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

Primary Purpose

Cushing Syndrome

Status

Terminated

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

ATR-101

Placebo

About this trial

This is an interventional treatment trial for Cushing Syndrome focused on measuring Endogenous Cushing's Syndrome, ATR-101, CS

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Massachusetts General Hospital
The Cleveland Clinic Foundation
Medical College of Wisconsin
The James Cook University Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

ATR-101

Placebo

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.

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.

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.

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.

Full Information

NCT ID

NCT03053271

First Posted

February 6, 2017

Last Updated

February 10, 2021

Sponsor

Millendo Therapeutics US, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT03053271

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

February 2021

Overall Recruitment Status

Terminated

Why Stopped

Slow enrollment

Study Start Date

April 13, 2017 (Actual)

Primary Completion Date

June 17, 2019 (Actual)

Study Completion Date

August 12, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Millendo Therapeutics US, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cushing Syndrome

Keywords

Endogenous Cushing's Syndrome, ATR-101, CS

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

4 (Actual)

8. Arms, Groups, and Interventions

Arm Title

ATR-101

Arm Type

Experimental

Arm Description

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.

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Intervention Type

Drug

Intervention Name(s)

ATR-101

Other Intervention Name(s)

nevanimibe HCl

Intervention Description

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.

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

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.

Primary Outcome Measure Information:

Title

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

Description

The number of subjects meeting the criterion was divided by the total number of subjects.

Time Frame

Through Day 85

Secondary Outcome Measure Information:

Title

The Proportion of Subjects With a Normal 24-hr UFC

Description

The number of subjects meeting the criterion was divided by the total number of subjects.

Time Frame

Through Day 85

Title

The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value

Description

The number of subjects meeting the criterion was divided by the total number of subjects.

Time Frame

Through Day 85

Title

The Proportion of Subjects With a Normal 24-hr UFC

Description

The number of subjects meeting the criterion was divided by the total number of subjects.

Time Frame

Through Day 57 and Day 85

Title

The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value

Description

The number of subjects meeting the criterion was divided by the total number of subjects.

Time Frame

Through Day 57 and Day 85

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

80 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

James Findling, MD

Organizational Affiliation

Medical College of Wisconsin

Official's Role

Principal Investigator

Facility Information:

Facility Name

Massachusetts General Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02114

Country

United States

Facility Name

The Cleveland Clinic Foundation

City

Cleveland

State/Province

Ohio

ZIP/Postal Code

44195

Country

United States

Facility Name

Medical College of Wisconsin

City

Milwaukee

State/Province

Wisconsin

ZIP/Postal Code

53051

Country

United States

Facility Name

The James Cook University Hospital

City

Middlesbrough

ZIP/Postal Code

TS4 3 BW

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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