Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants with CAH
Research type
Research Study
Full title
A 12-week, Phase 2 Open-label, Sequential Dose Cohort Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 Treatment in Participants with Congenital Adrenal Hyperplasia
IRAS ID
1007269
Contact name
Genevieve Shyffer
Contact email
Sponsor organisation
Crinetics Pharmaceuticals, Inc.
Research summary
Congenital Adrenal Hyperplasia, or CAH, is a medical term used to describe a group of rare diseases of the adrenal glands. The adrenal glands are located on top of the kidneys and produce hormones that control many important functions in the body. A hormone is a tiny chemical messenger that can “talk” to certain cells. The most common cause of CAH is a shortage in the production of an enzyme called 21-hydroxylase, or 21-OHD, resulting in less hormone than needed being produced (for example, cortisol and aldosterone). To compensate for this deficiency, other hormones, such as adrenocorticotropic hormone, or ACTH, increase. This type of CAH (known as classic CAH) affects about 1 in 15,000 people worldwide.
Current treatment of CAH involves either putting back or lowering hormones with medicines called glucocorticoids. Using glucocorticoids, it is difficult to strike the right balance between over and under treating CAH, which may result in unwanted side-effects. CRN04894, an investigational study drug, works by blocking the action of ACTH which reduces the amount of other hormones. The trial will study how safe CRN04894 is and how it works to lessen the effects of classic CAH. CRN04894 is an oral, once daily tablet.
The trial will study about 30 people with classic CAH in different countries. Those in the trial will be divided into 3 groups, which will determine the amount of CRN04894 they receive. Everyone will receive CRN04894 for up to 12 weeks with their current glucocorticoid replacement medicine. The total study time is up to 20 weeks. The tests you might have during the trial include, but are not limited to, physical examinations (doctor’s check-ups), blood and urine (pee) tests, ultrasound or MRIs, and filling out forms with questions. Crinetics Pharmaceuticals, Inc. is the study sponsor.REC name
North West - Liverpool Central Research Ethics Committee
REC reference
23/NW/0201
Date of REC Opinion
24 Aug 2023
REC opinion
Further Information Favourable Opinion