Efficacy and Safety of NB003 in Patients With Advanced Gastrointestinal Stromal Tumors (GIST)

NCT07379047 | Not Yet Recruiting Phase 2

• 1 other identifier: NB003-04
• Study Type: interventional
• Target: 255
• Locations: 2 countries
• Sites: 32

Brief 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). 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

Advanced; GIST - Gastrointestinal Stromal Tumor

Outcome Measures

Primary Outcomes (2)

• Progression-free Survival (PFS)

  for part 1

  From randomization until database cut-off, approximately 28 months

• Objective response rate (ORR)

  for part 2

  approximately 24 months since the first subject enrolled

Secondary Outcomes (8)

• Objective response rate (ORR)

  From randomization until database cut-off, approximately 28 months

• Progression-free survival (PFS)

  approximately 24 months since the first subject enrolled

• Duration of Response(DOR)

  approximately 24 months since the first subject enrolled

• Duration of Response(DOR)

  approximately 28 months since the first subject enrolled

• Overall Survival(OS)

  approximately 24-36 months

Study Arms (3)

NB003

EXPERIMENTAL

part1 treated arm

Drug: NB003

Regorafenib

ACTIVE COMPARATOR

160 mg QD in 28-day cycles.

Drug: NB003; Drug: regorafenib

NB003（part 2）

EXPERIMENTAL

part 2 single arm

Drug: NB003

Interventions

NB003 DRUG

15 mg BID on a continuous schedule in 28-day cycles.

NB003; NB003（part 2）; Regorafenib

regorafenib DRUG

Regorafenib is an approved multi-kinase inhibitor administered orally at a dose of 160 mg once daily (QD).

Regorafenib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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).

You may not qualify if:

• 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:
• 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%.
• 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.

Sponsors & Collaborators

• Ningbo Newbay Technology Development Co., Ltd: lead

Study Sites (32)

The First Affiliated Hospital of Anhui Medical University

Hefei, Anhui, China

Location

The Second Hospital of Anhui Medical University

Hefei, Anhui, China

Location

Beijing Cancer Hospital

Beijing, Beijing Municipality, China

Location

The First Affiliated Hospital of Chongqing Medical University

Chongqing, Chongqing Municipality, China

Location

The First Affiliated Hospital of Fujian Medical University

Fuzhou, Fujian, China

Location

The First Affiliated Hospital of Sun Yat-sen University

Guangzhou, Guandong, China

Location

Sun Yat-sen University Cancer Center

Guangzhou, Guangdong, China

Location

Guangxi Medical University Affiliated Cancer Hospital

Nanning, Guangxi, China

Location

The Affiliated Hospital of Guizhou Medical University

Guiyang, Guizhou, China

Location

The Fourth Hospital of Hebei Medical University

Shijiazhuang, Hebei, China

Location

Cancer Hospital Affiliated to Harbin Medical University

Harbin, Heilongjiang, China

Location

Henan Cancer Hospital

Zhengzhou, Henan, China

Location

The First Affiliated Hospital of Zhengzhou University

Zhengzhou, Henan, China

Location

Hubei Cancer Hospital

Wuhan, Hubei, China

Location

Union Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, Hubei, China

Location

Xiangya Hospital, Central South University

Changsha, Hunan, China

Location

Jiangsu Cancer Hospital

Nanjing, Jiangsu, China

Location

Jiangsu Province Hospital

Nanjing, Jiangsu, China

Location

The First Affiliated Hospital of Nanchang University

Nanchang, Jiangxi, China

Location

Jilin Cancer Hospital

Changchun, Jilin, China

Location

Shandong Cancer Hospital

Jinan, Shandong, China

Location

Shandong Provincial Hospital

Jinan, Shandong, China

Location

The Affiliated Hospital of Qingdao University

Qingdao, Shandong, China

Location

Fudan University Shanghai Cancer Center

Shanghai, Shanghai Municipality, China

Location

Renji Hospital Affiliated to Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, China

Location

Shanxi Cancer Hospital

Taiyuan, Shanxi, China

Location

Sichuan Cancer Hospital

Chengdu, Sichuan, China

Location

West China Hospital of Sichuan University

Chengdu, Sichuan, China

Location

Sir Run Run Shaw Hospital , affiliated with the Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Location

The First Affiliated Hospital of Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Location

Asan Medical Center

Seoul, South Korea

Location

Samsung Medical Center

Seoul, South Korea

Location

MeSH Terms

Conditions

Gastrointestinal Stromal Tumors

Interventions

regorafenib

Condition Hierarchy (Ancestors)

Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Digestive System Diseases; Gastrointestinal Diseases

Central Study Contacts

Zhi Zhang

CONTACT

+86-21-68880576; zzhang@newbaypharma.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 23, 2026
• First Posted: January 30, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): August 1, 2028
• Study Completion (Estimated): September 1, 2028
• Last Updated: April 24, 2026

Record last verified: 2026-04

Locations

CN (30); KR (2)