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

Purpose

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.

Condition

  • Cushing Syndrome

Eligibility

Eligible Ages
Between 18 Years and 80 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
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.
  • 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.
    Other names:
    • nevanimibe HCl
Placebo Comparator
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.
  • Drug: 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.

More Details

Status
Terminated
Sponsor
Millendo Therapeutics US, Inc.

Study Contact