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

Status:	Recruiting
Conditions:	Endocrine
Therapuetic Areas:	Endocrinology
Healthy:	No
Age Range:	18 - 80
Updated:	2/17/2019
Start Date:	February 16, 2017
End Date:	November 2020
Contact:	Marian Ijzerman, PhD
Email:	ijzerman@millendo.com
Phone:	734-845-9300

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

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.

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

We found this trial at

sites

Oregon Health and Sciences University

Portland, Oregon 97227

Principal Investigator: Maria Fleseriu, MD

Phone: 503-494-9546

829

from 91732

Portland, OR