Phase 2/3 Study of Atumelnant in Paediatric Participants with Congenital Adrenal Hyperplasia
Research type
Research Study
Full title
A Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Atumelnant Treatment in Pediatric Participants with Congenital Adrenal Hyperplasia Including a Long-Term Extension
IRAS ID
1012729
Contact name
Etienne Dumont
Contact email
Sponsor organisation
Crinetics Pharmaceuticals, Inc.
Research summary
Crinetics Pharmaceuticals, Inc.® is conducting a clinical study of a drug (atumelnant) for the treatment of congenital adrenal hyperplasia (CAH).
The purpose of this study is to determine whether atumelnant is a safe, well tolerated, and effective treatment for children and adolescents with CAH.
CAH is a disease that affects the adrenal glands, which produce important hormones like cortisol (that regulates the body's response to stress) and androgens (which affect normal growth and development). Patients with CAH produce too little cortisol. As a result, the body will produce more of another hormone, called adrenocorticotropic hormone (ACTH).
Standard treatment for CAH includes glucocorticoids (also called “steroids”) which reduce ACTH and androgen levels in the body but are often responsible for many of the symptoms associated with CAH. Atumelnant has been shown to block excess ACTH hormone production (which is the cause of CAH) and improve patients’ quality of life.
This study is being done in 3 parts: Part A, Part B, and Part C. In Part A, participants will receive atumelnant by mouth once daily for 8 weeks. Part A will last up to 18 weeks. In Part B, participants will be randomly assigned to take atumelnant or placebo by mouth once daily for 28 weeks. There will be a 2 in 3 chance of receiving atumelnant. Participation in Part B will last up to 38 weeks.
Upon completion of Part A or Part B, participants may have the option to enroll into a long-term extension (Part C) where everyone will receive atumelnant for up to 5 years.
In this study, approximately 153 participants will be enrolled globally with approximately 15 participants in the UK.REC name
London - West London & GTAC Research Ethics Committee
REC reference
25/LO/0676
Date of REC Opinion
28 Nov 2025
REC opinion
Further Information Favourable Opinion