GIST - Gastrointestinal Stromal TumorPhase 2/3GISTAR-1NCT07379047
Efficacy and Safety of NB003 in Patients With Advanced Gastrointestinal Stromal Tumors (GIST)
A Phase 2/3 study testing NB003 and regorafenib in people with GIST - Gastrointestinal Stromal Tumor and Advanced, run by Ningbo Newbay Technology Development Co., Ltd at 32 sites in 2 countries.
Trial summary
NB003-04 is a phase II/III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, and pharmacokinetic (PK) profile of NB003 in patients with gastrointestinal stromal tumors aged 18 years and above (or the legal adult age of consent per local regulations, whichever is older). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- GIST - Gastrointestinal Stromal Tumor, Advanced
- What is being tested?
- NB003 and regorafenib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 255 participants
- Where
- 32 sitesin 2 countries
- Study dates
- 11 May 2026to 1 Sep 2028
- Phase
- Phase 2/3
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
- Participants are >=18 years of age (or the legal adult age as per local regulations, whichever is older) at the time of signing the ICF.
- Participants, or legally authorized representatives permitted by local regulations, provide written informed consent for participation in the study.
- Participants who have histologically confirmed locally advanced, unresectable, or metastatic GIST.
- Part 1: Patients who have failed prior treatment with imatinib (including adjuvant therapy) and sunitinib for GIST due to disease progression or intolerance. Participants should also have no prior use of other TKI drugs.
- Part 2: Patients who have failed prior treatment with imatinib (including adjuvant therapy) for GIST due to disease progression or intolerance. Participants should also have no prior use of other TKI drugs.
- Participants with confirmed KIT gene mutation based on local or central laboratory molecular pathology reports. Mutation status must be determined using tissue-based PCR or DNA sequencing methods. The report should include results on the presence or absence of KIT exon 9/11/17 mutations for randomization stratification in Part 1 and efficacy-related analyses throughout the study. The molecular pathology report indicating KIT mutation status shall be submitted to the medical monitor for review during the screening period. If a local molecular pathology report is unavailable or provides insufficient information, archived tumor tissue samples or fresh biopsy samples must be provided for central laboratory confirmation of mutation status prior to enrollment.
Show all 22Show fewer
- Participants with at least one measurable lesion according to mRECIST.
- Participants with an ECOG PS of 0 to 1.
- Tumor sample requirement: Archival tumor samples in the form of formalin-fixed paraffin-embedded (FFPE) tissue sections or FFPE blocks obtained prior to enrollment, or tissue samples from a tumor biopsy (excisional, core needle, or fine-needle aspiration).
- Participants with an expected life expectancy of >=12 weeks.
- Participants shall have adequate organ and bone marrow function. Transfusions and/or treatment with erythropoietin and/or granulocyte colony stimulating factor/granulocyte macrophage colony stimulating factor (G-CSF/GM-CSF) are not permitted within 14 days prior to the screening laboratory blood draw under any circumstances. The criteria are defined as follows:
- Hemoglobin >=9 g/dL (5.59 mmol/L).
- Absolute neutrophil count (ANC) >=1.5 x 10^9/L (1500/mm^3).
- Platelet count >=100 x 10^9/L (100,000/mm^3).
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) <= 2.5 x upper limit of normal (ULN) (for participants without liver metastases); AST and ALT <= 5 x ULN (for participants with liver metastases).
- Total bilirubin <=1.5 x ULN. Participants with a confirmed diagnosis of Gilbert's syndrome (persistent or recurrent unconjugated hyperbilirubinemia in the absence of hemolysis or hepatic pathology) may be enrolled if total bilirubin <=3 x ULN.
- Creatinine clearance >=60 mL/min as calculated by the Cockcroft-Gault formula or 24-hour urine collection.
- International normalized ratio (INR) or prothrombin time (PT) and activated partial thromboplastin time (aPTT) <=1.5 x ULN; however, if a participant is receiving anticoagulant therapy, PT or aPTT within the therapeutic range of the anticoagulant is acceptable.
- Male participants: Male participants must agree to use contraception as detailed in Appendix 9 during the treatment period and for at least 6 months after the last dose of study treatment and refrain from donating sperm during this period.
- Female participants: A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:
- She is not a woman of childbearing potential (WOCBP) as defined in the Appendix 9; or ii) She is a WOCBP and agrees to follow the contraceptive guidance outlined in the Appendix 9 during the treatment period and for at least 6 months after the last dose of study treatment.
- WOCBP must have a negative serum pregnancy test result during screening within 14 days prior to enrollment.
Who cannot join
- Part 1: Participants who have received prior treatment with NB003 or regorafenib. Part 2: Participants who have received prior treatment with NB003 or sunitinib.
- Participants who have received any systemic anti-tumor therapy within 7 days prior to enrollment.
