Long-term Efficacy and Safety of Belzutifan and Lenvatinib in Patients with von Hippel-Lindau Disease-Associated Tumors and Other Advanced Solid Tumors

The primary objective of this study is to assess overall survival in Cohort A and Cohort B. This investigation aims to determine the long-term efficacy of belzutifan in patients with von Hippel-Lindau disease-associated tumors or other specified solid tumors. The secondary objective includes:

• Evaluation of safety and tolerability across all cohorts.

This clinical trial includes a total of 118 patients, comprising both male and female individuals. The study population consists of participants within the 3 to 17 year age range diagnosed with advanced solid tumors or von Hippel-Lindau disease-associated tumors. Eligible subjects are currently receiving active treatment as part of a belzutifan parent study. Specific malignancies represented in the study population include renal cell carcinoma, hepatocellular carcinoma, non-MSI-H/dMMR colorectal cancer, pancreatic ductal adenocarcinoma, biliary tract cancer, non-MSIH/dMMR endometrial cancer, esophageal squamous cell carcinoma, advanced pheochromocytoma/paraganglioma, pancreatic neuroendocrine tumor, advanced gastrointestinal stromal tumor, and advanced solid tumors with HIF-2α related genetic alterations. The primary objectives of the study are:

• To assess overall survival in Cohort A.
• To assess overall survival in Cohort B.

This Phase 3, multicenter, open-label, extension study is designed to evaluate the long-term efficacy and safety of belzutifan and other treatments in participants with von Hippel-Lindau (VHL) disease-associated tumors or advanced solid tumors. The research methodology focuses on assessing overall survival in Cohort A and Cohort B. The study involves participants currently receiving active treatment in a belzutifan parent study. Secondary endpoints include the number of participants experiencing adverse events and the number of participants who discontinue treatment due to these events. The estimated duration of the trial extends through January 30, 2030.

The experimental treatment consists of belzutifan, which is administered as a 240 mg film-coated tablet via the oral route.

The experimental treatment also includes lenvatinib, provided in a 20 mg capsule for oral administration.

The primary efficacy endpoint for the assessment of participants in Cohort A and Cohort B is overall survival.