Gastrointestinal Stromal TumorsPhase 2NCT06326346
GIST Oral Paclitaxel(Liporaxel)
A Phase 2 study testing Liporaxel in people with Gastrointestinal Stromal Tumors, run by Asan Medical Center at 1 site in South Korea.
Trial summary
The purpose of this study is to evaluate safety and efficacy of Liporaxel for patients with GIST who failed on prior standard treatments, including imatinib, sunitinib, and regorafenib, and with low P-glycoprotein expression. 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?
- Liporaxel
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 1 siteSouth Korea
- Study dates
- 20 Nov 2024to 31 Mar 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 metastatic and/or advanced GIST with CD117(+), DOG-1(+), or mutation in KIT or PDGFRα gene
- Failed (progressed and/or intolerable) after prior treatments for GIST, including at least imatinib and sunitinib, regorafenib.
- Adequate tissue obtained after treatment failure to imatinib, sunitinib, and regorafenib for P-glycoprotein immunohistochemistry (IHC) analysis, and showed P-glycoprotein expression of less than 6. (For patients with PDGFRα D842V mutation or other subtypes with poor response to tyrosine kinase inhibitors, tumor tissue obtained at any period can be used.)
- Eastern Cooperative Oncology Group (ECOG) performance status 0~2
- Resolution of all toxic effects of prior treatments to grade 0 or 1 by NCI-CTCAE version 5.0
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- At least one measurable lesion as defined by RECIST version 1.1.
- Adequate bone marrow, hepatic, renal, and other organ functions
- 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
- Washout period of previous TKIs or chemotherapy for more than 4 times the half life ((Imatinib and regorafenib need 1 week and sunitinib need 2 weeks.)
- 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
- Diabetes mellitus with clinically significant peripheral artery disease
- Acute and chronic liver disease and all chronic liver impairment.(But Patients with stable chronic hepatitis B are eligible)
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- Uncontrolled gastrointestinal toxicities with toxicity greater than NCI CTCAE grade 2
- 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
- The patient experienced any bleeding episode considered life-threatening, or any grade 3 or 4 bleeding event. (required transfusion or endoscopic or surgical intervention)
- Patient who underwent major surgery or is under recovery from surgery within 28 days from the study treatment
- 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.
- Patients with clinically suspected brain metastasis symptom, brain metastases as assessed by radiologic imaging.
- Alcohol or substance abuse disorder.
- Known severe hypersensitivity to paclitaxel
- Received paclitaxel-based treatment for GIST
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, Songpagu, 138-736
Min-Hee Ryu, MD, PhD, 82-2-3010-5936, miniryu@amc.seoul.kr
Hyung-Don Kim, MD, PhD, 82-2-3010-0236, kimhdmd@amc.seoul.kr
About this study
Official title: A Phase II Study of Oral Paclitaxel (Liporaxel) in GIST Patients With a Low P-glycoprotein Expression After Failure With Imatinib, Sunitinib and Regorafenib
The purpose of this study is to evaluate safety and efficacy of Liporaxel for patients with GIST who failed on prior standard treatments, including imatinib, sunitinib, and regorafenib, and with low P-glycoprotein expression.
Read the detailed description
The survival outcomes of patients with advanced and/or metastatic gastrointestinal stromal tumors (GIST) have markedly improved with application of imatinib, the KIT tyrosine kinase inhibitor (GlivecTM, Novartis) for the treatment. Recently, sunitinib (SuteneTM, Pfizer) and regorafenib (StivargaTM, Bayer) have shown efficacy in patients with disease progression on imatinib as second- and third-line treatment, respectively. However, most patients develop acquired resistance to KIT tyrosine kinase inhibitors and show disease progression.
While cytotoxic chemotherapeutic agents were expected to have minimal antitumor effect on GIST, recent preclinical studies have shown that 37 out of 89 different chemotherapeutic agents have shown antitumor effects on at least one or more GIST cell lines. Especially, topoisomerase II inhibitors, paclitaxel, bortezomib have shown promising results. Based on the results, the investigators conducted a single center, single arm phase II study to evaluate efficacy and safety of paclitaxel for patients with GIST who failed to prior imatinib and sunitinib. Overall efficacy was modest, although patients with low P-glycoprotein expression showed better efficacy compared to those with high P-glycoprotein expression. Subsequently, the investigators performed another phase 2 trial to evaluate efficacy of paclitaxel for previously treated GIST patients with low P-glycoprotein expression and have met the primary endpoint.
