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

Phase 2

Terminated

• Conditions: Cushing Syndrome
• Interventions: Drug: ATR-101; Drug: Placebo

• Registration Number: NCT03053271
• Lead Sponsor: Millendo Therapeutics US, Inc.

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-02-06
• Study First Submitted QC: 2017-02-10
• Study First Posted: 2017-02-15
• Study Start: 2017-04-13
• Primary Completion: 2019-06-17
• Study Completion: 2019-08-12
• Disposition First Submitted: 2019-10-16
• Results First Submitted: 2021-01-25
• Status Verified: 2021-02
• Results First Submitted QC: 2021-02-10
• Disposition First Submitted QC: 2021-02-10
• Last Update Submitted: 2021-02-10
• Results First Posted: 2021-03-02
• Disposition First Posted: 2021-03-02
• Last Update Posted: 2021-03-02

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 4

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ATR-101	ATR-101	During the 4-week randomized withdrawal period, eligible subjects will receive ATR-101 at the same dose level being used at the completion of the open-label dose-escalation period.
Placebo	Placebo	During the 4-week randomized withdrawal period, eligible subjects will receive a placebo that matches the same ATR-101 dose level being used at the completion of the open-label dose-escalation period.

Primary Outcome Measures

Name	Time	Method
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	Through Day 85	The number of subjects meeting the criterion was divided by the total number of subjects.

Secondary Outcome Measures

Name	Time	Method
The Proportion of Subjects With a Normal 24-hr UFC	Through Day 57 and Day 85	The number of subjects meeting the criterion was divided by the total number of subjects.
The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value	Through Day 57 and Day 85	The number of subjects meeting the criterion was divided by the total number of subjects.

Trial Locations

Locations (4)

Medical College of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

The James Cook University Hospital; 🇬🇧 Middlesbrough, United Kingdom

The Cleveland Clinic Foundation; 🇺🇸 Cleveland, Ohio, United States