Completed Lungs & Breathing NIHR-supported project Heart, Stroke & Blood

An open-label, long-term extension study to evaluate safety and efficacy in participants treated with crn04894

Funder NIHR (part of DHSC)
Recipient organisation NIHR University College London Hospitals Biomedical Research Centre
NIHR support Recorded as supported by this research centre
Period Feb 2025 — Oct 2025

In plain English

AI plain-English summary

A daily tablet called CRN04894 is being tested in 150 people with classic Congenital Adrenal Hyperplasia (CAH) across multiple countries to see if it is safe to take for up to 26 months. CAH is a rare genetic condition where the adrenal glands cannot produce enough of the hormones cortisol and aldosterone. The body tries to compensate by overproducing another hormone, ACTH, which causes harmful side effects. Current treatment with glucocorticoids is a difficult balancing act—too much or too little causes unwanted problems. CRN04894 works by blocking ACTH directly, potentially offering a more targeted way to control the condition without the constant risk of over- or under-treatment. If the trial shows CRN04894 is safe and effective long-term, it could replace the imprecise steroid regimens that people with classic CAH currently rely on. This would mean fewer side effects from overtreatment, such as weight gain and bone thinning, and fewer dangerous adrenal crises from undertreatment. The drug is an oral tablet taken once daily, which could simplify daily management for patients. The study is sponsored by Crinetics Pharmaceuticals.

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.

Researchers

Umasuthan Srirangalingam (Principal Investigator)

Related Research

Grants with similar aims, by meaning.

CRN-04894-09 (OLE) - An open-label, long-term extension study to evaluate safety and efficacy in participants treated with CRN04894
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
A 12-week, phase 2 open-label, sequential dose cohort study to evaluate the safety, efficacy, and pharmacokinetics of crn04894 treatment in participants with congential adrenal hyperplasia
CRN04894-03 - 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
NBI-74788-CAH3003 - A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment

Original classification

Cancer

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