Study type

Study type

Non-interventional study

Scope of the study

Effectiveness study (incl. comparative)
Non-interventional study

Non-interventional study design

Cohort
Study drug and medical condition

Study drug International non-proprietary name (INN) or common name

BELZUTIFAN

Medical condition to be studied

Von Hippel-Lindau disease
Renal cell carcinoma
Haemangioblastoma
Pancreatic neuroendocrine tumour
Population studied

Short description of the study population

The target sample size includes approximately 40 eligible patients with VHL disease-associated RCC and approximately 40 patients with VHL disease-associated CNS hemangioblastoma requiring treatment. Enrollment of patients with each tumor type will be stopped once the target sample size is reached with overall recruitment stopped once the sample size requirement for both tumor types is met.

Age groups

Adults (18 to < 46 years)
Adults (46 to < 65 years)
Adults (65 to < 75 years)
Adults (75 to < 85 years)
Adults (85 years and over)

Estimated number of subjects

100
Study design details

Main study objective

The study aims to evaluate the treatment effectiveness and long-term safety of patients treated with belzutifan in routine clinical practice.

Outcomes

The primary objectives are to describe the proportion of patients who undergo at least one renal tumor reductive surgery (e.g., nephrectomy) or locally directed therapy (e.g., radiofrequency ablation) and to describe the proportion of patients who undergo at least one CNS tumor reductive surgery (e.g., craniectomy) or locally directed therapy (e.g., radiation therapy).
Secondary objectives are to describe proportion of patients with treatment emergent SAEs, evaluate treatment patterns, and for participants with VHL disease-associated RCC and, separately, VHL disease-associated CNS hemangioblastoma, to describe the proportion of patients who develop metastatic disease (for RCC only), and proportion with occurrence of new VHL tumors or tumor type.

Data analysis plan

Participant data for those who undergo surgery or other tumor reductive procedures, development of metastatic disease for RCC or VHL disease-associated study conditions will be summarized descriptively using frequency of counts or descriptive statistics such as n, mean, 95% CI of the mean, SD, median, minimum, maximum separately for VHL disease-associated RCC and VHL disease-associated CNS hemangioblastoma.