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

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

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	14 Years and Over
Gender	All

More Inclusion & Exclusion 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 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.

- 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.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT07167329
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 2
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	José Claudio Casali da Rocha
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	N/A
Principal Investigator Affiliation	N/A
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Other
Overall Status	Recruiting
Countries	Brazil
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	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

Additional Details

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.

Arms & Interventions

Arms

Experimental: Oral Belzutifan (WELIREG™)

Patients with von Hippel-Lindau Syndrome presenting with lesions or neoplasms requiring treatment with oral Belzutifan (WELIREG™)

Interventions

Drug: - Belzutifan

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

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.