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Not yet recruiting NCT07554508

Belzutifan VHL RCC HK Prospective Registry

— Observational VHL - Von Hippel-Lindau Syndrome

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: VHL - Von Hippel-Lindau Syndrome. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Hong Kong
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Prospective Registry for Belzutifan in the Treatment of Von Hippel-Lindau (VHL) Disease

Overview

This study aims to evaluate the Belzutifan's efficacy, drug safety, disease course, and the health-economic impact of the treatment to Von Hippel-Lindau (VHL) syndrome.

Detailed description

Adult patients diagnosed with VHL syndrome who choose to use belzutifan for their treatment will be identified and invited to participate in this registry. Clinical data, laboratory tests, imaging results and QOLs will be collected from subjects at baseline, every 3 months for the first year, then every 6 months until month 36 or cessation of the medication, whichever occurs first.

Primary outcome measures

  • Objective response rate [Time frame: Month 6 and Month 12]
Secondary outcome measures (3)
  • Radiographic response [Time frame: Month 6 and Month 12, Month 18, Month 24, Month 30 and Month 36]
  • Change in Quality of Life score [Time frame: Baseline, Month 6 and Month 12]
  • Health-economic impact of belzutifan [Time frame: Month 6, Month 12, Month,18, Month 24, Month 30, Month 36]

Eligibility criteria

Inclusion criteria

  • Adult patients (age 18 or above) with confirmed VHL-spectrum disease through clinical and/or genetic diagnosis.
  • The patient decided to use Belzutifan for their treatment.

Exclusion criteria

  • Tumour requiring immediate resection.
  • Pregnancy or unwillingness to use contraception if of childbearing potential.
  • Uncontrolled comorbidities (e.g., active infection, severe cardiopulmonary disease).

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Case-only

Study locations

Hong Kong · 1 center
  • Prince of Wales Hospital — Shatin

Publications

  • Zhou T, Guan H, Wang L, Zhang Y, Rui M, Ma A. Health-Related Quality of Life in Patients With Different Diseases Measured With the EQ-5D-5L: A Systematic Review. Front Public Health. 2021 Jun 29;9:675523. doi: 10.3389/fpubh.2021.675523. eCollection 2021. PMID 34268287
  • Linehan WM, Pinto CA, Liu Y, Choo K, Gautam R, Fox C, Roy A, Li M, Bosan R, Nielsen D, Ryan B, Blake Z, Reynolds K, Rompre-Brodeur A, Pinto PA, Vocke C, Gurram S, Ball MW, Perini R, Srinivasan R. Longitudinal Evaluation of Clear-cell Renal Cell Carcinoma in von Hippel-Lindau Disease. Eur Urol. 2025 Jul;88(1):56-63. doi: 10.1016/j.eururo.2025.03.002. Epub 2025 Apr 23. PMID 40274483
  • Louise M Binderup M, Smerdel M, Borgwadt L, Beck Nielsen SS, Madsen MG, Moller HU, Kiilgaard JF, Friis-Hansen L, Harbud V, Cortnum S, Owen H, Gimsing S, Friis Juhl HA, Munthe S, Geilswijk M, Rasmussen AK, Moldrup U, Graumann O, Donskov F, Gronbaek H, Stausbol-Gron B, Schaffalitzky de Muckadell O, Knigge U, Dam G, Wadt KA, Bogeskov L, Bagi P, Lund L, Stochholm K, Ousager LB, Sunde L. von Hippel-Lin PMID 35709961
  • Jonasch E, Donskov F, Iliopoulos O, Rathmell WK, Narayan VK, Maughan BL, Oudard S, Else T, Maranchie JK, Welsh SJ, Thamake S, Park EK, Perini RF, Linehan WM, Srinivasan R; MK-6482-004 Investigators. Belzutifan for Renal Cell Carcinoma in von Hippel-Lindau Disease. N Engl J Med. 2021 Nov 25;385(22):2036-2046. doi: 10.1056/NEJMoa2103425. PMID 34818478

Identifiers

NCT: NCT07554508 · CRE-2025.563

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