Non-interventional post-authorization study of belzutifan in adult patients with von Hippel-Lindau disease-associated renal cell carcinoma, pancreatic neuroendocrine tumor and/or central nervous system hemangioblastoma (MK-6482-026)

Study type

• Study topic:
  Human medicinal product
• 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
• Anatomical Therapeutic Chemical (ATC) code:
  (L01XX74) belzutifan

  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.

Documents

Study, other information

D1_PPLS_EUPAS 108114_for pub_12Mar2025_V1-0_MK-6482-026-04.pdf