Can a Brain-Cancer drug take on a stubborn stomach tumor?

NCT ID NCT03556384

Completed Disease control Sponsor: Adam Burgoyne, MD, PhD Source: ClinicalTrials.gov ↗

First seen Jul 28, 2026 · Last updated Jul 29, 2026 · Updated 1 time

Summary

This phase 2 trial is testing whether the chemotherapy drug temozolomide can shrink tumors in people with a rare type of gastrointestinal stromal tumor (GIST) that has a mutation in the SDH gene. Standard GIST treatments do not work for this subtype. Participants take temozolomide pills for 21 days followed by a 7-day break, repeating every 28 days. The main goal is to see how many patients have their tumors shrink or disappear after six months of treatment.

What this could mean

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

Active substance: a chemotherapy drug called temozolomide (TMZ), taken as a pill for 21 days followed by 7 days off, in 28-day cycles
What this could lead to: If it works, this could offer a treatment option for people with SDH-mutant GIST, a rare cancer that does not respond to standard therapies.
What could go wrong: This is a small, early-phase trial with only 23 participants. Temozolomide has side effects like low blood counts and fatigue, and it may not shrink tumors in this specific cancer type.

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Conditions

The condition(s) this trial relates to.

cancer gastrointestinal stromal tumor Hematoma, Subdural neoplasm

As listed by the trial registrant

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

CANCER GASTROINTESTINAL STROMAL TUMORS GIST SDH

Contacts and locations

Locations

Fox Chase Cancer Center

Philadelphia, Pennsylvania, 19111, United States
Oregon Health & Science University

Portland, Oregon, 97239, United States
UC San Diego Moores Cancer Center

La Jolla, California, 92093, United States
University of Miami

Miami, Florida, 33136, United States

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