Real-World Effectiveness and Pharmacogenetics of Belzutifan in VHL Syndrome: The BELIEVE-VHL Trial
Trial Parameters
Brief Summary
The BELIEVE-VHL Trial is a prospective real-life study designed to evaluate the therapeutic effects, benefits, and adverse effects of belzutifan, as well as the timing of treatment response and disease progression in patients with von Hippel-Lindau (VHL) syndrome.
Eligibility Criteria
Inclusion Criteria: * Age ≥ 14 years. * Clinical or genetic confirmation of von Hippel-Lindau (VHL) syndrome. * Presence of measurable or progressive VHL-associated tumors, as defined by RECIST 1.1 or disease-specific imaging criteria. * ECOG performance status of 0-2. * Adequate bone marrow, hepatic, and renal function as defined by laboratory reference values. * Ability to swallow oral medication. * Provision of written informed consent prior to enrollment. Exclusion Criteria: * Age \< 14 years. * Absence of a confirmed diagnosis of von Hippel-Lindau (VHL) syndrome. * Presence of an active malignancy outside the VHL tumor spectrum within the past 3 years, except for adequately treated basal or squamous cell carcinoma of the skin, cervical carcinoma in situ, or other malignancies considered cured for \>2 years. * Known hypersensitivity or allergic reaction to belzutifan or any excipient in the formulation. * History of severe or uncontrolled cardiovascular disease, including but not l