Active — Not RecruitingPhase 2

Temozolomide (TMZ) in Advanced Succinate Dehydrogenase (SDH)-Mutant/Deficient Gastrointestinal Stromal Tumor (GIST)

United States23 participantsStarted 2018-09-12

Plain-language summary

Funding Source - FDA OOPD FDA-approved products for patients with unresectable or metastatic GIST include therapies such as imatinib and sunitinib. Although there are FDA-approved products for the treatment of advanced/metastatic GIST, these therapies are known to be ineffective in the SDH-mutant/deficient subtype and no known effective therapies exist. The purpose of this study is to investigate SDH-Mutant/Deficient Gastrointestinal Stromal cancer's response to the drug Temozolomide (TMZ) and aim to improve patient outcomes. Temozolomide is approved by the FDA for the treatment of newly diagnosed glioblastoma multiforme (GBM) and refractory anaplastic astrocytoma cancers. Temozolomide is considered experimental because it is not approved by the FDA for the treatment of SDH-Mutant/Deficient Gastrointestinal Stromal Tumor.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Patient has pathologically confirmed SDH-mutant/deficient GIST.
✓. Has recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy.
✓. Patient has an ECOG Performance Status of 0-2.
✓. Patient has adequate hematologic, hepatic and renal function.
✓. Female patient of childbearing potential has a negative serum or urine pregnancy within 72 hours prior to receiving the first dose of study medication.
✓. 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.
✓. 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.
✓. Has measurable or evaluable disease as per RECIST v1.1 (Appendix B).

Exclusion criteria

What they're measuring

Overall Response Rate

Timeframe: 6 months

Trial details

NCT IDNCT03556384

SponsorAdam Burgoyne, MD, PhD

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2025-06

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