This is a Phase 2 multicenter, randomized, double-blind, placebo controlled study of ATR-101 to evaluate the efficacy and safety of orally-administered ATR-101 in adults with endogenous Cushing's syndrome. Following wash-out (if needed), all eligible subjects will enter an open-label intra-subject dose-escalation period of 8 weeks' duration, followed either by a double-blind randomized withdrawal period of 4 weeks' duration (if the subject meets randomization criteria) or by an additional open label dosing period of 4 weeks' duration (if the subject does not meet randomization criteria).It is anticipated that the overall duration of the study per subject will range from approximately 16-22 weeks.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
4
During the 4-week randomized withdrawal period, subjects will be dosed for 4 weeks at the same dose level being used at the completion of the open-label dose-escalation period.
During the 4-week randomized withdrawal period, subjects will be dosed for 4 weeks with placebo that matches the same ATR-101 dose level being used at the completion of the open-label dose-escalation period.
Massachusetts General Hospital
Boston, Massachusetts, United States
The Cleveland Clinic Foundation
Cleveland, Ohio, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
The James Cook University Hospital
Middlesbrough, United Kingdom
The Proportion of Subjects With Either a Normal 24-hr Urinary Free Cortisol (UFC) or a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value
The number of subjects meeting the criterion was divided by the total number of subjects.
Time frame: Through Day 85
The Proportion of Subjects With a Normal 24-hr UFC
The number of subjects meeting the criterion was divided by the total number of subjects.
Time frame: Through Day 85
The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value
The number of subjects meeting the criterion was divided by the total number of subjects.
Time frame: Through Day 85
The Proportion of Subjects With a Normal 24-hr UFC
The number of subjects meeting the criterion was divided by the total number of subjects.
Time frame: Through Day 57 and Day 85
The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value
The number of subjects meeting the criterion was divided by the total number of subjects.
Time frame: Through Day 57 and Day 85
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