Phase 3 Study of Atumelnant in Adult Participants with Classic Congenital Adrenal Hyperplasia
Research type
Research Study
Full title
A Randomized, Double-blind, Multicenter, Placebo-controlled Study to Evaluate the Safety and Efficacy of Atumelnant in Adult Participants with Classic Congenital Adrenal Hyperplasia
IRAS ID
1012212
Contact name
Alejandro Ayala
Contact email
Sponsor organisation
Crinetics Pharmaceuticals, Inc.
Eudract number
2024-519579-24
Research summary
Crinetics Pharmaceuticals® 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 adults 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 (GC) 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.
In this study, adults with CAH will be randomly assigned to take atumelnant or placebo by mouth once daily. There will be a 2 in 3 chance of receiving atumelnant.
Participants will be enrolled in the study for 42 weeks, which includes 32 weeks of treatment.
During this study, all participants will reduce their current GC treatment. This reduction may cause a side effect called “adrenal insufficiency” due to the lack of cortisol.
To reduce the chances of adrenal insufficiency, participants will have weekly calls with the study doctor while GCs are reduced to check how they are feeling. In addition, participants will be educated on signs and symptoms of adrenal insufficiency and how to report them.
Upon completion of the treatment, participants may have the option to enroll into a separate 2-year extension study where everyone will receive atumelnant.
Approximately 150 participants will be enrolled globally with approximately 15 in the UK.REC name
North West - Haydock Research Ethics Committee
REC reference
25/NW/0267
Date of REC Opinion
24 Oct 2025
REC opinion
Further Information Favourable Opinion