- Participants who have undergone major surgery (excluding vascular access placement, tumor biopsy, and feeding tube placement) or major palliative interventions (such as transarterial chemoembolization) within 4 weeks prior to enrollment.
- Participants who have received radiation therapy to >30% of the bone marrow or extensive radiation therapy within 4 weeks prior to enrollment.
- Active infection, including active hepatitis B [defined as detectable HBV-DNA by local laboratory. Participants who are HBsAg positive or HBcAb positive at screening should have HBV-DNA tested] and active hepatitis C [defined as detectable HCV-RNA by local laboratory. Participants who are HCV antibody positive at screening should have HCV-RNA tested].
- Any of the following cardiac-related criteria:
Show all 25Show fewer
- Uncontrolled persistent (>4 weeks) hypertension (>140/90 mmHg).
- New York Heart Association (NYHA) Class (Appendix 11) III and IV heart disease, active ischemia, or other uncontrolled cardiac conditions such as angina.
- QTc interval Corrected QT interval using Fridericia's formula (QTcF) >=470 ms (based on 3 ECG results) or history of long QT syndrome.
- Resting ECG showing any clinically significant abnormalities in rhythm, conduction, or morphology (e.g., complete left bundle branch block, atrioventricular block third degree, atrioventricular block second degree, PR interval >250 ms).
- Left ventricular ejection fraction (LVEF) <50%.
- Any unresolved prior treatment toxicity greater than CTCAE Grade 1 at the start of study treatment, with the exception of alopecia, hemoglobin (see Inclusion Criterion #10), Grade 2 hypothyroidism stable on hormone replacement therapy, and Grade 2 pre-existing treatment-related neuropathy.
- Participants who have experienced a National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 Grade 3 or higher bleeding event within 4 weeks prior to enrollment.
- Participants who have experienced an arterial thrombotic or embolic event (e.g., cerebrovascular accident, including transient ischemic attack) within 6 months prior to enrollment, or a venous thrombotic event (including pulmonary embolism or deep vein thrombosis) within 3 months prior to enrollment.
- Participants who have experienced a seizure for any reason within 6 months prior to enrollment.
- Participants with known bleeding disorders or risk of intracranial hemorrhage (e.g., unexcised or unrepaired cerebral aneurysm, cerebrovascular malformation), or history of intracranial hemorrhage within 1 year prior to randomization.
- Participants with unhealed wounds, active ulceration, gastrointestinal perforation, or fracture within 3 months prior to enrollment.
- Participants with brain metastases.
- History of pancreatitis with prior tyrosine kinase inhibitors use, or current evidence of mild to moderate pancreatitis.
- Refractory nausea and vomiting, chronic gastrointestinal disease, inability to swallow the formulation, or significant bowel resection that may affect adequate absorption of NB003 and regorafenib.
- Any other clinically significant comorbidity, such as uncontrolled lung disorder, active infection, uncontrolled pericardial effusion, uncontrolled pleural effusion, or any other condition that, in the investigator's judgment, may affect protocol compliance, interfere with interpretation of study results, or increase participant safety risk.
- Participants judged by the investigator to be unwilling or unable (including incompetence) to comply with study procedures, restrictions, and requirements.
- Participants currently using (or unable to discontinue at least 2 weeks prior to enrollment) drugs or herbal supplements known to be strong inhibitors or inducers of CYP3A4, and other prohibited concomitant medications (Appendix 3).
- History of hypersensitivity to the active or inactive ingredients of NB003, regorafenib, or drugs with similar chemical structure or class to NB003 or regorafenib.
- History of other primary neoplasm malignant diagnosed or requiring treatment within 3 years prior to enrollment. The following prior malignancies are not exclusion criteria: completely resected basal cell and squamous cell skin cancers, cured localized prostate cancer, cured superficial or non-muscle invasive bladder cancer, and completely resected carcinoma in situ of any site.
