A Phase 2 interventional study of Belzutifan in Von Hippel Lindau, Von Hippel Lindau Disease and Von Hippel Lindau-Deficient Clear Cell Renal Cell Carcinoma, sponsored by José Claudio Casali da Rocha. Recruiting at 1 site in Brazil. Open to participants aged 14 Years and older. Per ClinicalTrials.gov, last updated 2025-09-11.
Sponsored by José Claudio Casali da Rocha · Phase 2, Interventional, and Treatment
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.
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
55 studies on the registry are indexed under von Hippel-Lindau Disease; 23 are open to participants now.
This study's planned enrollment of 100 is above the median of 28 across 31 interventional studies indexed under von Hippel-Lindau Disease.
Browse von Hippel-Lindau Disease studies →This is the only study on the registry with José Claudio Casali da Rocha as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
RECIST
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients with von Hippel-Lindau Syndrome presenting with lesions or neoplasms requiring treatment with oral Belzutifan (WELIREG™)
Drug: Belzutifan
Patients 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.
Time frame: From enrollment and initiation of treatment until the earliest of either documented disease progression or death from any cause, with follow-up of up to 104 weeks.
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.
Time frame: Baseline to 104 weeks
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.
Time frame: Baseline to 104 weeks.
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.
Time frame: Time Frame: Baseline to 104 weeks
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.
Time frame: Baseline to 104 weeks
Blood Transfusion Requirement
Number of participants requiring at least one blood transfusion during treatment with belzutifan. Unit of Measure: Percentage of participants.
Time frame: Baseline to 104 weeks
Patient-Reported Quality of Life (EORTC QLQ-C30)
Change in health-related quality of life scores measured by the EORTC QLQ-C30: The European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30, version 3.0, at baseline, months 1, 3, 6, 12, and annually thereafter. It consists of 28 items evaluating symptom burden and functional status, each scored on a 4-point Likert scale (1 = "Not at all," 2 = "A little," 3 = "Quite a bit," 4 = "Very much"). In addition, two items assess overall health and overall quality of life, each rated on a 7-point scale (1 = "Very poor" to 7 = "Excellent") All treatment modifications-including interruptions, dose reductions, or discontinuations-will be recorded throughout the study. Unit of Measure: Score from 0 to 100 (higher score indicates better quality of life).
Time frame: Baseline to 24 months
Pharmacoeconomic Evaluation of Belzutifan Therapy in Individuals with von Hippel-Lindau Syndrome
The pharmacoeconomic analysis will be conducted in the same cohort of participants treated with belzutifan. Retrospective data on direct medical costs, expressed in Brazilian Reais (R$), will be collected, including expenses related to medications, surgical procedures, and hospital admissions. Indirect costs, particularly those related to productivity losses, will also be assessed and quantified as a percentage reduction compared with healthy employees or as hours/days lost due to illness or treatment-related absenteeism during the preceding five years. All cost data will be analyzed on a per-patient-per-month (PPPM) basis, stratified by defined time intervals. Cost-effectiveness will be evaluated by comparing healthcare costs before and after belzutifan initiation, accounting for changes in procedure frequency and overall resource utilization. Unit of Measure: Brazilian Reais (R$) per PPPM.
Time frame: Retrospective analysis of the 5 years prior to belzutifan initiation and prospective follow-up for up to 2 years after treatment initiation.
Plan to share: Yes — The investigators plan to share anonymized clinical and laboratorial data, outcomes, side effects, pharmacogenomic information, and their correlation with treatment. This will enable collaboration with other researchers to combine findings and enhance statistical power.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report, Analytic code
Time frame: At any time of the study as the results are analyzed, publicly presented and published
Access criteria: Data will be shared after Transfer Data Agreement between Principal Investigators and institutions, based on new bilateral approval on Ethics and Research Committees of both involved oncology centers.
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Studies of the drug tested here: Belzutifan