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

Millendo Therapeutics, Inc.10 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Congenital Adrenal Hyperplasia

Interventions

ATR-101DRUG

125-1000 mg twice per week

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

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

Locations (6)

Johns Hopkins University

Baltimore, Maryland, United States

National Institutes of Health Clinical Center

Bethesda, Maryland, United States

University of Michigan

Ann Arbor, Michigan, United States

Mayo Clinic - Rochester

Rochester, Minnesota, United States

Outcomes

Primary Outcomes

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

17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level.

Time frame: Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.

Data from ClinicalTrials.gov

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