Metastatic Gastrointestinal Stromal Tumors (GIST)Phase 2NCT06772233
A Study of Regorafenib Combined With Envafolimab for Metastatic Gastrointestinal Stromal Tumors With Kit Gene Exon 17 Mutation That Failed Standard Treatment
A Phase 2 study testing regorafenib combined with envafolimab in people with Metastatic Gastrointestinal Stromal Tumors (GIST), run by Peking University at 1 site in China.
Trial summary
This study is a multicenter, prospective, randomized controlled Phase II clinical trial. The primary endpoint is to evaluate the efficacy and safety of regorafenib combined with envafolimab compared to physician's choice in patients with metastatic gastrointestinal stromal tumors harboring KIT exon 17 mutations who… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Metastatic Gastrointestinal Stromal Tumors (GIST)
- What is being tested?
- regorafenib combined with envafolimab
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteChina
- Study dates
- 30 Jul 2025to 30 Jul 2028
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years, no gender restriction;
- Pathologically confirmed gastrointestinal stromal tumor (GIST);
- At least one measurable target lesion according to mRECIST v1.1 criteria (non-lymph node lesion with a long axis ≥ 1.0 cm or long axis ≥ 2 slide thicknesses); imaging assessment within 14 days before the first dose;
- Progression or intolerance after treatment with imatinib, sunitinib, regorafenib, or ripretinib;
- Genetic testing includes primary or secondary KIT exon 17 mutation;
- Adequate organ and bone marrow function, defined as follows:
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- Hematology: Absolute neutrophil count (ANC) ≥ 1.5 × 10^9/L; platelet count (PLT) ≥ 75 × 10^9/L; hemoglobin (HGB) ≥ 9.0 g/dL. No use of granulocyte colony-stimulating factor (G-CSF), granulocyte-macrophage colony-stimulating factor (GM-CSF), red blood cell transfusion, or platelet transfusion within 14 days before testing; Liver and kidney function: For patients without liver metastasis, total bilirubin (TBIL) ≤ 1.5 × upper limit of normal (ULN); alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 × ULN. For patients with liver metastasis: TBIL ≤ 1.5 × ULN; ALT and AST ≤ 5 × ULN. Kidney function: serum creatinine (Scr) ≤ 1.5 × ULN; Adequate coagulation function, defined as international normalized ratio (INR) ≤ 1.5 or prothrombin time (PT) ≤ 1.5 × ULN; if the patient is on anticoagulant therapy, PT should be within the intended range of the anticoagulant;
- Provide 15 paraffin-embedded tissue sections before enrollment for immune microenvironment testing;
- ECOG PS score 0-2;
- Signed informed consent.
Who cannot join
- Unable to tolerate previous regorafenib treatment or previously received immune checkpoint inhibitors;
- Pregnant or breastfeeding;
- Expected survival less than 3 months;
- Underwent major surgery or experienced significant trauma within 4 weeks before the first blood draw during the screening period, or expected to need major surgery during the study;
- Currently have active ulcers or gastrointestinal bleeding;
- History of interstitial lung disease or non-infectious pneumonia; history of active tuberculosis;
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- Uncontrolled pleural effusion, pericardial effusion, or ascites requiring repeated drainage;
- Clinically diagnosed autoimmune disease; HIV or HCV positive; HBV-DNA exceeding laboratory normal range; acute CMV infection;
- Patients with central nervous system metastasis;
- Patients with other malignancies within the past five years;
- Immunosuppressed subjects, including those with known immunodeficiency; currently using systemic steroids (except for recent or current use of inhaled steroids);
- Uncontrolled hypertension: Despite aggressive antihypertensive therapy, sequential measurements show systolic blood pressure ≥160 mmHg and diastolic blood pressure ≥100 mmHg on three consecutive occasions;
- Subjects deemed by the investigator to be unable or unwilling to comply with the study protocol requirements.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Beijing Cancer Hospital, 52 Fucheng Road
Beijing, Beijing Municipality, 100142
Jian Li, +861088196088, oncogene@163.com
About this study
Official title: A Phase II, Open-label, Multicenter, Randomized Controlled Clinical Trial Comparing Regorafenib Combined With Envafolimab to Physician's Choice in Patients With Metastatic Gastrointestinal Stromal Tumors Harboring KIT Exon 17 Mutations Refractory to Standard Treatment.
