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

February 16, 2021 updated by: Millendo Therapeutics, Inc.

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

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 Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

10

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 78 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ATR-101
Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.
Drug: ATR-101
125-1000 mg twice per week
Other Names:
  • Nevanimibe hydrochloride

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Millendo Therapeutics, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 18, 2016

Primary Completion (Actual)

August 17, 2017

Study Completion (Actual)

August 17, 2017

Study Registration Dates

First Submitted

June 1, 2016

First Submitted That Met QC Criteria

June 14, 2016

First Posted (Estimate)

June 17, 2016

Study Record Updates

Last Update Posted (Actual)

March 10, 2021

Last Update Submitted That Met QC Criteria

February 16, 2021

Last Verified

February 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ATR-101-201

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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