Sponsor: Roswell Park Cancer Institute (other)
Phase: 2
Start date: Jan. 11, 2024
Planned enrollment: 60
SurVaxM (SVN53-67/M57-KLH) is an investigational cancer vaccine studied primarily alongside temozolomide for glioblastoma. Human trial results are available, but the published efficacy evidence comes from small, nonrandomized studies. A randomized, placebo-controlled phase 2b trial, SURVIVE, reached its data cutoff in July 2026; comparative results had not been reported in the sources reviewed. (pmc.ncbi.nlm.nih.gov)
The vaccine contains a modified peptide derived from survivin (BIRC5), a protein associated with tumor-cell survival, linked to keyhole limpet hemocyanin (KLH). It is intended to prompt immune recognition of survivin-expressing cancer cells. Trial participants developed survivin-directed antibodies and CD8⁺ T-cell responses; whether those responses improve clinical outcomes remains to be established. (pmc.ncbi.nlm.nih.gov)
In a single-arm phase 2a study of newly diagnosed glioblastoma, 60 of 63 evaluable patients (95.2%) remained progression-free six months after diagnosis. Median progression-free survival was 11.4 months and median overall survival was 25.9 months, both measured from the first vaccine dose. Participants had also undergone surgery and chemoradiation and received adjuvant temozolomide, so these figures cannot establish how much benefit, if any, SurVaxM added. (pmc.ncbi.nlm.nih.gov)
An earlier phase 1 study in nine patients with recurrent malignant glioma reported one complete and one partial radiologic response; median progression-free and overall survival were 17.6 and 86.6 weeks, respectively. The small sample and lack of a control group limit interpretation. (pmc.ncbi.nlm.nih.gov)
In the phase 2a study, investigators attributed no serious adverse events to SurVaxM or its vaccine additives. The most common adverse event was a mild injection-site reaction; two patients developed localized inflammation of tissue beneath the skin at injection sites, considered most likely related to the Montanide adjuvant. Blood-count changes were attributed to temozolomide. These studies were too small to rule out uncommon risks. (pmc.ncbi.nlm.nih.gov)
Last updated: Sep 2026
Goal: Evaluate the efficacy and safety of adding the survivin-targeted vaccine SurVaxM to temozolomide in progressing metastatic neuroendocrine neoplasms.
Patients: Adults with measurable, survivin-positive, progressing metastatic well-differentiated grade 3 neuroendocrine tumors of gastrointestinal, pancreatic, or thoracic origin, or neuroendocrine carcinoma of any origin except small-cell lung cancer. Patients must have received at least one prior systemic therapy and have ECOG performance status 0–1 or Karnofsky performance status at least 80.
Design: Phase IIa, single-arm study. Although the trial description mentions comparison with temozolomide alone, the listed design has no randomized allocation or temozolomide-only arm.
Treatments: Temozolomide is an oral alkylating agent given daily on days 1–5 of each 28-day cycle for up to one year, with possible continuation at the investigator’s discretion. SurVaxM is an investigational peptide vaccine designed to elicit immune responses against survivin-expressing tumor cells. It is given subcutaneously with Montanide adjuvant and sargramostim every two weeks for four doses; patients benefiting without progression or unacceptable toxicity may receive additional doses at weeks 24, 36, and 48. Small, nonrandomized glioma studies have demonstrated immune responses and reported clinical activity, but have not established that SurVaxM improves outcomes; its efficacy in neuroendocrine neoplasms remains unknown.
Outcomes: Primary measures are progression-free survival at six months and adverse events through one year, graded by CTCAE v5.0. Secondary measures include RECIST v1.1 response and clinical benefit at 3, 6, 9, and 12 months; overall survival; time to progression; and anti-survivin IgG titer response. Exploratory analyses assess immune markers, MGMT status, and radiographic tumor growth rate.
