Trial Outcomes & Findings for A Study of the Efficacy and Safety of Relacorilant in Patients With Endogenous Cushing Syndrome (NCT NCT03697109)

Last Updated: 2025-07-16

Results Overview

Loss of response with respect to HTN was measured using 6 criteria: 1) an increase in SBP of at least 5 mm Hg, 2) an increase in DBP of at least 5 mm Hg, 3) an increase in SBP and/or DBP of at least 5 mm Hg, 4) use of HTN rescue medication, 5) treatment discontinuation, and 6) missing 24-hour ambulatory blood pressure monitoring (ABPM) measurement at the end of the RW Phase. Blood pressure was measured using ABPM. Use of rescue medication was defined as any increase, modification, or addition of antihypertensive medication due to worsening HTN. Treatment discontinuation reports the number of patients who discontinued study treatment in the RW Phase for any reason.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

152 participants

Primary outcome timeframe

Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

Results posted on

2025-07-16

Participant Flow

A total of 404 patients were screened and 152 were enrolled.

Participant milestones

Participant milestones
Measure	Relacorilant (OL Phase) Patients will receive relacorilant increased sequentially from 100 mg once daily to a maximum dose of 400 mg once daily. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Relacorilant (RW Phase) Patients who meet any of the response criteria will advance to the RW Phase of the study and receive the same dose of relacorilant as the last dose administered in the OL Phase. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Placebo (RW Phase) Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug
Open-label (OL) Phase STARTED	152	0	0
Open-label (OL) Phase COMPLETED	95	0	0
Open-label (OL) Phase NOT COMPLETED	57	0	0
Randomized-withdrawal (RW) Phase STARTED	0	30	32
Randomized-withdrawal (RW) Phase COMPLETED	0	27	31
Randomized-withdrawal (RW) Phase NOT COMPLETED	0	3	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Relacorilant (OL Phase) Patients will receive relacorilant increased sequentially from 100 mg once daily to a maximum dose of 400 mg once daily. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Relacorilant (RW Phase) Patients who meet any of the response criteria will advance to the RW Phase of the study and receive the same dose of relacorilant as the last dose administered in the OL Phase. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Placebo (RW Phase) Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug
Open-label (OL) Phase Adverse Event	24	0	0
Open-label (OL) Phase Lost to Follow-up	2	0	0
Open-label (OL) Phase Lack of Efficacy	6	0	0
Open-label (OL) Phase Withdrawal by Subject	21	0	0
Open-label (OL) Phase Physician Decision	2	0	0
Open-label (OL) Phase Death	2	0	0
Randomized-withdrawal (RW) Phase Adverse Event	0	1	0
Randomized-withdrawal (RW) Phase Lack of Efficacy	0	1	0
Randomized-withdrawal (RW) Phase Withdrawal by Subject	0	1	1

Baseline Characteristics

A Study of the Efficacy and Safety of Relacorilant in Patients With Endogenous Cushing Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Relacorilant (OL Phase) n=152 Participants Patients will receive relacorilant increased sequentially from 100 mg once daily to a maximum dose of 400 mg once daily. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.
Age, Continuous	50.4 years STANDARD_DEVIATION 13.23 • n=5 Participants
Sex: Female, Male Female	127 Participants n=5 Participants
Sex: Female, Male Male	25 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	28 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	123 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=5 Participants
Race (NIH/OMB) Black or African American	18 Participants n=5 Participants
Race (NIH/OMB) White	121 Participants n=5 Participants
Race (NIH/OMB) More than one race	6 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	6 Participants n=5 Participants
Body weight	93.77 kg STANDARD_DEVIATION 24.660 • n=5 Participants
Body Mass Index (BMI)	34.738 kg/m^2 STANDARD_DEVIATION 32.675 • n=5 Participants
Waist circumference	114.85 cm STANDARD_DEVIATION 18.0355 • n=5 Participants
Hypertension (HTN) only	31 Participants n=5 Participants
Diabetes mellitus (DM) or impaired glucose tolerance (IGT) only	50 Participants n=5 Participants
HTN and DM/IGT	71 Participants n=5 Participants

PRIMARY outcome

Timeframe: Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

Population: The analysis population was patients in the Intent-to-Treat (ITT-RW) Population who met the HTN response criteria at conclusion of the OL Phase. The ITT-RW Population included all patients who were randomized in the RW Phase and received at least 1 dose of study drug post randomization.

