UCLH patient first in world to receive a new investigational drug in a phase 1 trial

A UCLH patient with a rare hormonal condition is the first in the world to receive a new investigational drug in a first-in-human trial.

The investigational drug was given as part of a phase 1 trial (meaning an early phase trial) which aims to assess the safety and tolerability of the investigational drug in people with congenital adrenal hyperplasia (CAH).

The first dose was given at the NIHR UCLH Clinical Research Facility (CRF) by team led Dr Umasuthan Srirangalingam.

UCLH has one of the largest cohorts of CAH patients in the UK, providing highly specialised multi-disciplinary care for this group. Around 1 in 10,000 to 20,000 people have CAH, in which the adrenal glands are not able to make the hormones cortisol and aldosterone. Affected patients make excess male hormones such as testosterone. This can cause a number of problems, especially for female patients, including excess hair growth, acne and infertility.[1]

The only treatment available at present for patients with CAH is to replace the two steroid hormones they lack. Replacement therapy, may be needed from birth, often is lifelong, and sometimes, despite high doses of steroids, fails to control patient symptoms. Patients can become profoundly unwell without enough steroid or suffer from the consequences of too much steroid.[2]

Researchers at the CRF are now looking at a new investigational drug developed by pharmaceutical company Lundbeck called Lu AG13909.

Lu AG13909 is a monoclonal antibody that targets a hormone called ‘ACTH’. Lu AG13909 is designed to block the signalling of ACTH in the adrenal gland.

This phase 1 study aims to recruit 12 patients. Patients will be given the investigational drug via an intravenous infusion and researchers will monitor the effects of the drug.

Dr Srirangalingam said: “We have been fortunate to be the first team worldwide to deliver this new investigational drug to patients as part of this trial. We now have the opportunity to evaluate if it is efficacious and generally well tolerated. This kind of trial not only requires a dedicated group of patients who volunteer to take part but also a critical infrastructure to set-up and run them, and we have been fortunate to be able to access the facilities and expertise at the CRF and I’d like to thank both.”

Professor Bryan Williams, Director of Research at UCLH said: “I am pleased to see our patients, once again, being the first in the world to get access to potential new medicines. It is particularly exciting because this shows that we can lead in many other areas of clinical research that we want to develop, where we can align outstanding clinical expertise with our early phase research capability at UCLH.”

References

1. Merke DP, Auchus RJ. Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency. N Engl J Med 2020;383(13):1248-61. doi: 10.1056/NEJMra1909786 [published Online First: 2020/09/24]

2. Ng SM, Stepien KM, Krishan A. Glucocorticoid replacement regimens for treating congenital adrenal hyperplasia. Cochrane Database Syst Rev 2020;3(3):Cd012517. doi: 10.1002/14651858.CD012517.pub2 [published Online First: 2020/03/20]