Burden on patient: Moderate. Oral temozolomide can be taken at home, but the initial four vaccine visits occur every two weeks, with up to three later injection visits. Repeated blood collection and CT or MRI scans add monitoring and travel; no mandatory new biopsy or intensive pharmacokinetic sampling is specified.
Last updated: Sep 2026
Inclusion Criteria:
* Age ≥ 18 years of age
* Have a Karnofsky performance status ≥ 80 or Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1 (i.e. the patient must be able to care for himself/ herself with occasional help from others)
* Measurable, pathologically confirmed diagnosis of neuroendocrine tumor of gastrointestinal, pancreatic, or thoracic origin with ki67\>20% (well-differentiated G3 NETs) or neuroendocrine carcinoma of any origin excluding small cell lung carcinoma
* Patients must have documented radiographic progression, determined as clinically significant by the treating provider, within the last twelve months on two CT or MRI scans performed at least four weeks apart per RECIST v1.1 criteria. In the case of retreatment, progression may be defined by the treating provider (e.g., clinical, radiographic, biochemical)
* Patients must have failed at least one prior systemic therapy
* Patients who have been on somatostatin analogues (SSA) may continue to take SSA while on study treatment
* Archival neuroendocrine tumor tissue must test positive for survivin presence by clinical immunohistochemistry prior to study enrollment
* Absolute neutrophil count (ANC) ≥ 1.5 x 10\^9/L (obtained within 14 days prior to enrollment)
* Platelets ≥ 100 x 10\^9/L (obtained within 14 days prior to enrollment)
* Hemoglobin (Hgb) \> 9g/dL (obtained within 14 days prior to enrollment)
* Plasma total bilirubin: ≤ 1.5 x upper limit of normal (ULN) (obtained within 14 days prior to enrollment)
* Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 4 x ULN (obtained within 14 days prior to enrollment)
* Creatinine clearance ≥ 60 mL/min (per Cockroft-Gault equation) (obtained within 14 days prior to enrollment)
* Patients on full-dose anticoagulants (e.g., warfarin or low molecular weight \[LMW\] heparin) must meet the following criteria:
* No active bleeding or pathological condition that carries a high risk of bleeding (e.g., tumor involving major vessels or known varices, which carries a significant risk of bleeding in investigator's opinion)
* Participants of child-bearing potential must agree to use adequate contraceptive methods (e.g., hormonal or barrier method of birth control; abstinence) prior to study entry. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately
* Participant must understand the investigational nature of this study and sign an independent ethics committee/institutional review board approved written informed consent form prior to receiving any study related procedure
Exclusion Criteria:
* Patients who have received temozolomide in the advanced disease setting either alone or as part of a combination therapy will be excluded if they progressed while on it
* Has received prior treatment with SurVaxM
* Received an investigational agent within 30 days prior to enrollment
* Participants who have received checkpoint inhibitors within 3 months prior to study enrollment or, those who have not recovered from adverse events due to agents administered more than 4 weeks earlier
* Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, bradycardia, tachycardia or psychiatric illness/social situations that would limit compliance with study requirements and, which in the treating physicians' opinion would jeopardize the ability of the patient to receive the treatment outlined in this protocol with reasonable safety
* Patients with a concurrent or prior malignancy are ineligible unless they are patients with curatively treated carcinoma-in-situ or basal cell carcinoma of the skin. Patients who have been free of disease (any prior malignancy) for at least 3 years are eligible for this study
* Known history of an autoimmune disorder
* Known human immunodeficiency virus (HIV) positivity or acquired immunodeficiency syndrome (AIDS) related illness or other serious medical illness
* Systemic corticosteroid therapy \> 2mg of dexamethasone or equivalent per day at study entry
* Pregnant or nursing female participants
* Unwilling or unable to follow protocol requirements
* Any condition which in the Investigator's opinion deems the participant an unsuitable candidate to receive study drug
* Patients with Hepatitis B or Hepatitis C may be included if there are adequately controlled viral titers and no drug-drug interactions, testing not required
Buffalo, New York, 14263, United States
[email protected] / 716-845-1300
Status: Recruiting