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New hope for VHL patients: belzutifan trial targets rare tumor disorder

NCT ID NCT07167329

Recruiting now Disease control Sponsor: José Claudio Casali da Rocha Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This study tests the drug belzutifan in 100 people with von Hippel-Lindau (VHL) syndrome, a genetic condition that causes tumors in various organs. Researchers will track how well the drug shrinks tumors and how long it takes for the disease to progress, while also monitoring side effects like anemia. The goal is to see if belzutifan can effectively control VHL-related tumors in a real-world setting.

What this could mean

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

Active substance

belzutifan

What this could lead to

If successful, this could show that belzutifan effectively shrinks or controls VHL-related tumors, offering a targeted treatment option for this rare genetic condition.

What could go wrong

This is a phase 2 study with only 100 participants, so results may not apply to all VHL patients. Side effects like anemia are possible, and the drug may not work for everyone.

Disclaimer Read more

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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Conditions

The condition(s) this trial relates to.

endolymphatic sac tumor hemangioblastoma hereditary clear cell renal cell carcinoma Neuroectodermal Tumors, Primitive paraganglioma pheochromocytoma pheochromocytoma-paraganglioma primitive neuroectodermal tumor renal cell carcinoma von Hippel-Lindau disease

As listed by the trial registrant

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

ENDOLYMPHATIC SAC TUMOR HEMANGIOBLASTOMA (HB) OF THE CENTRAL NERVOUS SYSTEM (CNS) PHEOCHROMOCYTOMA/PARAGANGLIOMA PNET RETINAL ANGIOMATOUS PROLIFERATION VON HIPPEL LINDAU VON HIPPEL LINDAU DISEASE VON HIPPEL LINDAU-DEFICIENT CLEAR CELL RENAL CELL CARCINOMA

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • AC Camargo Cancer Center

    RECRUITING

    São Paulo, São Paulo, 01509900, Brazil

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••