A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia

A Phase 2, Multicenter Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia


Lead Sponsor: Millendo Therapeutics, Inc.

Source Millendo Therapeutics, Inc.
Brief Summary

This is a Phase 2 multicenter, single-blind, multiple dose study to evaluate the safety and efficacy of orally administered ATR-101 in subjects with classic congenital adrenal hyperplasia (CAH). Treatment duration will range from a minimum of approximately 2 months to 6 months per subject. A subject may receive a minimum of one dose level or up to a maximum of 5 dose levels, in sequentially increasing dose strengths. Each dose level will last 28 days.

Overall Status Completed
Start Date 2016-05-18
Completion Date 2017-08-17
Primary Completion Date 2017-08-17
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101 Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.
Enrollment 10

Intervention Type: Drug

Intervention Name: ATR-101

Description: 125-1000 mg twice per week

Arm Group Label: ATR-101

Other Name: Nevanimibe hydrochloride



Inclusion Criteria: - Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on: Documented genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or historical documentation of elevated 17-hydroxyprogesterone - Biochemical marker of disease status of 17-hydroxyprogesterone ≥ 4 times the upper limit of normal - Chronic glucocorticoid replacement therapy for at least 6 consecutive months - Stable glucocorticoid and mineralocorticoid regimen for at least 1 month Exclusion Criteria: - Non-classic CAH - Other causes of adrenal insufficiency - Surgery within the previous 3 months prior to screening or planned surgery during study participation - History of active cancer requiring medical or surgical therapy within the past 6 months



Minimum Age:

18 Years

Maximum Age:

80 Years

Healthy Volunteers:


Johns Hopkins University | Baltimore, Maryland, 21287, United States
National Institutes of Health Clinical Center | Bethesda, Maryland, 20892, United States
University of Michigan | Ann Arbor, Michigan, 48109, United States
Mayo Clinic - Rochester | Rochester, Minnesota, 55905, United States
The University of Oklahoma - Tulsa Schusterman Center | Tulsa, Oklahoma, 74135, United States
The Children's Hospital of Philadelphia | Philadelphia, Pennsylvania, 10021, United States
Location Countries

United States

Verification Date


Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: ATR-101

Type: Experimental

Description: Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.

Patient Data No
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Intervention Model Description: Single treatment arm with up to 5 planned dosing periods with escalating doses. Subjects were dosed with each dose level for 2 weeks, followed by 2 weeks of matching placebo (subjects blinded).

Primary Purpose: Treatment

Masking: None (Open Label)

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