Clinical trial NCT03053271: 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
Overall status: TERMINATED
Phase: PHASE2
Conditions: Cushing Syndrome
Keywords: Endogenous Cushing's Syndrome
ATR-101
CS
Lead sponsor: Millendo Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2017-02-15
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: 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
timeFrame: Through Day 85
Secondary outcomes:
measure: The Proportion of Subjects With a Normal 24-hr UFC
timeFrame: Through Day 85
measure: The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value
timeFrame: Through Day 85
measure: The Proportion of Subjects With a Normal 24-hr UFC
timeFrame: Through Day 57 and Day 85
measure: The Proportion of Subjects With a Reduction in 24-hr UFC of ≥ 50% Relative to Their Baseline Value
timeFrame: Through Day 57 and Day 85
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Cleveland
country: United States
facility: The Cleveland Clinic Foundation
state: Ohio
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Middlesbrough
country: United Kingdom
facility: The James Cook University Hospital
Study officials:
affiliation: Medical College of Wisconsin
name: James Findling, MD
role: PRINCIPAL_INVESTIGATOR
References: