New drug combo shows promise for rare nerve tumors

NCT ID NCT03433183

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial tested two drugs, selumetinib and sirolimus, in 21 people with advanced malignant peripheral nerve sheath tumors (MPNST), a rare cancer often linked to neurofibromatosis type 1. The goal was to see if the combination could shrink or stabilize tumors. The study measured how many patients had their disease controlled for at least four treatment cycles.

What this could mean

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

Active substance
Selumetinib and sirolimus
What this could lead to
If successful, this combination could offer a new treatment option to slow or stop tumor growth in patients with advanced malignant peripheral nerve sheath tumors.
What could go wrong
This is a small, early-phase trial with only 21 participants. The results may not apply to all patients, and the combination may cause side effects without improving survival.

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.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Johns Hopkins University

    Baltimore, Maryland, 21287, United States

  • National Cancer Institute

    Bethesda, Maryland, 20892, United States

  • Washington University

    St Louis, Missouri, 63110, United States

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