NCT07554508

Brief Summary

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.

Trial Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
100

participants targeted

Target at P50-P75 for all trials

Timeline
36mo left

Started May 2026

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress1%
May 2026Mar 2029

First Submitted

Initial submission to the registry

April 21, 2026

Completed
7 days until next milestone

First Posted

Study publicly available on registry

April 28, 2026

Completed
3 days until next milestone

Study Start

First participant enrolled

May 1, 2026

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2028

Expected
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 31, 2029

Last Updated

May 5, 2026

Status Verified

April 1, 2026

Enrollment Period

2.7 years

First QC Date

April 21, 2026

Last Update Submit

April 29, 2026

Conditions

VHL - Von Hippel-Lindau Syndrome

Outcome Measures

Primary Outcomes (1)

  • Objective response rate

    RECIST 1.1

    Month 6 and Month 12

Secondary Outcomes (3)

  • Radiographic response

    Month 6 and Month 12, Month 18, Month 24, Month 30 and Month 36

  • Change in Quality of Life score

    Baseline, Month 6 and Month 12

  • Health-economic impact of belzutifan

    Month 6, Month 12, Month,18, Month 24, Month 30, Month 36

Study Arms (1)

Belzutifan Group

Subjects received Belzutifan

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

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

You may qualify if:

  • 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.

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Prince of Wales Hospital

Shatin, Hong Kong

Location

Related Publications (4)

  • 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: 34268287BACKGROUND

  • 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: 40274483BACKGROUND

  • 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.

    PMID: 35709961BACKGROUND

  • 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: 34818478BACKGROUND

MeSH Terms

Conditions

von Hippel-Lindau Disease

Condition Hierarchy (Ancestors)

Neurocutaneous SyndromesNervous System DiseasesAngiomatosisVascular DiseasesCardiovascular DiseasesCiliopathiesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, Inborn

Central Study Contacts

David Ka-Wai LEUNG, FRCS, MBChB

CONTACT

35053933davidleung@surgery.cuhk.edu.hk

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

April 21, 2026

First Posted

April 28, 2026

Study Start

May 1, 2026

Primary Completion (Estimated)

December 31, 2028

Study Completion (Estimated)

March 31, 2029

Last Updated

May 5, 2026

Record last verified: 2026-04

Locations

HK(1)