VHL - Von Hippel-Lindau SyndromeNCT07554508
Belzutifan VHL RCC HK Prospective Registry
An observational study of VHL - Von Hippel-Lindau Syndrome, run by Chinese University of Hong Kong at 1 site in Hong Kong.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- VHL - Von Hippel-Lindau Syndrome
- How many people
- 100 participants
- Where
- 1 siteHong Kong
- Study dates
- 22 Dec 2025to 31 Mar 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult patients diagnosed with VHL syndrome who choose to use belzutifan for their treatment
Who may be able to join
- 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.
Who cannot join
- Tumour requiring immediate resection.
- Pregnancy or unwillingness to use contraception if of childbearing potential.
- Uncontrolled comorbidities (e.g., active infection, severe cardiopulmonary disease).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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.
Read the 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.
- Conditions
- VHL - Von Hippel-Lindau Syndrome
- Medical subject headings
- von Hippel-Lindau Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Belzutifan Group | Subjects received Belzutifan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective response rate
RECIST 1.1
Measured: Month 6 and Month 12
Secondary outcomes (3)
Radiographic response
By RECIST 1.1
Measured: Month 6 and Month 12, Month 18, Month 24, Month 30 and 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
Measured: Baseline, Month 6 and Month 12
Health-economic impact of belzutifan
By per-patient emergence department and inpatient cost
Measured: Month 6, Month 12, Month,18, Month 24, Month 30, Month 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Apr 2026
- Study start22 Dec 2025
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Mar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for VHL - Von Hippel-Lindau SyndromeStudies of the same condition, open and under way.
- More trials run by Chinese University of Hong KongThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chinese University of Hong Kong
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, NG Chi Fai, Chinese University of Hong Kong
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07554508
- Sponsor's protocol ID
- CRE-2025.563
- Status last verified
- Oct 2026
- First submitted
- 21 Apr 2026
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
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.
PubMed 34268287Linehan 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.
PubMed 40274483Louise 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.
PubMed 35709961Jonasch 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.
PubMed 34818478
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Registry for Belzutifan in the Treatment of Von Hippel-Lindau (VHL) Disease
- ClinicalTrials.gov ID
- NCT07554508
- Acronym / sponsor ID
- CRE-2025.563
- Conditions
- VHL - Von Hippel-Lindau Syndrome
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Chinese University of Hong KongUniversity, hospital or other
- Study start
- 22 Dec 2025 (actual)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 31 Mar 2029 (estimated)
- First posted
- 28 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Belzutifan Group | – | –Subjects received Belzutifan |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult patients diagnosed with VHL syndrome who choose to use belzutifan for their treatment
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (3 listed)
- Tumour requiring immediate resection.
- Pregnancy or unwillingness to use contraception if of childbearing potential.
- Uncontrolled comorbidities (e.g., active infection, severe cardiopulmonary disease).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Objective response rate
Time frame: Month 6 and Month 12
RECIST 1.1
3 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2026.
- Background: PubMed 34268287Zhou 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.
- Background: PubMed 40274483Linehan 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.
- Background: PubMed 35709961Louise 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.
- Background: PubMed 34818478Jonasch 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.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of VHL - Von Hippel-Lindau Syndrome, run by Chinese University of Hong Kong at 1 site in Hong Kong.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Hong Kong. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact David Ka-Wai LEUNG, FRCS, MBChB (35053933, davidleung@surgery.cuhk.edu.hk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective response rate (time frame: Month 6 and Month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07554508, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Oct 2026.