Outcome measures

Outcome measures
Measure	Relacorilant (RW Phase) n=21 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive the same dose of relacorilant as the last dose administered in the OL Phase. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Placebo (RW Phase) n=22 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug.	Placebo (RW Phase) Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug
Number of Patients With Loss of Response With Respect to Hypertension During the RW Phase. SBP	3 Participants	1 Participants	—
Number of Patients With Loss of Response With Respect to Hypertension During the RW Phase. DBP	1 Participants	1 Participants	—
Number of Patients With Loss of Response With Respect to Hypertension During the RW Phase. SBP and/or DBP	3 Participants	4 Participants	—
Number of Patients With Loss of Response With Respect to Hypertension During the RW Phase. Use of rescue medication	0 Participants	7 Participants	—
Number of Patients With Loss of Response With Respect to Hypertension During the RW Phase. Treatment discontinuation	2 Participants	3 Participants	—
Number of Patients With Loss of Response With Respect to Hypertension During the RW Phase. Missing ABPM at end of RW Phase	2 Participants	3 Participants	—

PRIMARY outcome

Timeframe: OL Phase: Up to 22 weeks; RW Phase: up to 18 weeks after completion of the OL Phase

Population: The analysis population was patients in the Safety Population, which included all enrolled patients who received at least 1 dose of study drug.

Outcome measures

Outcome measures
Measure	Relacorilant (RW Phase) n=152 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive the same dose of relacorilant as the last dose administered in the OL Phase. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Placebo (RW Phase) n=30 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug.	Placebo (RW Phase) n=32 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug
Number of Patients With 1 or More Treatment-emergent Adverse Events (TEAEs) as Graded by CTCAE v5.0.	147 Participants	23 Participants	27 Participants

SECONDARY outcome

Timeframe: Before and at time intervals up to 2 hours post glucose drink at Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

Population: The analysis population was patients in the ITT-RW Population who had DM/IGT with or without HTN at Baseline.

Outcome measures

Outcome measures
Measure	Relacorilant (RW Phase) n=18 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive the same dose of relacorilant as the last dose administered in the OL Phase. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Placebo (RW Phase) n=20 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug.	Placebo (RW Phase) Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug
Change in Area Under the Concentration-time Curve of Blood Glucose (AUCglucose) During the RW Phase	1.91 hours x mmol/L Interval -1.138 to 4.966	4.81 hours x mmol/L Interval 2.096 to 7.529	—

SECONDARY outcome

Timeframe: Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

Population: The analysis population was patients in the ITT-RW Population who had DM/IGT with or without HTN at Baseline.

Outcome measures

Outcome measures
Measure	Relacorilant (RW Phase) n=16 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive the same dose of relacorilant as the last dose administered in the OL Phase. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Placebo (RW Phase) n=19 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug.	Placebo (RW Phase) Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug
Change in Hemoglobin HbA1c During the RW Phase	0.06 Percentage Interval -0.27 to 0.383	0.28 Percentage Interval -0.022 to 0.578	—

SECONDARY outcome

Timeframe: Before and 2 hours post glucose drink at Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

Population: The analysis population was patients in the ITT-RW Population who had DM/IGT with or without HTN at Baseline.

Plasma glucose was measured using the 2-hour Oral Glucose Tolerance Test (oGTT).

Outcome measures

Outcome measures
Measure	Relacorilant (RW Phase) n=15 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive the same dose of relacorilant as the last dose administered in the OL Phase. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Placebo (RW Phase) n=19 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug.	Placebo (RW Phase) Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug
Change in 2-hour Plasma Glucose During the RW Phase	0.52 mmol/L Interval -1.06 to 2.09	2.96 mmol/L Interval 1.55 to 4.37	—

SECONDARY outcome

Timeframe: Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

Population: The analysis population was patients in the ITT-RW Population who had HTN with or without DM/IGT at Baseline.

Blood pressure was measured by 24-hour ABPM.

