An experimental study of Belzutifan impact on catecholamine metabolism
Research type
Research Study
Full title
An experimental pilot study to investigate changes in catecholamine synthesis and metabolism in patients with molecularly profiled phaeochromocytoma and paraganglioma taking Belzutifan
IRAS ID
362095
Contact name
Ruth Casey
Contact email
Sponsor organisation
Cambridge University Hospital NHS Foundation Trust and University of Cambridge (joint sponsors)
Duration of Study in the UK
1 years, 0 months, 7 days
Research summary
Phaeochromocytomas and paragangliomas (PPGL) are rare hormone producing tumours. These tumours can produce excess levels of adrenaline through a metabolic pathway in the tumour cells that depend on a precursor amino acid called tyrosine and a rate limiting enzyme in the pathway called tyrosine hydroxylase. Recently, a new drug called Belzutifan, licensed as an anti-cancer for patients with a hereditary cancer syndrome called called Von Hippel Lindau, has demonstrated an impact on circulating adrenaline levels in a single patient with PPGL. This suggests that Belzutifan may have an independant impact on the metabolism of catecholamines. This study aims to investigate the impact that Belzutifan has on catecholamine metabolism by investigating changes in the level of an amino acid that starts the pathway of catecholamine metabolism (tyrosine). This change in tyrosine will be measured before and daily for five days in participants with adrenaline producing PPGL recruited to take Belzutifan 120mg daily for five days. This pilot experimental study should help identify if the physiology of catecholamine metabolism is altered by the drug Belzutifan.
REC name
London - Camden & Kings Cross Research Ethics Committee
REC reference
25/PR/1644
Date of REC Opinion
22 Jan 2026
REC opinion
Further Information Favourable Opinion