80 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Has a confirmed diagnosis of endogenous Cushing's syndrome.
- Requires medical treatment of hypercortisolemia.
Meets at least one of the following criteria:
- Has type 2 diabetes mellitus.
- Has impaired glucose tolerance.
- Has hypertension.
- Has non-endogenous source of hypercortisolemia
- Has uncontrolled, clinically significant hypothyroidism or hyperthyroidism
- Has poorly controlled hypertension
- Has Stage ≥ 4 renal failure
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02804750
|United States, California|
|Laguna Hills, California, United States, 92653|
|United States, Colorado|
|Aurora, Colorado, United States, 80045|
|United States, Florida|
|Fort Lauderdale, Florida, United States, 33312|
|Miami, Florida, United States, 33136|
|United States, Indiana|
|Indianapolis, Indiana, United States, 46202|
|United States, Kentucky|
|Covington, Kentucky, United States, 41011|
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|United States, Missouri|
|Saint Louis, Missouri, United States, 63110|
|United States, New York|
|New York, New York, United States, 10016|
|United States, Ohio|
|Cleveland, Ohio, United States, 44195|
|United States, Pennsylvania|
|Pittsburgh, Pennsylvania, United States, 15212|
|United States, Virginia|
|Richmond, Virginia, United States, 23119|
|Salford, Manchester, United Kingdom|
|Study Director:||Andreas G Moraitis, MD||Corcept Therapeutics|
Documents provided by Corcept Therapeutics:
|Responsible Party:||Corcept Therapeutics|
|Other Study ID Numbers:||
|First Posted:||June 17, 2016 Key Record Dates|
|Results First Posted:||September 25, 2019|
|Last Update Posted:||October 15, 2019|
|Last Verified:||October 2019|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
Type 2 Diabetes
Impaired Glucose Intolerance
Adrenal Corticotrophic Hormone (ACTH)
Primary Pigmented Nodular Adrenal Disease (PPNAD)
Dorsocervical Fat Pad
Adrenal Gland Diseases
Endocrine System Diseases