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

Millendo Therapeutics

Status and phase: Terminated; Phase 2

Conditions: Cushing Syndrome

Treatments: Drug: Placebo; Drug: ATR-101

Study type: Interventional

Funder types: Industry

Identifiers

NCT03053271

ATR-101-301

Details and patient eligibility

About

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.

Enrollment: 4 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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4 participants in 2 patient groups, including a placebo group

ATR-101

Experimental group

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.

Treatment:

Drug: ATR-101

Placebo

Placebo Comparator group

Description:

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.

Treatment:

Drug: Placebo