Belzutifan VHL RCC HK Prospective Registry

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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: VHL - Von Hippel-Lindau 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.

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

• Study Contact: Name: David Ka-Wai LEUNG, FRCS, MBChB; Phone Number: 35053933; Email: davidleung@surgery.cuhk.edu.hk

Study Locations

• Hong Kong
  • Shatin, Hong Kong
    • Prince of Wales Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult patients diagnosed with VHL syndrome who choose to use belzutifan for their treatment

Description

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).

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Belzutifan Group; Subjects received Belzutifan

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective response rate	RECIST 1.1	Month 6 and Month 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Radiographic response	By RECIST 1.1	Month 6 and Month 12, Month 18, Month 24, Month 30 and Month 36
Health-economic impact of belzutifan	By per-patient emergence department and inpatient cost	Month 6, Month 12, Month,18, Month 24, Month 30, Month 36
Change in Quality of Life score	By EQ-5D-5L. Score range from 0-100.The higher the score the better quality of life	Baseline, Month 6 and Month 12

Collaborators and Investigators

• Sponsor: Chinese University of Hong Kong

Publications and helpful links

General Publications

• 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.
• 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.
• 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.
• 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-Lindau disease: Updated guideline for diagnosis and surveillance. Eur J Med Genet. 2022 Aug;65(8):104538. doi: 10.1016/j.ejmg.2022.104538. Epub 2022 Jun 13.

Study record dates

Study Major Dates

• Study Start (Estimated): May 1, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): March 31, 2029

Study Registration Dates

• First Submitted: April 21, 2026
• First Submitted That Met QC Criteria: April 21, 2026
• First Posted (Actual): April 28, 2026

Study Record Updates

• Last Update Posted (Actual): May 5, 2026
• Last Update Submitted That Met QC Criteria: April 29, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Ciliopathies; Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, Inborn; Congenital Abnormalities; Abnormalities, Multiple; Neurocutaneous Syndromes; Angiomatosis; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; von Hippel-Lindau Disease
• Other Study ID Numbers: CRE-2025.563

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No