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

Congenital Adrenal Hyperplasia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02804178
• Other study ID #: ATR-101-201
• Status: Completed
• Phase: Phase 2
• Start date: May 18, 2016
• Est. completion date: August 17, 2017

Study information

• Verified date: February 2021
• Source: Millendo Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: August 17, 2017
• Est. primary completion date: August 17, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Adrenal Hyperplasia, Congenital
• Adrenocortical Hyperfunction
• Adrenogenital Syndrome
• Congenital Adrenal Hyperplasia
• Hyperplasia

Intervention

Drug:
• ATR-101
125-1000 mg twice per week

Locations

Country	Name	City	State
United States	University of Michigan	Ann Arbor	Michigan
United States	Johns Hopkins University	Baltimore	Maryland
United States	National Institutes of Health Clinical Center	Bethesda	Maryland
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Mayo Clinic - Rochester	Rochester	Minnesota
United States	The University of Oklahoma - Tulsa Schusterman Center	Tulsa	Oklahoma

Sponsors (1)

Lead Sponsor	Collaborator
Millendo Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.