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This is a prospective, multicenter, phase 4, single-arm study designed to evaluate the effectiveness and safety of surufatinib in adult patients with advanced neuroendocrine neoplasms (NENs) treated in routine clinical practice. Approximately 350 patients with histologically or cytologically confirmed advanced NENs and at least one measurable lesion (RECIST 1.1) will receive oral surufatinib, either as monotherapy (300 mg once daily in 4-week cycles) or in combination regimens (250 mg once daily), according to the treating physician's judgment and protocol guidance.
Participants will be followed with regular imaging, laboratory tests, cardiac assessments, and patient-reported outcome questionnaires to monitor tumor response, side effects, quality of life, and treatment adherence. The primary outcomes include progression-free survival, objective response rate, disease control rate, and overall survival; safety will be assessed by the type, frequency, and severity of adverse events. The findings are expected to inform standardized, evidence-based use of surufatinib and help optimize individualized treatment strategies for patients with advanced NENs in the real-world setting.
This is a prospective, multicenter, phase 4, single-arm, open-label study designed to systematically characterize the real-world use of surufatinib in adult patients with advanced neuroendocrine neoplasms (NENs). Surufatinib is an oral small-molecule tyrosine kinase inhibitor with dual antitumor activity through anti-angiogenic and immune-modulating mechanisms, and has shown clinically meaningful efficacy in phase 3 trials in both pancreatic and extra-pancreatic NETs. However, evidence is still limited regarding its effectiveness, safety profile, patterns of use (monotherapy versus combination regimens, line of therapy, cross-line use), and impact on quality of life in routine clinical practice in China.
Approximately 350 patients with histologically or cytologically confirmed advanced NENs and at least one measurable lesion (RECIST 1.1) will be enrolled from multiple centers. According to the treating physician's judgment and protocol guidance, patients will receive oral surufatinib either as monotherapy (300 mg once daily in 4-week cycles) or in combination regimens (250 mg once daily), and treatment will continue until disease progression, unacceptable toxicity, withdrawal of consent, death, or end of study. Tumor assessments, laboratory tests, cardiac monitoring, and patient-reported outcome questionnaires (including generic and NET-specific quality-of-life scales and a medication-adherence scale) will be performed at regular intervals during follow-up. The study will estimate progression-free survival, objective response rate, disease control rate, and overall survival, describe the incidence and severity of treatment-emergent adverse events, and explore how baseline clinicopathological features and treatment patterns are associated with outcomes, with the aim of developing evidence-based, standardized, and individualized clinical pathways for the real-world use of surufatinib in advanced NENs.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Surufatinib Treatment | Experimental | Adult patients with histologically or cytologically confirmed advanced neuroendocrine neoplasms (NENs) will receive oral surufatinib according to routine clinical practice and protocol guidance. Surufatinib will be administered either as monotherapy (300 mg once daily in continuous 4-week cycles) or as part of combination therapy (250 mg once daily) at the discretion of the treating physician. Treatment will continue until radiologic or clinical disease progression, unacceptable toxicity, withdrawal of consent, death, or end of study, whichever occurs first. Concomitant anticancer therapies, dose modifications, and interruptions are permitted and will be managed in accordance with the protocol and local standard of care. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Surufatinib | Drug | Surufatinib (Sulanda, HMPL-012) is an oral small-molecule tyrosine kinase inhibitor that selectively targets VEGFR1-3, FGFR1 and CSF1R, exerting both anti-angiogenic and immune-modulating effects. In this study, surufatinib will be administered as film-coated tablets once daily: 300 mg QD for monotherapy or 250 mg QD when used in combination regimens, according to protocol guidance and the treating physician's judgment. Treatment is given in continuous 4-week cycles and continued until disease progression, unacceptable toxicity, withdrawal of consent, death, or end of study. Dose interruptions and reductions are allowed and will be managed following the protocol and the approved prescribing information. |
| Measure | Description | Time Frame |
|---|---|---|
| Progression Free Survival (PFS) | From the date of enrollment to the date of the first onset of disease progression or death from any cause, whichever comes first. | up to 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Objective response rate (ORR) | CR + PR rate according to the RECIST version 1.1 guidelines. | up to 12 months |
| Disease control rate (DCR) | CR + PR + SD rate according to the RECIST version 1.1 guidelines. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Wenquan Wang, M.D. Ph.D | Contact | +86 21 31587861 | wang.wenquan@zs-hospital.sh.cn | |
| Xu Han, M.D. Ph.D | Contact | +86 21 31587861 | han.xu1@zs-hospital.sh.cn |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Huadong Hospital Affiliated to Fudan University | Recruiting | Shanghai | China |
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| ID | Term |
|---|---|
| D018358 | Neuroendocrine Tumors |
| ID | Term |
|---|---|
| D017599 | Neuroectodermal Tumors |
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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| ID | Term |
|---|---|
| C000717729 | surufatinib |
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Prospective multicenter phase 4, single-group assignment, open-label study of surufatinib in patients with advanced neuroendocrine neoplasms
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This is an open-label study; no masking is implemented for any study personnel or participants.
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| up to 12 months |
| Overall survival (OS) | From the date of enrollment to the date of the first onset of death from any cause | up to 36 months |
| Huashan Hospital Affiliated to Fudan University | Recruiting | Shanghai | China |
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| Renji Hospital affiliated to Shanghai Jiaotong University School of Medicine | Recruiting | Shanghai | China |
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| Shanghai General Hospital shanghai jiao tong university school of medicine | Recruiting | Shanghai | China |
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| D009380 | Neoplasms, Nerve Tissue |