New pill shows promise for rare kidney cancer in VHL patients

NCT ID NCT03401788

First seen Apr 14, 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.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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

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.

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.