A Phase II Study of Temozolomide and Survivin Long Peptide Vaccine (SurVaxM) in Patients With Progressing Metastatic Neuroendocrine Carcinomas (NECs)

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Trial Details

Sponsor: Roswell Park Cancer Institute (other)

Phase: 2

Start date: Jan. 11, 2024

Planned enrollment: 60

Trial ID: NCT06202066
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More trial details at ClinicalTrials.gov More info

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Investigational Drug AI Analysis

chevron Show for: SVN53-67/M57-KLH Peptide Vaccine (SurVaxM, DRU-2017-5947)

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Goal: To evaluate whether adding the survivin-targeted vaccine SurVaxM to temozolomide improves disease control in patients with progressing metastatic neuroendocrine tumors or neuroendocrine carcinomas, while characterizing the safety and tolerability of the combination. The primary efficacy focus is 6-month progression-free survival, with additional assessment of radiographic response, survival, immune response, MGMT status, and tumor growth kinetics.

Patients: Adults with measurable, pathologically confirmed metastatic and progressing neuroendocrine tumors of gastrointestinal, pancreatic, or thoracic origin with Ki-67 greater than 20%, including well-differentiated grade 3 NETs, or neuroendocrine carcinoma of any origin except small cell lung cancer. Eligible patients must have radiographic progression within the prior 12 months, prior failure of at least one systemic therapy, ECOG performance status 0–1 or Karnofsky performance status at least 80, adequate marrow, hepatic, and renal function, and tumor tissue positive for survivin by immunohistochemistry. Prior temozolomide is not allowed if the patient progressed on it in the advanced-disease setting, and patients with prior SurVaxM, recent checkpoint inhibitor therapy, active autoimmune disease, significant uncontrolled illness, pregnancy, or high-dose systemic corticosteroid use are excluded.

Design: This is an open-label, single-arm phase IIa treatment study. All enrolled patients receive temozolomide plus SurVaxM; there is no randomization or concurrent control arm, although the study is framed around assessing the added value and safety of the combination relative to historical experience with temozolomide. Patients may continue treatment for up to 1 year in the absence of progression or unacceptable toxicity, with possible continuation of temozolomide at investigator discretion.

Treatments: Temozolomide is given orally once daily on days 1–5 of each 28-day cycle for up to 1 year, provided there is no disease progression or unacceptable toxicity. Temozolomide is an alkylating agent that damages tumor-cell DNA and is an established systemic option used in selected patients with advanced neuroendocrine neoplasms, particularly pancreatic NETs and higher-grade well-differentiated disease. SurVaxM, also known as SVN53-67/M57-KLH, is given subcutaneously with Montanide ISA-51 and sargramostim every 2 weeks for 4 priming doses; patients deriving clinical benefit without progression or unacceptable toxicity may receive additional doses at weeks 24, 36, and 48. SurVaxM is an investigational peptide vaccine targeting survivin, an inhibitor-of-apoptosis protein overexpressed in multiple cancers and associated with adverse prognosis; the vaccine is designed to generate survivin-specific cellular and humoral immune responses against survivin-expressing tumor cells. In prior early-phase glioblastoma studies, SurVaxM was immunogenic and generally well tolerated, with mostly low-grade injection-site reactions and encouraging single-arm survival signals when combined with standard temozolomide-based therapy, but randomized efficacy data are not yet established.

Outcomes: The primary outcomes are progression-free survival at 6 months and incidence of adverse events graded by NCI CTCAE v5.0 through up to 1 year. Secondary outcomes include RECIST v1.1 response and clinical benefit rates at 3, 6, 9, and 12 months; overall survival; time to progression; and anti-survivin IgG titer response, defined as titers greater than 30,000. Exploratory analyses include peripheral blood immune markers associated with response, MGMT status correlated with outcome, and radiographic tumor growth rate before and during treatment.

Burden on patient: The expected patient burden is moderate. Temozolomide is oral and administered on a short 5-day schedule each 28-day cycle, which limits infusion-chair time, but the vaccine component requires repeated clinic visits for subcutaneous injections every 2 weeks during the priming phase and additional booster visits at approximately 6, 9, and 12 months for patients benefiting from therapy. Patients also undergo serial blood collections for safety and immune correlative testing, as well as CT or MRI scans for disease assessment; these procedures are generally consistent with oncology trial monitoring and do not appear to include mandatory fresh tumor biopsies or intensive pharmacokinetic sampling. Overall, the main added burden beyond standard temozolomide-based care is the vaccine visit schedule and immune-monitoring blood draws.

Last updated: May 2026

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Roswell Park Cancer Institute

Buffalo, New York, 14263, United States

[email protected] / 716-845-1300

Status: Recruiting

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