NCT07167329

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

The BELIEVE-VHL Trial: A Real-world Longitudinal Study on Belzutifan's Effectiveness, Pharmacogenetics, and Pharmacoeconomics in Von Hippel-Lindau (VHL) Syndrome Using the HIF2α Inhibitor Belzutifan

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 100 (estimated)
• Sponsor: José Claudio Casali da Rocha · Academic / Other
• Sex: All
• Age: 14 Years
• Healthy volunteers: Not accepted

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.

Detailed description

PRIMARY OBJECTIVE: To evaluate the therapeutic effects, benefits, and adverse effects associated with belzutifan treatment, as well as the timing of treatment response and/or disease progression. SECONDARY OBJECTIVES 1. To evaluate the association of host intrinsic factors with toxicity and treatment response in a Brazilian cohort of patients with von Hippel-Lindau syndrome treated with belzutifan. 2. To assess hemoglobin and erythropoietin levels during the first six months of treatment, and to document the need for subcutaneous erythropoietin supplementation in patients who develop grade 2-3 anemia, fatigue, or hypoxia. 3. To evaluate the potential impact of erythropoietin supplementation on tumor growth during belzutifan treatment. 4. To assess health-related quality of life and patient perceptions regarding VHL syndrome using validated questionnaires and instruments. 5. To conduct a pharmacoeconomic analysis in the cohort of patients with access to belzutifan, assessing its impact on healthcare costs compared to the natural history of the disease.

Conditions

• 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

Interventions

Type	Name	Description
DRUG	Belzutifan	Patients with Von Hippel-Lindau Syndrome presenting with lesions or neoplasms requiring treatment with oral Belzutifan.

Timeline

• Start date: 2024-01-01
• Primary completion: 2026-02-01
• Completion: 2030-01-01
• First posted: 2025-09-11
• Last updated: 2025-09-11

Locations

1 site across 1 country: Brazil