Liporaxel is an oral formulation of paclitaxel, and showed high bioavailability, safety, and antitumor effect from non-clinical studies. From a phase 1 clinical trial for metastatic solid tumor patients, Liporaxel showed adequate PK/PD profiles. Compared to intravenous paclitaxel, administration is relatively easier and has better safety in terms of hypersensitivity reaction which is caused by the admixture of intravenous formulation of paclitaxel. From a multicenter phase 3 trial conducted in South Korea for patients with advanced gastric adenocarcinoma who failed on first-line palliative chemotherapy (DREAM trial), Liporaxel showed non-inferiority compared to intravenous paclitaxel in terms of both safety and efficacy, and it is currently approved for the second-line treatment in advanced gastric cancer. Also, Liporaxel proved safety and potential efficacy in patients with HER2 negative metastatic breast cancer as first-line chemotherapy, and currently multicenter phase 3 trial in ongoing (OPTIMAL3 trial).
The purpose of this study is to evaluate safety and efficacy of Liporaxel for patients with GIST who failed on prior standard treatments, including imatinib, sunitinib, and regorafenib, and with low P-glycoprotein expression.
- Conditions
- Gastrointestinal Stromal Tumors
- Being tested
- Liporaxel
- 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.
LiporaxelDrug
Liporaxel 200mg/m2 twice daily orally on day 1, 8, and 15 every 4 weeks (1 cycle = 4 weeks)
| Study group | Role | What they receive |
|---|---|---|
| Oral Paclitaxel (Liporaxel) | Experimental | Liporaxel 200mg/m2 twice daily orally on day 1, 8, and 15 every 4 weeks (1 cycle = 4 weeks) Receives: Drug: Liporaxel |
What is being measured
The primary outcome is the main question the study is designed to answer.
16 week disease control rate
according to Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1)
Measured: 16 weeks
Secondary outcomes (4)
Progression-free survival
time from the date of first administration of palliative first-line chemotherapy to the date of the first objectively documented tumor progression or death, whichever occurs first)
Measured: until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 36 months
Overall survival
time from the date of first administration of palliative first-line chemotherapy to the date of death due to any cause
Measured: through study completion, an average of 3 years
Objective response rate
according to Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1)
Measured: every 4 weeks for the initial two evaluation, then every 8 weeks
Adverse event assessed by NCI-CTCAE Version 5.0
assessed by NCI-CTCAE Version 5.0
Measured: until 28 days from the last administration of the investigational product
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2024
- Study start20 Nov 2024
- Primary completion31 Mar 2027 (estimated)
- Study completion31 Mar 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
- 28 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
- NCT06326346
- Sponsor's protocol ID
- AMC2401
- Status last verified
- Apr 2026
- First submitted
- 17 Mar 2024
- Last updated
- 7 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- 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 II Study of Oral Paclitaxel (Liporaxel) in GIST Patients With a Low P-glycoprotein Expression After Failure With Imatinib, Sunitinib and Regorafenib
- ClinicalTrials.gov ID
- NCT06326346
- Acronym / sponsor ID
- AMC2401
- Conditions
- Gastrointestinal Stromal Tumors
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 28 participants (estimated)
- Lead sponsor
- Asan Medical CenterUniversity, hospital or other
- Study start
- 20 Nov 2024 (actual)
- Primary completion
- 31 Mar 2027 (estimated)
- Study completion
- 31 Mar 2027 (estimated)
- First posted
- 22 Mar 2024 (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 purpose of this study is to evaluate safety and efficacy of Liporaxel for patients with GIST who failed on prior standard treatments, including imatinib, sunitinib, and regorafenib, and with low P-glycoprotein expression.
