RecruitingPhase 2NCT05661643

The Efficacy and Safety of Temozolomide in SDH-deficient GIST

A Phase 2 Study to Evaluate the Efficacy and Safety of Temozolomide in Advanced Gastrointestinal Stromal Tumor Patients With SDH Deficiency

Sponsor

Asan Medical Center

Enrollment

29 participants

Start Date

Jun 28, 2023

Study Type

INTERVENTIONAL

Conditions

The goal of this clinical trial is to investigate the efficacy and safety of temozolomide in SDH deficiency GIST patients.

Min Age: 20 Years

Age 20 years or older, at the time of acquisition of informed consent
Histologically confirmed GIST with CD117(+), DOG-1(+)
Wild type GIST without KIT or PDGFRα gene mutations determined by Sanger sequencing and panel sequencing
Eastern Cooperative Oncology Group (ECOG) performance status 0 ~ 2
Resolution of all adverse events with prior treatments to grade 0 or 1 by NCI-CTCAE version 5.0
At least one measurable lesion by RECIST version 1.1.
Adequate bone marrow, hepatic, renal, and other organ functions, before adjuvant imatinib treatment
Neutrophil >1,500/mm3
Platelet > 100,000/mm3
Hemoglobin >8.0 g/dL
Total bilirubin < 1.5 x upper limit of normal (ULN)
AST/ALT < 2.5 x ULN
Creatinine <1.5 x ULN
Life expectancy ≥12 weeks
Disease progression or discontinuation of treatment due to intolerable toxicity at least with palliative 1st line imatinib .
Washout period of previous TKIs or chemotherapy for more than 4 times the half life ((Imitinib and regorafenib need 1 week and sunitinib need 2 weeks.)
Provision of a signed written informed consent

Confirmed GIST with KIT or PDGFRα gene mutations determined by Sanger sequencing and panel sequencing
Women of child-bearing potential who are pregnant or breast feeding
Women or men who are not willing to use effective contraception entering the study period or until at least 6 months after the last study drug administration
If any of the following applies within ≤ 6 months prior to starting study enrollment : Myocardial Infarction, severe instable angina, coronary/peripheral bypass, NYHA class III or IV congestive heart failure, stroke or transient ischemic attack, treatment required severe arrhythmia
Uncontrolled infection
Acute and chronic liver disease and all chronic liver impairment.(But Patients with stable chronic hepatitis B are eligible
Acute, or chronic medical or psychiatric condition or laboratory abnormality such as active uncontrolled infection that difficult to study participation in the judgment of the investigator
Known diagnosis of HIV infection (HIV testing is not mandatory).
History of another primary malignancy that is currently clinically significant or currently requires active intervention.
Alcohol or substance abuse disorder
The patients with NTRK fusion
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