Outcome measures

Outcome measures
Measure	Relacorilant (RW Phase) n=21 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive the same dose of relacorilant as the last dose administered in the OL Phase. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Placebo (RW Phase) n=22 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug.	Placebo (RW Phase) Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug
Change in SBP and DBP During the RW Phase SBP	3.95 mm Hg Interval 0.47 to 7.424	10.43 mm Hg Interval 6.538 to 14.329	—
Change in SBP and DBP During the RW Phase DBP	2.86 mm Hg Interval -0.199 to 5.928	6.53 mm Hg Interval 3.224 to 9.832	—

SECONDARY outcome

Timeframe: Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

Population: The analysis population was patients in the ITT-RW Population.

Outcome measures

Outcome measures
Measure	Relacorilant (RW Phase) n=26 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive the same dose of relacorilant as the last dose administered in the OL Phase. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Placebo (RW Phase) n=30 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug.	Placebo (RW Phase) Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug
Change in Body Weight During the RW Phase	-1.21 kg Interval -2.685 to 0.269	0.54 kg Interval -0.851 to 1.939	—

SECONDARY outcome

Timeframe: Week 22 (end of OL Phase) and up to Week 36 (Week 12 of RW Phase)

Population: The analysis population was patients in the ITT-RW Population who had DM/IGT at Baseline and received antidiabetic medication during the RW Phase.

Outcome measures

Outcome measures
Measure	Relacorilant (RW Phase) n=7 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive the same dose of relacorilant as the last dose administered in the OL Phase. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Placebo (RW Phase) n=8 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug.	Placebo (RW Phase) Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug
Number of Patients With Any Increase or Modification in Diabetes Medication During the RW Phase	1 Participants	0 Participants	—

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

Population: The analysis population was patients in the ITT-RW Population.

The Cushing QoL patient questionnaire, which evaluates the health-related QoL in patients with Cushing syndrome, comprises 12 questions, each with 5 possible answers. The total score ranges from 12-60, with a higher score indicating improvement in QoL. The Cushing QoL instrument addresses known problem areas associated with Cushing syndrome including trouble sleeping, wound healing/bruising, irritability/mood swings/anger, self-confidence, physical changes, ability to participate in activities, interactions with friends and family, memory issues, and future health concerns.

Outcome measures

Outcome measures
Measure	Relacorilant (RW Phase) n=27 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive the same dose of relacorilant as the last dose administered in the OL Phase. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Placebo (RW Phase) n=31 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug.	Placebo (RW Phase) Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug
Change in Cushing Quality of Life (QoL) Normalized Total Score During the RW Phase	0.59 score on a scale Interval -4.28 to 5.46	-0.58 score on a scale Interval -5.14 to 3.98	—

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 22 (end of OL Phase) and Week 36 (Week 12 of RW Phase)

Population: The analysis population was patients in the ITT-RW Population.

Tissue fat mass was measured by dual energy X-ray absorptiometry (DXA) scan. Reported are change in in absolute tissue fat mass and change in percent tissue fat mass.

Outcome measures

Outcome measures
Measure	Relacorilant (RW Phase) n=17 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive the same dose of relacorilant as the last dose administered in the OL Phase. Relacorilant: Relacorilant is supplied as 100 mg capsules for oral dosing.	Placebo (RW Phase) n=22 Participants Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug.	Placebo (RW Phase) Patients who meet any of the response criteria will advance to the RW Phase of the study and receive placebo matched to study drug. Placebo: Placebo matched to study drug
Percent Change in Tissue Fat Mass During the RW Phase Absolute Tissue Fat Mass	-0.64 Percent Change Interval -1.684 to 0.4	1.67 Percent Change Interval 0.776 to 2.573	—
Percent Change in Tissue Fat Mass During the RW Phase Percent Tissue Fat Mass	-0.14 Percent Change Interval -1.031 to 0.759	1.66 Percent Change Interval 0.887 to 2.437	—

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 22 (end of OL Phase) and up to Week 36 (Week 12 of RW Phase)

The Global Clinical Response assessment measures the patient's signs and symptoms of endogenous hypercortisolism in 7 clinical categories: 1) glucose parameters, 2) blood pressure parameters, 3) body composition parameters, 4) clinical appearance, 5) strength parameters, 6) psychiatric health/cognitive function parameters, and 7) quality of life using the Cushing QoL score. The overall response based on the totality of signs and symptoms is rated as +1 for improved, 0 for unchanged, and -1 for worsened. Each patient's final score is the median of ratings given by 3 members of the Data Review Board.