Pilot Study Evaluating the Effects of Belzutifan on Catecholamine Metabolism in Adults with Pheochromocytoma and Paraganglioma

What this Trial Is About

Researchers are studying the impact of a drug called Belzutifan on the production and metabolism of adrenaline and noradrenaline, collectively known as catecholamines, in adults with pheochromocytoma or paraganglioma. This pilot Phase 2 study aims to identify changes in catecholamine synthesis by measuring tyrosine, which starts the catecholamine metabolism process, before, during, and after treatment. The study will include 12 patients treated at a national referral center. Participants will take Belzutifan 120mg daily for five days as part of a 14-day study protocol. Blood samples will be collected to measure plasma tyrosine and metanephrines at baseline, during treatment, and after treatment up to day 14. Genetic testing results from routine care will be recorded, and tumor tissue may be sequenced to investigate genetic changes affecting drug impact. Throughout the study, patients will visit the clinic daily for seven days and again on days 10 and 14. Researchers will monitor blood pressure, heart rate, blood counts, and liver function tests, and record any symptoms and medication changes. The main outcome measures are tyrosine and plasma metanephrine levels, assessed to understand how Belzutifan affects catecholamine metabolism and safety over the 14-day period.

An Experimental Study of Belzutifan Impact on Catecholamine Metabolism