Trial locations and contacts
32 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
32 sites in 2 countries
China30 sites, 30 recruiting
- Recruiting
Beijing Cancer Hospital
Beijing, Beijing Municipality
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Jilin Cancer Hospital
Changchun, Jilin
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Xiangya Hospital, Central South University
Changsha, Hunan
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Sichuan Cancer Hospital
Chengdu, Sichuan
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
West China Hospital of Sichuan University
Chengdu, Sichuan
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
The First Affiliated Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
The First Affiliated Hospital of Fujian Medical University
Fuzhou, Fujian
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
The First Affiliated Hospital of Sun Yat-sen University
Guangzhou, Guandong
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Sun Yat-sen University Cancer Center
Guangzhou, Guangdong
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
The Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Sir Run Run Shaw Hospital , affiliated with the Zhejiang University School of Medicine
Hangzhou, Zhejiang
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
The First Affiliated Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Cancer Hospital Affiliated to Harbin Medical University
Harbin, Heilongjiang
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Recruiting
The Second Hospital of Anhui Medical University
Hefei, Anhui
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Shandong Cancer Hospital
Jinan, Shandong
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Shandong Provincial Hospital
Jinan, Shandong
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
The First Affiliated Hospital of Nanchang University
Nanchang, Jiangxi
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Jiangsu Cancer Hospital
Nanjing, Jiangsu
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Jiangsu Province Hospital
Nanjing, Jiangsu
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Guangxi Medical University Affiliated Cancer Hospital
Nanning, Guangxi
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Recruiting
The Affiliated Hospital of Qingdao University
Qingdao, Shandong
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Fudan University Shanghai Cancer Center
Shanghai, Shanghai Municipality
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Renji Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
The Fourth Hospital of Hebei Medical University
Shijiazhuang, Hebei
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Shanxi Cancer Hospital
Taiyuan, Shanxi
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Hubei Cancer Hospital
Wuhan, Hubei
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Recruiting
Union Hospital Tongji Medical College Huazhong University of Science and Technology
Wuhan, Hubei
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
Henan Cancer Hospital
Zhengzhou, Henan
Study central contact, +86-21-68880576, NB003@newbaypharma.com
- Not yet recruiting
The First Affiliated Hospital of Zhengzhou University
Zhengzhou, Henan
Study central contact, +86-21-68880576, NB003@newbaypharma.com
South Korea2 sites, 2 recruiting
- Not yet recruiting
Asan Medical Center
Seoul
Study central contact, +82230105935, NB003@newbaypharma.com
- Not yet recruiting
Samsung Medical Center
Seoul
Study central contact, +82234101754, NB003@newbaypharma.com
About this study
Official title: A Multicenter, Open-label Clinical Study to Evaluate the Efficacy and Safety of NB003 in Patients With Advanced Gastrointestinal Stromal Tumors (GIST)
NB003-04 is a phase II/III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, and pharmacokinetic (PK) profile of NB003 in patients with gastrointestinal stromal tumors aged 18 years and above (or the legal adult age of consent per local regulations, whichever is older). Participants who are eligible for this study are those who have experienced disease progression or documented intolerance following treatment with either imatinib and sunitinib or following treatment with imatinib.
This study consists of two parts. Part 1 (hereinafter referred to as Part 1) compares the efficacy of NB003 versus regorafenib in patients who need a third-line therapy for GIST who have failed sequential therapy with imatinib and sunitinib. Part 2 (hereinafter referred to as Part 2) evaluates the efficacy of NB003 in patients who need a second-line therapy for GIST who have failed treatment with imatinib.
- Conditions
- GIST - Gastrointestinal Stromal Tumor, Advanced
- Being tested
- NB003, regorafenib
- 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.
NB003Drug
15 mg BID on a continuous schedule in 28-day cycles.
regorafenibDrug
Regorafenib is an approved multi-kinase inhibitor administered orally at a dose of 160 mg once daily (QD).
| Study group | Role | What they receive |
|---|---|---|
| NB003 | Experimental | part1 treated arm Receives: Drug: NB003 |
| Regorafenib | Active comparator | 160 mg QD in 28-day cycles. Receives: Drug: NB003; Drug: regorafenib |
| NB003（part 2） | Experimental | part 2 single arm Receives: Drug: NB003 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progression-free Survival (PFS)
for part 1
Measured: From randomization until database cut-off, approximately 28 months
Objective response rate (ORR)
for part 2
Measured: approximately 24 months since the first subject enrolled
Secondary outcomes (8)
Objective response rate (ORR)
for part 1
Measured: From randomization until database cut-off, approximately 28 months
Progression-free survival (PFS)
for part 2
Measured: approximately 24 months since the first subject enrolled
Duration of Response(DOR)
for part 2
Measured: approximately 24 months since the first subject enrolled
Duration of Response(DOR)
for part 1
Measured: approximately 28 months since the first subject enrolled
Overall Survival(OS)
for part 1
Measured: approximately 24-36 months
Disease Control Rate (DCR)
for part 1
Measured: Approximately 28 months since first subject enrolled
Overall Survival(OS)
for part 2
Measured: approximately 24-36 months
Disease Control Rate (DCR)
for part 2
Measured: Approximately 24 months since first subject enrolled
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2026
- Study start11 May 2026
- Primary completion1 Aug 2028 (estimated)
- Study completion1 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GIST - Gastrointestinal Stromal TumorStudies of the same condition, open and under way.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 255 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ningbo Newbay Technology Development Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07379047
- Sponsor's protocol ID
- NB003-04
- Acronym
- GISTAR-1
- Status last verified
- Jun 2026
- First submitted
- 23 Jan 2026
- Last updated
- 10 Jun 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided