Can a Brain-Cancer drug take on a stubborn stomach tumor?
NCT ID NCT03556384
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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23 people
The number who actually took part.
- Started
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Sep 2018
- Finished
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Nov 2024
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Patient has pathologically confirmed SDH-mutant/deficient GIST. 2. Has recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy. 3. Patient has an ECOG Performance Status of 0-2. 4. Patient has adequate hematologic, hepatic and renal function. 5. Female patient of childbearing potential has a negative serum or urine pregnancy within 72 hours prior to receiving the first dose of study medication. 6. Female patient of childbearing potential agrees to use 2 methods of birth control or be surgically sterile, or abstain from heterosexual activity for the course of the study through 120 days after the last dose of study medication. 7. Male patient with a partner of childbearing potential agrees to use an adequate method of contraception starting with the first dose of study therapy through 120 days after the last dose of study therapy. 8. Has measurable or evaluable disease as per RECIST v1.1 (Appendix B). 9. Life expectancy of \>12 weeks. Exclusion Criteria: 1. Patients who have had major surgery within 4 weeks of initiation of study medication. 2. Patients who are receiving other concurrent anti-neoplastic therapy (e.g., chemotherapy, targeted therapy, immunotherapy, or radiotherapy) at the start of study treatment. 3. Patients with known brain metastases should be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events. 4. Evidence of severe or uncontrolled systemic diseases \[e.g., unstable or uncompensated respiratory, cardiac (including life threatening arrhythmias)\]. 5. Unresolved toxicity ≥ CTCAE Grade 2 from previous anti-cancer therapy except alopecia (if applicable) unless agreed that the patient can be entered after discussion with the Medical Monitor. 6. Presence of cardiac impairment class III and IV definitions; OR history of myocardial infarction/active ischemic heart disease within one year of study entry; OR uncontrolled dysrhythmias; OR poorly controlled angina. 7. Pregnant or breast-feeding females. 8. Medical condition such as uncontrolled infection (including HIV), uncontrolled diabetes mellitus or cardiac disease which, in the opinion of the treating physician, would make this protocol unreasonably hazardous for the patient. 9. Any concurrent condition which in the investigator's opinion makes it undesirable for the subject to participate in this trial or which would jeopardize compliance with the protocol. 10. Patients who cannot swallow oral formulations of the agent
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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
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Fox Chase Cancer Center
Philadelphia, Pennsylvania, 19111, United States
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Oregon Health & Science University
Portland, Oregon, 97239, United States
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UC San Diego Moores Cancer Center
La Jolla, California, 92093, United States
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University of Miami
Miami, Florida, 33136, United States
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