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Real-World Effectiveness and Pharmacogenetics of Belzutifan in VHL Syndrome: The BELIEVE-VHL Trial

Testing Belzutifan's effectiveness for VHL Syndrome in real-world settings.

Recruiting
14 years and older
All
Phase 2

The BELIEVE-VHL Trial is a study to see how well a medicine called belzutifan works for people with von Hippel-Lindau (VHL) syndrome. VHL syndrome is a condition where people develop tumors in different parts of their bodies. The study looks at how the medicine helps, any side effects, and how quickly it works or if the disease gets worse. It also checks if certain factors affect how well the medicine works and if extra medicine is needed for low red blood cells, called anemia.

  • Study lasts six months, checking blood and health regularly.
  • Participants must be 14 or older and have a confirmed VHL diagnosis.
  • Risks include possible side effects from belzutifan, such as fatigue or anemia.

To join, you must be able to take pills and not have any other serious health issues or allergies to the medicine. The study is important because it helps understand how belzutifan can improve life for people with VHL and its cost-effectiveness. If you have a VHL diagnosis and meet the criteria, participating could help advance treatment knowledge.

Study details
    Von Hippel Lindau
    Von Hippel Lindau Disease
    Von Hippel Lindau-Deficient Clear Cell Renal Cell Carcinoma
    Hemangioblastoma (HB) of the Central Nervous System (CNS)
    PNET
    Retinal Angiomatous Proliferation
    Pheochromocytoma/Paraganglioma
    Endolymphatic Sac Tumor

NCT07167329

José Claudio Casali da Rocha

31 July 2026

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