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

Cushing Syndrome Clinical Trial

Official title:

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ATR-101 for the Treatment of Cushing's Syndrome

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03053271
• Other study ID #: ATR-101-301
• Status: Terminated
• Phase: Phase 2
• Start date: April 13, 2017
• Est. completion date: August 12, 2019

Study information

• Verified date: February 2021
• Source: Millendo Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 4
• Est. completion date: August 12, 2019
• Est. primary completion date: June 17, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Cushing Syndrome
• Syndrome

Intervention

Drug:
• ATR-101
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.
• Placebo
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.

Locations

Country	Name	City	State
United Kingdom	The James Cook University Hospital	Middlesbrough
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	The Cleveland Clinic Foundation	Cleveland	Ohio
United States	Medical College of Wisconsin	Milwaukee	Wisconsin

Sponsors (1)

Lead Sponsor	Collaborator
Millendo Therapeutics US, Inc.

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Through Day 85
Secondary	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.	Through Day 85
Secondary	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.	Through Day 85
Secondary	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.	Through Day 57 and Day 85
Secondary	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.	Through Day 57 and Day 85