Studies belzutifan in people with von Hippel-Lindau syndrome in real-world care
Tests belzutifan in people aged 14 and older with von Hippel-Lindau syndrome.
Plain-language title generated from the registry record
Registry title: Real-World Effectiveness and Pharmacogenetics of Belzutifan in VHL Syndrome: The BELIEVE-VHL Trial
Key facts
- Where
- São Paulo, BrazilSee the site
- Phase
- Phase 2
Where in Brazil
- AC Camargo Cancer CenterSão Paulo, BrazilTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- José Claudio Casali da Rocha
- Trial code
- NCT07167329
- Record updated
- 11 September 2025
- Original description
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.Read full original descriptionShow less
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
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.
Inclusion: 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: Radiotherapy administered within 4 weeks prior to study enrollment; Major surgical procedure, including for VHL-related tumors, within 4 weeks prior to study enrollment, or immediate need for surgical intervention for tumor management; Malabsorption secondary to prior gastrointestinal surgery or active gastrointestinal disease; Current use of concomitant medications known to interact with belzutifan and significantly alter its bioavailability; Anticipated low adherence to or planned interruption of belzutifan therapy; Absence of a confirmed diagnosis of von Hippel-Lindau (VHL) syndrome; Known hypersensitivity or allergic reaction to belzutifan or any excipient in the formulation; History of severe or uncontrolled cardiovascular disease, including but not limited to unstable angina, myocardial infarction within the past 6 months, congestive heart failure requiring treatment, or uncontrolled hypertension; Active infectious diseases, including HIV, hepatitis B, or hepatitis C; Immunosuppressed status, whether due to underlying disease or ongoing therapy; History of significant bleeding disorders, including bleeding diathesis, thrombocytopenia, or coagulopathy; Age < 14 years; 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.