Detailed description
The survival outcomes of patients with advanced and/or metastatic gastrointestinal stromal tumors (GIST) have markedly improved with application of imatinib, the KIT tyrosine kinase inhibitor (GlivecTM, Novartis) for the treatment. Recently, sunitinib (SuteneTM, Pfizer) and regorafenib (StivargaTM, Bayer) have shown efficacy in patients with disease progression on imatinib as second- and third-line treatment, respectively. However, most patients develop acquired resistance to KIT tyrosine kinase inhibitors and show disease progression.
While cytotoxic chemotherapeutic agents were expected to have minimal antitumor effect on GIST, recent preclinical studies have shown that 37 out of 89 different chemotherapeutic agents have shown antitumor effects on at least one or more GIST cell lines. Especially, topoisomerase II inhibitors, paclitaxel, bortezomib have shown promising results. Based on the results, the investigators conducted a single center, single arm phase II study to evaluate efficacy and safety of paclitaxel for patients with GIST who failed to prior imatinib and sunitinib. Overall efficacy was modest, although patients with low P-glycoprotein expression showed better efficacy compared to those with high P-glycoprotein expression. Subsequently, the investigators performed another phase 2 trial to evaluate efficacy of paclitaxel for previously treated GIST patients with low P-glycoprotein expression and have met the primary endpoint.
Liporaxel is an oral formulation of paclitaxel, and showed high bioavailability, safety, and antitumor effect from non-clinical studies. From a phase 1 clinical trial for metastatic solid tumor patients, Liporaxel showed adequate PK/PD profiles. Compared to intravenous paclitaxel, administration is relatively easier and has better safety in terms of hypersensitivity reaction which is caused by the admixture of intravenous formulation of paclitaxel. From a multicenter phase 3 trial conducted in South Korea for patients with advanced gastric adenocarcinoma who failed on first-line palliative chemotherapy (DREAM trial), Liporaxel showed non-inferiority compared to intravenous paclitaxel in terms of both safety and efficacy, and it is currently approved for the second-line treatment in advanced gastric cancer. Also, Liporaxel proved safety and potential efficacy in patients with HER2 negative metastatic breast cancer as first-line chemotherapy, and currently multicenter phase 3 trial in ongoing (OPTIMAL3 trial).
The purpose of this study is to evaluate safety and efficacy of Liporaxel for patients with GIST who failed on prior standard treatments, including imatinib, sunitinib, and regorafenib, and with low P-glycoprotein expression.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Oral Paclitaxel (Liporaxel) | Experimental | Drug: LiporaxelLiporaxel 200mg/m2 twice daily orally on day 1, 8, and 15 every 4 weeks (1 cycle = 4 weeks) |
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 metastatic and/or advanced GIST with CD117(+), DOG-1(+), or mutation in KIT or PDGFRα gene
- Failed (progressed and/or intolerable) after prior treatments for GIST, including at least imatinib and sunitinib, regorafenib.
- Adequate tissue obtained after treatment failure to imatinib, sunitinib, and regorafenib for P-glycoprotein immunohistochemistry (IHC) analysis, and showed P-glycoprotein expression of less than 6. (For patients with PDGFRα D842V mutation or other subtypes with poor response to tyrosine kinase inhibitors, tumor tissue obtained at any period can be used.)
- Eastern Cooperative Oncology Group (ECOG) performance status 0~2
Key exclusion criteria (16 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
- Diabetes mellitus with clinically significant peripheral artery disease
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 |
|---|---|---|
| LiporaxelLiporaxel 200mg/m2 twice daily orally on day 1, 8, and 15 every 4 weeks (1 cycle = 4 weeks) | Drug | Oral Paclitaxel (Liporaxel) |
Primary outcome
16 week disease control rate
Time frame: 16 weeks
according to Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1)
4 secondary outcomes
Progression-free survival
Time frame: until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 36 months
Overall survival
Time frame: through study completion, an average of 3 years
Objective response rate
Time frame: every 4 weeks for the initial two evaluation, then every 8 weeks
Adverse event assessed by NCI-CTCAE Version 5.0
Time frame: until 28 days from the last administration of the investigational product
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.
- Individual participant dataSharing planned: No
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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 Liporaxel 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 16 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?
16 week disease control rate (time frame: 16 weeks). 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.