Gastrointestinal Stromal TumorsPhase 2NCT05385549
5 Years of Adjuvant Imatinib in Patients With Gastrointestinal Stromal Tumor With a High Risk
A Phase 2 study testing Imatinib Mesylate in people with Gastrointestinal Stromal Tumors, run by Asan Medical Center at 1 site in South Korea.
Trial summary
In this study, the investigators aim to investigate the efficacy and safety of 5 years of adjuvant imatinib treatment in patients with tumor rupture defined by Nishida classification or those with a tumor size 10cm or larger and a mitotic index of 10/50HPFs or higher. 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?
- Imatinib Mesylate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 1 siteSouth Korea
- Study dates
- 7 Sep 2022to 30 Apr 2030
- 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(+), or mutation in KIT or PDGFRα gene
- (1) Completely resected localized GIST (R0 resection) within 12 weeks prior to the start of the adjuvant imatinib.
- After complete resection(R0 resection), High risk GIST according to Modified NIH criteria and ongoing adjuvant imatinib treatment.
- 4) High risk GIST according to Modified NIH criteria,
- Tumor rupture according to Nishida classification or
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- tumor size >10cm and mitosis >10/50 HPF 5) Eastern Cooperative Oncology Group (ECOG) performance status 0 ~ 2 6) 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 7) Provision of a signed written informed consent
Who cannot join
- 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 3 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)
- Patients who had reduced the dose of imatinib to less than 300 mg/day due to toxicity.
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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 PDGFRα D842V mutation
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
Yoon-Koo Kang, MD, PhD, +82-2-3010-3230, ykkang@amc.seoul.kr
Min-Hee Ryu, MD, PhD, +82-2-3010-5935, miniryu@amc.seoul.kr
About this study
Official title: A Phase 2 Study of 5 Years of Adjuvant Imatinib in Patients With Gastrointestinal Stromal Tumor With a High Risk of Recurrence Following Surgical Resection
In this study, the investigators aim to investigate the efficacy and safety of 5 years of adjuvant imatinib treatment in patients with tumor rupture defined by Nishida classification or those with a tumor size 10cm or larger and a mitotic index of 10/50HPFs or higher.
Read the detailed description
Localized resectable GISTs can be cured with surgical resection, but no effective therapy had been established for patients with unresectable and/or metastatic GISTs and their prognosis was extremely poor before the advent of imatinib.
Imatinib mesylate is an oral tyrosine-kinase inhibitor (TKI) with activity against KIT, PDGFRA, ABL, and DDR. The efficacy of imatinib was first shown in the pivotal B2222 trial and confirmed by two subsequent randomized phase III trials. The standard dose of imatinib was established as a 400mg once daily dose upfront high-dose imatinib treatment with a 800mg daily dose showed a higher efficacy in terms of progression-free survival (PFS) in patients with GISTs harboring KIT exon 9 mutations but also higher toxicity. The median time-to-progression (TTP) with imatinib was about 2 years in the extended follow-up results of the B2222 trial. Imatinib efficacy correlates with primary KIT mutations and patients with KIT exon 9 mutations had worse survival outcomes than those with KIT exon 11 mutations.
Surgical resection is the mainstay of the treatment of localized GIST. However, in a proportion of patients, a high recurrence rate was observed, which prompted the investigation of the clinical efficacy of adjuvant imatinib treatment. The ACOSSOG Z9001 study showed that one year of adjuvant imatinib treatment after surgical resection in patients with a tumor size of 3cm of larger improved recurrence-free survival compared to placebo. This study was the first to demonstrate the efficacy of adjuvant imatinib treatment. Subsequently, SSG XVIII/AIO study showed that 3 years of adjuvant imatinib improved recurrence-free survival and overall survival compared to one year of adjuvant imatinib in High risk patients defined by modified NIH criteria. Based on this study in patients classified as high risk by the modified NIH criteria, 3 years adjuvant imatinib is the standard of care.
However, given that a substantial proportion of such patients show disease recurrence, the PERSIST5 study recently showed that 5 years of imatinib treatment may further reduce the recurrence. In addition, clinical outcomes of the patients classified as high risk by the modified NIH criteria are heterogeneous, and some of these patients show particularly poor clinical outcomes. The investigators analyzed the clinical outcomes of 222 high risk GIST patients who received 3 years of adjuvant imatinib treatment following surgical resection. Among these, patients with tumor rupture defined by Nishida classification or those with a tumor size 10cm or larger and a mitotic index of 10/50HPFs or higher had poor clinical outcomes with a 5-year recurrence-free survival of 50%.
In this study, the investigators aim to investigate the efficacy and safety of 5 years of adjuvant imatinib treatment in patients with tumor rupture defined by Nishida classification or those with a tumor size 10cm or larger and a mitotic index of 10/50HPFs or higher.
- Conditions
- Gastrointestinal Stromal Tumors
- Being tested
- Imatinib Mesylate
- 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.
Imatinib MesylateDrug
Imatinib Mesylate, 400 mg once daily, oral. 5 years of adjuvant imatinib treatment (standard treatment 3years + IP treatment : 2yesrs)
| Study group | Role | What they receive |
|---|---|---|
| 5 years of adjuvant imatinib treatment | Experimental | Not described Receives: Drug: Imatinib Mesylate |
What is being measured
The primary outcome is the main question the study is designed to answer.