This study is a multicenter, prospective, randomized controlled Phase II clinical trial. The primary endpoint is to evaluate the efficacy and safety of regorafenib combined with envafolimab compared to physician's choice in patients with metastatic gastrointestinal stromal tumors harboring KIT exon 17 mutations who have failed standard treatments.
Read the detailed description
This multicenter, prospective, randomized, controlled phase II clinical trial aims to explore the efficacy and safety of regorafenib combined with envafolimab in treating metastatic GIST with KIT exon 17 mutation that has failed standard treatment. It also seeks to investigate the correlation between the immune microenvironment and the efficacy of immunotherapy.
The study includes patients with histologically confirmed advanced metastatic GIST containing the KIT exon 17 mutation, requiring at least one evaluable lesion. Using a block randomization method, the study is open-label and assigns patients to either the treatment group or the control group in a 1:1 ratio. The treatment group receives regorafenib combined with envafolimab, while the control group continues physician's choice until disease progression, intolerable toxicity, or voluntary withdrawal from the trial.
The governing principle for physician decision-making in the control group was selection based on prior medication tolerability, genotype, etc.:
- Continued maintenance therapy with the originally effective TKI at the same dose: The patient achieved at least stable disease (SD) or partial response (PR) during prior treatment, with progression-free survival (PFS) exceeding 6 months, and the adverse reactions were tolerable.
- Combination therapy with two TKIs: Different drugs were selected for maintenance based on distinct actionable mutations identified in the patient's tissue or peripheral blood genetic testing, OR a combination of drugs previously effective and well-tolerated was used, OR the combination therapy was chosen by referencing past tolerability.
A total of 100 patients are planned to be enrolled, with imaging assessments conducted at baseline and every two months during treatment.
- Being tested
- regorafenib combined with envafolimab, Control Group
- Keywords
- gastrointestinal stromal tumors, KIT exon 17 mutations
- 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.
regorafenib combined with envafolimabDrug
-Regorafenib: Specification: 40 mg/tablet Dosage: 120 mg, taken orally once daily for 3 weeks, followed by 1 week off, until disease progression or intolerable toxicity occurs.
- Envafolimab:
Specification: 200 mg/vial Dosage: 200 mg, administered via subcutaneous injection once every 2 weeks, until disease progression or intolerable toxicity occurs.
Control GroupDrug
The governing principle for physician decision-making in the control group was selection based on prior medication tolerability, genotype, etc.: a. Continued maintenance therapy with the originally effective TKI at the same dose: The patient achieved at least stable disease (SD) or partial response (PR) during prior treatment, with progression-free survival (PFS) exceeding 6 months, and the adverse reactions were tolerable. b. Combination therapy with two TKIs: Different drugs were selected for maintenance based on distinct actionable mutations identified in the patient's tissue or peripheral blood genetic testing, OR a combination of drugs previously effective and well-tolerated was used, OR the combination therapy was chosen by referencing past tolerability.
| Study group | Role | What they receive |
|---|---|---|
| regorafenib combined with envafolimab | Experimental | * Regorafenib: Specification: 40 mg/tablet Dosage: 120 mg, taken orally once daily for 3 weeks, followed by 1 week off, until disease progression or intolerable toxicity occurs.
* Envafolimab: Specification: 200 mg/vial Dosage: 200 mg, administered via subcutaneous injection once every 2 weeks, until disease progression or intolerable toxicity occurs. Receives: Drug: regorafenib combined with envafolimab |
| Physician's choice | Active comparator | The governing principle for physician decision-making in the control group was selection based on prior medication tolerability, genotype, etc.:
1. Continued maintenance therapy with the originally effective TKI at the same dose: The patient achieved at least stable disease (SD) or partial response (PR) during prior treatment, with progression-free survival (PFS) exceeding 6 months, and the adverse reactions were tolerable.
