A Phase 2 Study of Belzutifan (PT2977, MK-6482) for the Treatment of Von Hippel Lindau (VHL) Disease-Associated Renal Cell Carcinoma (RCC) (MK-6482-004)

To join this study, you need to have a confirmed diagnosis of Von Hippel-Lindau (VHL) disease that’s caused by a change in your VHL gene. You should also have at least one kidney tumour that can be measured during scans. Importantly, these kidney tumours shouldn't require immediate surgery – the study is for people where watchful waiting or non-immediate treatment is suitable. You might also have other VHL-related growths in other parts of your body, which is fine.

However, you won't be able to join if you've already had belzutifan or similar medicines (called HIF-2α inhibitors) before. You also can't have had any other systemic anti-cancer treatments (like chemotherapy or some other targeted therapies that affect the whole body) in the past. If your kidney tumour needs urgent surgery, or if scans show the cancer has spread to other distant parts of your body, you won't be able to take part.

If you join this study, you'll be taking the study medicine, belzutifan, by mouth every day. This treatment will continue for an extended period, potentially for several years, as long as it's helping you and you're not experiencing significant side effects. You'll need to have regular check-ups and scans to see how your kidney tumours are responding. These scans will happen about 12 weeks (roughly 3 months) after you start the medicine, and then every 12 weeks for at least three years. After this, scans will typically occur every 24 weeks (about 6 months), or more often if your doctors believe it's necessary. During these visits, the study team will also monitor any other growths related to your VHL disease.