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

Status:	Completed
Conditions:	Endocrine, Hematology
Therapuetic Areas:	Endocrinology, Hematology
Healthy:	No
Age Range:	18 - 80
Updated:	4/17/2018
Start Date:	May 18, 2016
End Date:	August 17, 2017

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.

Inclusion Criteria:

- Documented historical diagnosis of classic CAH due to 21-OHD based on: Documented
genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or
historical documentation of elevated 17-OHP

- Biochemical marker of disease status of 17-OHP ≥ 4 X ULN

- 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

We found this trial at

sites

Children's Hospital of Philadelphia

South 34th Street
Philadelphia, Pennsylvania 19104

215-590-1000