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

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

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.

Conditions9

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