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

NCT03053271 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: ATR-101-301
• Study Type: interventional
• Target: 4
• Locations: 2 countries
• Sites: 4

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.

Conditions

Cushing Syndrome

Keywords

Endogenous Cushing's Syndrome; ATR-101; CS

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• The Proportion of Subjects With a Normal 24-hr UFC

  Through Day 85

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

  Through Day 85

• The Proportion of Subjects With a Normal 24-hr UFC

  Through Day 57 and Day 85

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

  Through Day 57 and Day 85

Study Arms (2)

ATR-101

EXPERIMENTAL

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.

Drug: ATR-101

Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Interventions

ATR-101 DRUG

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.

Also known as: nevanimibe HCl

ATR-101

Placebo DRUG

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.

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Millendo Therapeutics US, Inc.: lead

Study Sites (4)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

The Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53051, United States

Location

The James Cook University Hospital

Middlesbrough, TS4 3 BW, United Kingdom

Location

MeSH Terms

Conditions

Cushing Syndrome

Interventions

N-(2,6-bis(1-methylethyl)phenyl)-N'-((1-(4-(dimethylamino)phenyl)cyclopentyl)methyl)urea hydrochloride

Condition Hierarchy (Ancestors)

Adrenocortical Hyperfunction; Adrenal Gland Diseases; Endocrine System Diseases

Limitations and Caveats

Early termination leading to small numbers of subjects analyzed

Results Point of Contact

• Title: Clinical Trial Information
• Organization: Millendo Therapeutics

millendo@millendo.com

+1 734 845-9000

Study Officials

• James Findling, MD

  Medical College of Wisconsin

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 6, 2017
• First Posted: February 15, 2017
• Study Start: April 13, 2017
• Primary Completion: June 17, 2019
• Study Completion: August 12, 2019
• Last Updated: March 2, 2021
• Results First Posted: March 2, 2021

Record last verified: 2021-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (3); GB (1)