2. Combination therapy with two TKIs: Different drugs were selected for maintenance based on distinct actionable mutations identified in the patient's tissue or peripheral blood genetic testing, OR a combination of drugs previously effective and well-tolerated was used, OR the combination therapy was chosen by referencing past tolerability. Receives: Drug: Control Group |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progression-Free Survival (PFS)
Assessed by the investigator according to mRECIST 1.1 criteria
Measured: From enrollment to disease progression or death, whichever came first, assessed up to 2 years
Secondary outcomes (3)
Overall Survival (OS)
Overall Survival (OS): Assessed by the investigator according to mRECIST 1.1 criteria.
Measured: Overall Survival (OS): from enrollment to death, assessed up to 2 years
Objective Response Rate (ORR)
Objective Response Rate (ORR): Assessed by the investigator according to mRECIST 1.1 criteria.
Measured: assessed up to 2 years
Incidence of Treatment-Emergent Adverse Events (TEAEs)
The safety parameters of this study include clinical symptoms, vital signs, physical examinations, and laboratory tests (routine, urinalysis, blood biochemistry, thyroid function, coagulation function, etc.). Adverse events (AEs) observed will be evaluated according to version 5.0 of the NCI-CTCAE, including type, incidence, severity, onset and end times, whether they are serious adverse events, and their relationship to the study drug.
The assessment of postoperative complications includes type, incidence, and grading (based on the Clavien-Dindo classification system).
Measured: assessed up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2025
- Study start30 Jul 2025 (estimated)
- Primary completion30 Jul 2027 (estimated)
- Study completion30 Jul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Metastatic Gastrointestinal Stromal Tumors (GIST)Studies of the same condition, open and under way.
- More trials run by Peking UniversityThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jian Li, Dr., Principal investigator, Peking University Cancer Hospital & Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06772233
- Sponsor's protocol ID
- GIST 006
- Status last verified
- Jul 2025
- First submitted
- 30 Dec 2024
- Last updated
- 31 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Open-label, Multicenter, Randomized Controlled Clinical Trial Comparing Regorafenib Combined With Envafolimab to Physician's Choice in Patients With Metastatic Gastrointestinal Stromal Tumors Harboring KIT Exon 17 Mutations Refractory to Standard Treatment.
- ClinicalTrials.gov ID
- NCT06772233
- Acronym / sponsor ID
- GIST 006
- Keywords
- gastrointestinal stromal tumors, KIT exon 17 mutations
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Peking UniversityUniversity, hospital or other
- Study start
- 30 Jul 2025 (estimated)
- Primary completion
- 30 Jul 2027 (estimated)
- Study completion
- 30 Jul 2028 (estimated)
- First posted
- 13 Jan 2025 (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.
This study is a multicenter, prospective, randomized controlled Phase II clinical trial. The primary endpoint is to evaluate the efficacy and safety of regorafenib combined with envafolimab compared to physician's choice in patients with metastatic gastrointestinal stromal tumors harboring KIT exon 17 mutations who have failed standard treatments.
Detailed description
This multicenter, prospective, randomized, controlled phase II clinical trial aims to explore the efficacy and safety of regorafenib combined with envafolimab in treating metastatic GIST with KIT exon 17 mutation that has failed standard treatment. It also seeks to investigate the correlation between the immune microenvironment and the efficacy of immunotherapy.
The study includes patients with histologically confirmed advanced metastatic GIST containing the KIT exon 17 mutation, requiring at least one evaluable lesion. Using a block randomization method, the study is open-label and assigns patients to either the treatment group or the control group in a 1:1 ratio. The treatment group receives regorafenib combined with envafolimab, while the control group continues physician's choice until disease progression, intolerable toxicity, or voluntary withdrawal from the trial.
The governing principle for physician decision-making in the control group was selection based on prior medication tolerability, genotype, etc.:
- Continued maintenance therapy with the originally effective TKI at the same dose: The patient achieved at least stable disease (SD) or partial response (PR) during prior treatment, with progression-free survival (PFS) exceeding 6 months, and the adverse reactions were tolerable.
- Combination therapy with two TKIs: Different drugs were selected for maintenance based on distinct actionable mutations identified in the patient's tissue or peripheral blood genetic testing, OR a combination of drugs previously effective and well-tolerated was used, OR the combination therapy was chosen by referencing past tolerability.
