Belzutifan for VHL-associated tumors: patient registry
Official title: A Prospective Registry for Belzutifan in the Treatment of Von Hippel-Lindau (VHL) Disease
Run by Chinese University of Hong Kong · NCT07554508 · Data updated 2026-11-12
This registry collects real-world data on adults with VHL who choose belzutifan to see how well it works and how safe it is over time.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 100 (estimated) |
| Sponsor | Chinese University of Hong Kong Academic / other |
| Locations | 1 site (Shatin) |
| Status | Recruiting |
| Started | December 22, 2025 |
| Main results expected | December 31, 2028 |
| Study ends | March 31, 2029 |
| Registry record updated | October 22, 2026 |
| Trial ID | NCT07554508 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Adults (18+) with a confirmed VHL-spectrum diagnosis who choose to use belzutifan for their treatment and consent to participate.
✕Probably not a fit
Patients who do not receive belzutifan or who discontinue the drug early may have limited benefit from participation.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational registry follows adults with Von Hippel-Lindau syndrome who opt to use belzutifan. Data are collected at baseline, every 3 months for the first year, and every 6 months thereafter up to 36 months or until stopping the medication. Information includes clinical status, laboratory results, imaging findings, and quality of life to understand treatment patterns, safety, disease course, and economic impact in routine care. No intervention beyond standard-of-care belzutifan use is required, and participants are identified through the collaborating center in Hong Kong. The study aims to provide real-world evidence to inform clinical practice and health-system decisions.
Why it matters
Potential benefit: If successful, the registry could improve understanding of belzutifan's real-world safety and effectiveness in VHL patients and help guide future care decisions.
How similar studies have gone: Real-world registries in similar kidney/central nervous system VHL contexts have provided useful insights, though this exact belzutifan registry is relatively novel.
Who can join: the full rules
Show full inclusion / exclusion 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).
Where it's running
-
Prince of Wales Hospital
Shatin, Hong Kong Recruiting
Study contacts
- Study coordinator: David Ka-Wai LEUNG, FRCS, MBChB
- Email: davidleung@surgery.cuhk.edu.hk
- Phone: 35053933
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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CAIX-targeted PET imaging to find tumors in people with von Hippel‑Lindau (VHL) disease
This trial gives a single dose of a CAIX-targeting radiolabeled antibody and does a PET/CT to see if the scan can find VHL‑related tumors in adults with VHL or in people with VHL-associated tumors needing surgery.
-
Biorepository for studying retinal diseases
This study is collecting health information and samples from people with and without eye diseases to see how these conditions develop and respond to treatments.
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CAIX-targeted 18F-T2 PET/CT imaging in solid tumors
This study tests a CAIX-specific radioactive tracer to see if PET/CT can help visualize solid tumors and checks safety and radiation exposure in adults with certain cancers.
-
Extension program for people previously treated with belzutifan (MK-6482)
This extension lets people with advanced solid tumors or VHL-related tumors who were already taking belzutifan in another study continue the drug so researchers can see how long they live and watch for long-term effects.
-
Real-world belzutifan outcomes and pharmacogenetics in von Hippel-Lindau syndrome
This study will test belzutifan in people aged 14 and older with VHL syndrome to see how well it controls VHL-associated tumors, what side effects occur, and how genetic factors affect response.
-
Registry for Rare Diseases
This study is creating a detailed registry to collect information on rare diseases like amyloidosis and sarcoidosis to help improve how they are diagnosed and treated.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.