New hope for rare GIST: targeted pill shows promise in early trial

NCT ID NCT07434843

Recruiting now Disease control Sponsor: Dana-Farber Cancer Institute Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 study tests the drug pemigatinib in 24 people with advanced SDH-deficient GIST, a rare stomach tumor that has spread and cannot be removed by surgery. The goal is to see if the drug can shrink tumors and how well patients tolerate it. Participants take a daily pill, and the study measures tumor response and 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: Pemigatinib (a targeted therapy pill that blocks certain proteins to slow tumor growth)
What this could lead to: If it works, this could offer a new treatment option for people with a rare type of GIST that has few alternatives.
What could go wrong: This is a small, early-phase trial with only 24 participants, so results may not apply to everyone. The drug may cause side effects or fail to shrink tumors.

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.

gastrointestinal stromal tumor

As listed by the trial registrant

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

GASTROINTESTINAL STROMAL TUMOR SDH GENE MUTATION

Contacts and locations

Locations

Dana Farber Cancer Institute
RECRUITING

Boston, Massachusetts, 02215, United States

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