Recipient organisationNIHR Sheffield Clinical Research Facility
NIHR supportRecorded as supported by this research centre
PeriodFeb 2025 — Dec 2027
In plain English
AI plain-English summary
Around 150 people with classic congenital adrenal hyperplasia will take an experimental once-daily tablet called CRN04894 for up to 26 months to test its long-term safety. This matters because current treatment for CAH—replacing or suppressing hormones with glucocorticoids—is a difficult balancing act. Too much or too little medication causes serious side effects, and many patients never achieve stable control. CRN04894 takes a different approach: it blocks the hormone ACTH, which is overproduced in CAH and drives the hormone imbalance. By reducing ACTH activity directly, the drug may allow patients to lower their glucocorticoid doses or avoid the worst fluctuations. If the trial shows CRN04894 is safe over many months, it could offer a fundamentally new treatment option for a rare disease that affects about 1 in 15,000 people worldwide. The drug is an oral tablet, not an injection, which would make long-term management simpler for patients. The study is sponsored by Crinetics Pharmaceuticals and includes physical exams, blood and urine tests, and imaging.
View original technical description
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 level of other hormones. The trial will aim to find out the the long-term safety of CRN04894. CRN04894 is an oral, once daily tablet.The trial will study about 150 people with classic CAH in different countries. There are two groups of participants: Those currently taking CRN04894 in group 1, and those not currently taking CRN04894 in group 2. The total study time is up to 26 months. 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.
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