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.
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.
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.
Single group
Belzutifan
DrugPatients with Von Hippel-Lindau Syndrome presenting with lesions or neoplasms requiring treatment with oral Belzutifan.
Objective Tumor Response per RECIST 1.1
Evaluation of target lesion size according to RECIST 1.1 criteria at baseline, weeks 12, 24, 52, and annually, up to 104 weeks. Imaging modalities will include MRI for solid tumors, 68Ga-DOTATATE PET for pancreatic neuroendocrine tumors (PNET), and retinal fluorescein angiography (FA) for retinal lesions. Unit of Measure: Percentage of participants with objective response.
Incidence of Anemia (per CTCAE v5.0)
Number of participants developing grade ≥2 anemia during treatment with belzutifan, evaluated according to CTCAE version 5.0. Correlate hematologic outcomes with tumor response (RECIST 1.1) and lesion dynamics to identify potential predictive biomarkers. Unit of Measure: Percentage of participants.
Mean Hemoglobin Level Over Time
Longitudinal change in hemoglobin levels (g/dL) at months 1, 3, 6, 12, and annually thereafter. Unit of Measure: g/dL.
Erythropoietin Levels Over Time
Longitudinal change in endogenous erythropoietin levels (mIU/mL) at months 1, 3, 6, 12, and annually thereafter. Unit of Measure: mIU/mL.
Requirement for Erythropoietin Supplementation
Proportion of participants requiring subcutaneous erythropoietin supplementation for symptomatic or grade ≥3 anemia, including dose, frequency, and duration. Unit of Measure: Percentage of participants.
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José Claudio Casali da Rocha
Lead sponsor
AC Camargo Cancer Center
Sponsor institution
AC Camargo Cancer Center
Collaborator
