Trial · NCT07167329
Real-World Effectiveness and Pharmacogenetics of Belzutifan in VHL Syndrome: The BELIEVE-VHL Trial
NCT07167329, Phase 2, Recruiting, José Claudio Casali da Rocha
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Real-World Effectiveness and Pharmacogenetics of Belzutifan in VHL Syndrome: The BELIEVE-VHL Trial. Trials, record NCT07167329. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07167329
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Real-World Effectiveness and Pharmacogenetics of Belzutifan in VHL Syndrome: The BELIEVE-VHL Trial
- NCT number
- NCT07167329
- Status
- Recruiting
- Phases
- Phase 2
- Enrollment
- 100 participants, estimated
- Lead sponsor
- José Claudio Casali da Rocha
- Conditions
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
- Interventions
- Belzutifan
- First posted
- 2025-09-11
- Last updated
- 2025-09-11
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Objective Tumor Response per RECIST 1.1 | 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. | 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. |
| Secondary | Incidence of Anemia (per CTCAE v5.0) | Baseline to 104 weeks | 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. |
| Secondary | Mean Hemoglobin Level Over Time | Baseline to 104 weeks. | Longitudinal change in hemoglobin levels (g/dL) at months 1, 3, 6, 12, and annually thereafter. Unit of Measure: g/dL. |
| Secondary | Erythropoietin Levels Over Time | Time Frame: Baseline to 104 weeks | Longitudinal change in endogenous erythropoietin levels (mIU/mL) at months 1, 3, 6, 12, and annually thereafter. Unit of Measure: mIU/mL. |
| Secondary | Requirement for Erythropoietin Supplementation | Baseline to 104 weeks | Proportion of participants requiring subcutaneous erythropoietin supplementation for symptomatic or grade ≥3 anemia, including dose, frequency, and duration. Unit of Measure: Percentage of participants. |
| Secondary | Blood Transfusion Requirement | Baseline to 104 weeks | Number of participants requiring at least one blood transfusion during treatment with belzutifan. Unit of Measure: Percentage of participants. |
| Secondary | Patient-Reported Quality of Life (EORTC QLQ-C30) | Baseline to 24 months | 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). |
| Secondary | Pharmacoeconomic Evaluation of Belzutifan Therapy in Individuals with von Hippel-Lindau Syndrome | Retrospective analysis of the 5 years prior to belzutifan initiation and prospective follow-up for up to 2 years after treatment initiation. | 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. |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers16
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT07167329
GET https://conductscience.org/api/trials/v1/NCT07167329/registered