A UCLH patient is the first person in the UK to receive a new investigational drug being tested for a disorder caused by genetic mutations, called classic congenital adrenal hyperplasia (CAH).
CAH, which occurs in approximately one in every 10-15,000 people worldwide, impairs cortisol synthesis in the adrenal gland. When cortisol – the hormone that regulates the body’s response to disease or stress – is impaired, it results in persistently high levels of a hormone known as ACTH which then results in the build-up of other hormones produced by the adrenal gland, collectively known as androgens.
To stay healthy, people with CAH must take daily life-long medication. At present, the only approved medications for people with CAH are two forms of steroids, including glucocorticoids. Glucocorticoids are powerful medications which allow the body to respond to stress and reduce inflammation. However, prolonged administration of high doses of glucocorticoids may result in significant long-term safety concerns for patients.
The investigational drug being tested in this global study is called CRN04894. CRN04894 blocks the action of the ACTH hormone. In turn, this can help reduce the levels of unwanted androgens that build up in CAH. If successful in studies and approved by regulatory agencies, it is hoped this drug will be a set a new standard of treatment for CAH, where there is currently a large unmet need.
The primary goals of this study are to find out how safe and effective CRN04894 is at different doses and also to determine if the drug produces the expected effect in the body by reducing excess adrenal androgens caused by CAH.
Dr Umasuthan Srirangalingam, Consultant in Endocrinology & Diabetes, leading the study at UCLH, said that “steroid treatment for people with CAH is essential but it can be associated with several side-effects in the long term. Ultimately, CRN04894 may allow us to reduce the amount of glucocorticoids administered while helping maintain control of adrenal androgens. It is crucially important that we help to assess how effective these new treatments are in the hope that they can be introduced to the wider CAH community.”
The study is being run at UCLH at the NIHR UCLH Clinical Research Facility. It is also taking place at 3 other sites in the UK and further sites across 7 countries.
