A Multicenter, Open-label Clinical Study to Evaluate the Efficacy and Safety of NB003 in Patients With Advanced Gastrointestinal Stromal Tumors (GIST) (GISTAR-1)

A Multicenter, Open-label Clinical Study to Evaluate the Efficacy and Safety of NB003 in Patients With Advanced Gastrointestinal Stromal Tumors (GIST) (GISTAR-1)

Gastrointestinal Stromal Tumor

Not yet recruiting

2/3

Start Date 04/01/2026

Age of Trial (yrs) -.1

Gleevec-resistant

KIT/PDGFRA inhibitor

Inhibit KIT and PDGFRa

Specifically GIST and only GIST

NB003-04

Ningbo Newbay Technology Development Co., Ltd

Zhi Zhang +86-21-68880576 zzhang@newbaypharma.com

Randomized

Oral

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.

Inclusion Criteria (Partial - Use NCT number link to see full criteria at clinicaltrials.gov)

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.