Clinical trial evaluates small molecule inhibitor in von Hippel-Lindau disease-associated renal cell carcinoma

Renal cell carcinoma

Bilateral VHL renal tumors
Photo courtesy of NCI Visuals Online

Patients with von Hippel-Lindau disease-associated renal cell carcinoma may be eligible to participate in a new clinical trial at the NIH Clinical Center.

Von Hippel-Lindau (VHL) disease is a hereditary condition that can be passed down through generations of a family. People with VHL have an increased risk of developing tumors in several organs, including renal cell carcinoma (RCC) in the kidneys. Because there are no approved drugs specifically for VHL-associated RCC, patients usually must undergo one or more surgeries to limit the spread of their tumors. Ramaprasad Srinivasan, M.D., Ph.D., of the Urologic Oncology Branch is leading a trial of a new drug currently known as PT2977. PT2977 is a small molecule inhibitor that blocks a protein that regulates the response of cells to changes in oxygen levels. The normal regulation of this protein is disrupted in tumors occurring in patients with VHL disease, leading to very high levels of the protein. Investigators want to see if PT2977 can suppress the activity of this protein and thus shrink RCC tumors. This could reduce the need for surgery for patients with VHL-associated RCC.  

Clinicaltrials.gov identifier: NCT03401788
NCI Protocol ID: NCI-18-C-0138
Official Title: An Open-Label Phase 2 Study to Evaluate PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma

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Summary Posted: 10/2018