VHL - Von Hippel-Lindau Syndrome
Conditions
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.
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.
Interventions
None listed
Sponsors
Chinese University of Hong Kong
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Objective response rate | Month 6 and Month 12 | RECIST 1.1 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Radiographic response | Month 6 and Month 12, Month 18, Month 24, Month 30 and Month 36 | By RECIST 1.1 |
| Change in Quality of Life score | Baseline, Month 6 and Month 12 | By EQ-5D-5L. Score range from 0-100.The higher the score the better quality of life |
| Health-economic impact of belzutifan | Month 6, Month 12, Month,18, Month 24, Month 30, Month 36 | By per-patient emergence department and inpatient cost |
Countries
Hong Kong
Contacts
CONTACTDavid Ka-Wai LEUNG, FRCS, MBChB
Outcome results
None listed