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

Overview

About this study

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.

 

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

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

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Alice Chang, M.D.

Closed for enrollment

More information

Publications

Publications are currently not available

Additional contact information

Non-cancer trials contact form

Phone: 800-664-4542 (toll-free)

International patient clinical studies questions