A Study of ATR-101 for the Treatment of Endogenous Cushing's Syndrome

• Status: Terminated
• Trial end date: 2019-08-12
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Millendo Therapeutics US, Inc.; Millendo Therapeutics, Inc.

Criteria

Inclusion Criteria:

• Confirmed diagnosis of endogenous Cushing's syndrome

• Baseline UFC 1.3 to 10 × upper limit of normal (ULN)

• If previous pituitary surgery, participants must be at least 3 months since surgery at the time of screening

• BMI between 18 and 60 kg/m2, inclusive

Exclusion Criteria:

• Pseudo-Cushing's syndrome, cyclic Cushing's syndrome or current iatrogenic Cushing's syndrome

• Candidates for surgical treatment of Cushing's syndrome, unless surgery is not anticipated to occur during the study

• Normal late night salivary cortisol or 24-hr urine free cortisol

• Radiotherapy of the pituitary within 6 months