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NCT Number: NCT07554508

Belzutifan VHL RCC HK Prospective Registry

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.

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Key information

Conditions

VHL - Von Hippel-Lindau Syndrome Abnormalities, Multiple Angiomatosis Cardiovascular Diseases Ciliopathies Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Nervous System Diseases Neurocutaneous Syndromes Vascular Diseases von Hippel-Lindau Disease

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Prince of Wales Hospital

Shatin, Hong Kong

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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).

Treatment and study plan

Primary outcomes

  1. Objective response rate

    Time frame: Month 6 and Month 12

    RECIST 1.1

Secondary outcomes

  1. Radiographic response

    Time frame: Month 6 and Month 12, Month 18, Month 24, Month 30 and Month 36

    By RECIST 1.1

  2. Change in Quality of Life score

    Time frame: Baseline, Month 6 and Month 12

    By EQ-5D-5L. Score range from 0-100.The higher the score the better quality of life

  3. Health-economic impact of belzutifan

    Time frame: Month 6, Month 12, Month,18, Month 24, Month 30, Month 36

    By per-patient emergence department and inpatient cost

Study contacts

Contact information is provided by the study sponsor or research team.

David Ka-Wai LEUNG, FRCS, MBChB

CONTACT

[email protected]

35053933

Sponsors and collaborators

Lead sponsor

Chinese University of Hong Kong

Other

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2028
Study completion
2029
First posted
Apr 28, 2026
Registry last updated
May 5, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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