PFS
Progression-free survival (PFS) per the RECIST v1.1 is defined as the time from the date of first dosing of Imatinib to the date of progression or death due to any cause
Measured: up to 5years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2022
- Study start7 Sep 2022
- Primary completion30 Apr 2030 (estimated)
- Study completion30 Apr 2030 (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
- 35 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
- NCT05385549
- Sponsor's protocol ID
- AMC2202
- Status last verified
- Aug 2025
- First submitted
- 18 May 2022
- Last updated
- 8 Aug 2025
- 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 8 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study of 5 Years of Adjuvant Imatinib in Patients With Gastrointestinal Stromal Tumor With a High Risk of Recurrence Following Surgical Resection
- ClinicalTrials.gov ID
- NCT05385549
- Acronym / sponsor ID
- AMC2202
- Conditions
- Gastrointestinal Stromal Tumors
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 35 participants (estimated)
- Lead sponsor
- Asan Medical CenterUniversity, hospital or other
- Study start
- 7 Sep 2022 (actual)
- Primary completion
- 30 Apr 2030 (estimated)
- Study completion
- 30 Apr 2030 (estimated)
- First posted
- 23 May 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.
In this study, the investigators aim to investigate the efficacy and safety of 5 years of adjuvant imatinib treatment in patients with tumor rupture defined by Nishida classification or those with a tumor size 10cm or larger and a mitotic index of 10/50HPFs or higher.
Detailed description
Localized resectable GISTs can be cured with surgical resection, but no effective therapy had been established for patients with unresectable and/or metastatic GISTs and their prognosis was extremely poor before the advent of imatinib.
Imatinib mesylate is an oral tyrosine-kinase inhibitor (TKI) with activity against KIT, PDGFRA, ABL, and DDR. The efficacy of imatinib was first shown in the pivotal B2222 trial and confirmed by two subsequent randomized phase III trials. The standard dose of imatinib was established as a 400mg once daily dose upfront high-dose imatinib treatment with a 800mg daily dose showed a higher efficacy in terms of progression-free survival (PFS) in patients with GISTs harboring KIT exon 9 mutations but also higher toxicity. The median time-to-progression (TTP) with imatinib was about 2 years in the extended follow-up results of the B2222 trial. Imatinib efficacy correlates with primary KIT mutations and patients with KIT exon 9 mutations had worse survival outcomes than those with KIT exon 11 mutations.
Surgical resection is the mainstay of the treatment of localized GIST. However, in a proportion of patients, a high recurrence rate was observed, which prompted the investigation of the clinical efficacy of adjuvant imatinib treatment. The ACOSSOG Z9001 study showed that one year of adjuvant imatinib treatment after surgical resection in patients with a tumor size of 3cm of larger improved recurrence-free survival compared to placebo. This study was the first to demonstrate the efficacy of adjuvant imatinib treatment. Subsequently, SSG XVIII/AIO study showed that 3 years of adjuvant imatinib improved recurrence-free survival and overall survival compared to one year of adjuvant imatinib in High risk patients defined by modified NIH criteria. Based on this study in patients classified as high risk by the modified NIH criteria, 3 years adjuvant imatinib is the standard of care.
However, given that a substantial proportion of such patients show disease recurrence, the PERSIST5 study recently showed that 5 years of imatinib treatment may further reduce the recurrence. In addition, clinical outcomes of the patients classified as high risk by the modified NIH criteria are heterogeneous, and some of these patients show particularly poor clinical outcomes. The investigators analyzed the clinical outcomes of 222 high risk GIST patients who received 3 years of adjuvant imatinib treatment following surgical resection. Among these, patients with tumor rupture defined by Nishida classification or those with a tumor size 10cm or larger and a mitotic index of 10/50HPFs or higher had poor clinical outcomes with a 5-year recurrence-free survival of 50%.
In this study, the investigators aim to investigate the efficacy and safety of 5 years of adjuvant imatinib treatment in patients with tumor rupture defined by Nishida classification or those with a tumor size 10cm or larger and a mitotic index of 10/50HPFs or higher.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 5 years of adjuvant imatinib treatment | Experimental | Drug: Imatinib Mesylate |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Age 20 years or older, at the time of acquisition of informed consent
- Histologically confirmed GIST with CD117(+), DOG-1(+), or mutation in KIT or PDGFRα gene
- (1) Completely resected localized GIST (R0 resection) within 12 weeks prior to the start of the adjuvant imatinib.
- After complete resection(R0 resection), High risk GIST according to Modified NIH criteria and ongoing adjuvant imatinib treatment.
- 4) High risk GIST according to Modified NIH criteria,
Key exclusion criteria (11 listed)
- 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 3 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)
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 |
|---|---|---|
| Imatinib MesylateImatinib Mesylate, 400 mg once daily, oral. 5 years of adjuvant imatinib treatment (standard treatment 3years + IP treatment : 2yesrs) | Drug | 5 years of adjuvant imatinib treatment |
Primary outcome
PFS
Time frame: up to 5years
Progression-free survival (PFS) per the RECIST v1.1 is defined as the time from the date of first dosing of Imatinib to the date of progression or death due to any cause
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 8 Aug 2025.
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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 Imatinib Mesylate 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 13 inclusion and 11 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 8 Aug 2025. 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 Ryu Min-Hee, 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?
PFS (time frame: up to 5years). 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 8 Aug 2025.