New pill shows promise for rare kidney cancer in VHL patients

NCT ID NCT03401788

Ongoing Disease control Sponsor: Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA) Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This phase 2 trial is testing a daily pill called belzutifan in 50 people with VHL disease who have kidney cancer. The goal is to see if the drug can shrink tumors. Researchers are also monitoring how long any tumor shrinkage lasts and checking for side effects.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Belzutifan (a pill taken once daily)
What this could lead to: If successful, belzutifan could become a new treatment option to shrink kidney tumors in people with VHL disease, potentially reducing the need for surgery.
What could go wrong: This is a small, early-phase trial with only 50 participants, so results may not apply to everyone. The drug may not work for all patients or could cause side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

hereditary clear cell renal cell carcinoma renal cell carcinoma von Hippel-Lindau disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

VHL - VON HIPPEL-LINDAU SYNDROME VHL GENE INACTIVATION VHL GENE MUTATION VHL SYNDROME VHL-ASSOCIATED CLEAR CELL RENAL CELL CARCINOMA VHL-ASSOCIATED RENAL CELL CARCINOMA

Contacts and locations

Locations

Aarhus University Hospital

Aarhus, Denmark
Cambridge University Hospital

Cambridge, United Kingdom
Hospital Georges Pompidou

Paris, France
Huntsman Cancer Institute

Salt Lake City, Utah, 84112, United States
MD Anderson Cancer Center

Houston, Texas, 77030, United States
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States
University of Michigan

Ann Arbor, Michigan, 48109, United States
University of Pennsylvania Medical Center

Philadelphia, Pennsylvania, 19104, United States
University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15232, United States
Vanderbilt Medical Center

Nashville, Tennessee, 37232, United States

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