A total of 100 patients are planned to be enrolled, with imaging assessments conducted at baseline and every two months during treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| regorafenib combined with envafolimab | Experimental | Drug: regorafenib combined with envafolimab* Regorafenib: Specification: 40 mg/tablet Dosage: 120 mg, taken orally once daily for 3 weeks, followed by 1 week off, until disease progression or intolerable toxicity occurs. * Envafolimab: Specification: 200 mg/vial Dosage: 200 mg, administered via subcutaneous injection once every 2 weeks, until disease progression or intolerable toxicity occurs. |
| Physician's choice | Active comparator | Drug: Control GroupThe governing principle for physician decision-making in the control group was selection based on prior medication tolerability, genotype, etc.: 1. Continued maintenance therapy with the originally effective TKI at the same dose: The patient achieved at least stable disease (SD) or partial response (PR) during prior treatment, with progression-free survival (PFS) exceeding 6 months, and the adverse reactions were tolerable. 2. Combination therapy with two TKIs: Different drugs were selected for maintenance based on distinct actionable mutations identified in the patient's tissue or peripheral blood genetic testing, OR a combination of drugs previously effective and well-tolerated was used, OR the combination therapy was chosen by referencing past tolerability. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Age ≥ 18 years, no gender restriction;
- Pathologically confirmed gastrointestinal stromal tumor (GIST);
- At least one measurable target lesion according to mRECIST v1.1 criteria (non-lymph node lesion with a long axis ≥ 1.0 cm or long axis ≥ 2 slide thicknesses); imaging assessment within 14 days before the first dose;
- Progression or intolerance after treatment with imatinib, sunitinib, regorafenib, or ripretinib;
- Genetic testing includes primary or secondary KIT exon 17 mutation;
Key exclusion criteria (13 listed)
- Unable to tolerate previous regorafenib treatment or previously received immune checkpoint inhibitors;
- Pregnant or breastfeeding;
- Expected survival less than 3 months;
- Underwent major surgery or experienced significant trauma within 4 weeks before the first blood draw during the screening period, or expected to need major surgery during the study;
- Currently have active ulcers or gastrointestinal bleeding;
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 |
|---|---|---|
| regorafenib combined with envafolimab-Regorafenib: Specification: 40 mg/tablet Dosage: 120 mg, taken orally once daily for 3 weeks, followed by 1 week off, until disease progression or intolerable toxicity occurs. * Envafolimab: Specification: 200 mg/vial Dosage: 200 mg, administered via subcutaneous injection once every 2 weeks, until disease progression or intolerable toxicity occurs. | Drug | regorafenib combined with envafolimab |
| Control GroupThe governing principle for physician decision-making in the control group was selection based on prior medication tolerability, genotype, etc.: a. Continued maintenance therapy with the originally effective TKI at the same dose: The patient achieved at least stable disease (SD) or partial response (PR) during prior treatment, with progression-free survival (PFS) exceeding 6 months, and the adverse reactions were tolerable. b. Combination therapy with two TKIs: Different drugs were selected for maintenance based on distinct actionable mutations identified in the patient's tissue or peripheral blood genetic testing, OR a combination of drugs previously effective and well-tolerated was used, OR the combination therapy was chosen by referencing past tolerability. | Drug | Physician's choice |
Primary outcome
Progression-Free Survival (PFS)
Time frame: From enrollment to disease progression or death, whichever came first, assessed up to 2 years
Assessed by the investigator according to mRECIST 1.1 criteria
3 secondary outcomes
Overall Survival (OS)
Time frame: Overall Survival (OS): from enrollment to death, assessed up to 2 years
Objective Response Rate (ORR)
Time frame: assessed up to 2 years
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Time frame: assessed up to 2 years
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 31 Jul 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.
Jian Li, Dr. principal investigator
Peking University Cancer Hospital & Institute
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 regorafenib combined with envafolimab in people with Metastatic Gastrointestinal Stromal Tumors (GIST), run by Peking University at 1 site in China.
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 10 inclusion and 13 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: opening soon, as of the sponsor's last update on 31 Jul 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 China. 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 Jian Li, Dr. (+861088196088, oncogene@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Progression-Free Survival (PFS) (time frame: From enrollment to disease progression or death, whichever came first, assessed up to 2 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 31 Jul 2025.