Gastrointestinal Stromal TumorsPhase 2GISTNCT05661643
The Efficacy and Safety of Temozolomide in SDH-deficient GIST
A Phase 2 study testing Temozolomide capsule in people with Gastrointestinal Stromal Tumors, run by Asan Medical Center at 1 site in South Korea.
Trial summary
The goal of this clinical trial is to investigate the efficacy and safety of temozolomide in SDH deficiency GIST patients. Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Gastrointestinal Stromal Tumors
- What is being tested?
- Temozolomide capsule
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 1 siteSouth Korea
- Study dates
- 28 Jun 2023to 31 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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
Who cannot join
- 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
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- 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
- 5)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
South Korea1 site, 1 recruiting
- Recruiting
Asan Medical Center, University of Ulsan College of Medicine
Seoul, Seoul, 138-736
Min-Hee Ryu, MD, PhD, +82-2-3010-5935, miniryu@amc.seoul.kr
About this study
Official title: A Phase 2 Study to Evaluate the Efficacy and Safety of Temozolomide in Advanced Gastrointestinal Stromal Tumor Patients With SDH Deficiency
The goal of this clinical trial is to investigate the efficacy and safety of temozolomide in SDH deficiency GIST patients.
Read the detailed description
Wild type GISTs are less responsive to imatinib with a response rate of 23.1-44.6% and a median progressiion-free survival of 12.3-12.8 months. The efficacy of imatinib is limited in particular in SDH deficienctGIST with a reported response of 2%. Therefore, the development of a new therapeutic agents is urgently needed.
Recently, a study of TKI-resistant SDH-deficient preclinical model showed that temozolomide, an alkylating agent, promotes DNA damage in tumor cells, leading to tumor cell killing. In a retrospective analysis, 2 out of 5 SDH deficient GIST patients treated with temozolomide showed partial response, suggesting its efficacy in this patient population.
Based on these findings,The goal of this clinical trial is to investigate the efficacy and safety of temozolomide in SDH deficiency GIST patients. In addition, for exploratory purposes, aim to investigate the efficacy and safety of temozolomide in KIT and PDGFRA wild-type GIST without SDH deficiency.
- Conditions
- Gastrointestinal Stromal Tumors
- Being tested
- Temozolomide capsule
- Medical subject headings
- Gastrointestinal Stromal Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Temozolomide capsuleDrug
Temozolomide 200 mg/m2 is administered orally for 1-5 days of each cycle, and then canceled for 23 days (a total of 28 days is 1 cycle)
| Study group | Role | What they receive |
|---|---|---|
| temozolomide treatment | Experimental | Not described Receives: Drug: Temozolomide capsule |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective respone rate in SDH deficiency wild type GIST
complet response+partial response defined by RECIST v1.1
Measured: up to 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2022
- Study start28 Jun 2023
- Primary completion1 Dec 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gastrointestinal Stromal TumorsStudies of the same condition, open and under way.
- More trials run by Asan Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 29 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Asan Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Min-Hee Ryu, Asan Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05661643
- Sponsor's protocol ID
- AMC2203
- Acronym
- GIST
- Status last verified
- Apr 2026
- First submitted
- 14 Dec 2022
- Last updated
- 7 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study to Evaluate the Efficacy and Safety of Temozolomide in Advanced Gastrointestinal Stromal Tumor Patients With SDH Deficiency
- ClinicalTrials.gov ID
- NCT05661643
- Acronym / sponsor ID
- GIST / AMC2203
- Conditions
- Gastrointestinal Stromal Tumors
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 29 participants (estimated)
- Lead sponsor
- Asan Medical CenterUniversity, hospital or other
- Study start
- 28 Jun 2023 (actual)
- Primary completion
- 1 Dec 2026 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 22 Dec 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to investigate the efficacy and safety of temozolomide in SDH deficiency GIST patients.
Detailed description
Wild type GISTs are less responsive to imatinib with a response rate of 23.1-44.6% and a median progressiion-free survival of 12.3-12.8 months. The efficacy of imatinib is limited in particular in SDH deficienctGIST with a reported response of 2%. Therefore, the development of a new therapeutic agents is urgently needed.
Recently, a study of TKI-resistant SDH-deficient preclinical model showed that temozolomide, an alkylating agent, promotes DNA damage in tumor cells, leading to tumor cell killing. In a retrospective analysis, 2 out of 5 SDH deficient GIST patients treated with temozolomide showed partial response, suggesting its efficacy in this patient population.
Based on these findings,The goal of this clinical trial is to investigate the efficacy and safety of temozolomide in SDH deficiency GIST patients. In addition, for exploratory purposes, aim to investigate the efficacy and safety of temozolomide in KIT and PDGFRA wild-type GIST without SDH deficiency.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| temozolomide treatment | Experimental | Drug: Temozolomide capsule |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Temozolomide capsuleTemozolomide 200 mg/m2 is administered orally for 1-5 days of each cycle, and then canceled for 23 days (a total of 28 days is 1 cycle) | Drug | temozolomide treatment |
Primary outcome
Objective respone rate in SDH deficiency wild type GIST
Time frame: up to 4 years
complet response+partial response defined by RECIST v1.1
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Apr 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Temozolomide capsule in people with Gastrointestinal Stromal Tumors, run by Asan Medical Center at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 7 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Min-Hee Ryu, MD, PhD (82-2-3010-5936, miniryu@amc.seoul.kr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective respone rate in SDH deficiency wild type GIST (time frame: up to 4 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Apr 2026.