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

Phase 2

Completed

• Conditions: Congenital Adrenal Hyperplasia
• Interventions: Drug: ATR-101

• Registration Number: NCT02804178
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-05-18
• Study First Submitted: 2016-06-01
• Study First Submitted QC: 2016-06-14
• Study First Posted: 2016-06-17
• Primary Completion: 2017-08-17
• Study Completion: 2017-08-17
• Disposition First Submitted: 2019-10-16
• Disposition First Submitted QC: 2020-02-14
• Disposition First Posted: 2020-02-21
• Results First Submitted: 2021-01-27
• Results First Submitted QC: 2021-01-27
• Status Verified: 2021-02
• Last Update Submitted: 2021-02-16
• Results First Posted: 2021-02-17
• Last Update Posted: 2021-03-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

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

Trial Locations

Locations (6)

Mayo Clinic - Rochester; 🇺🇸 Rochester, Minnesota, United States

The Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

National Institutes of Health Clinical Center; 🇺🇸 Bethesda, Maryland, United States

University of Michigan; 🇺🇸 Ann Arbor, Michigan, United States

Johns Hopkins University; 🇺🇸 Baltimore, Maryland, United States

The University of Oklahoma - Tulsa Schusterman Center; 🇺🇸 Tulsa, Oklahoma, United States