Experimental vaccine aims to rally immune system against recurrent brain cancer
NCT ID NCT03299309
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a vaccine called PEP-CMV in 30 children and young adults (ages 3–35) whose medulloblastoma or malignant glioma has come back. The vaccine is designed to train the immune system to attack tumor cells that carry a common virus protein (CMV). Participants also receive chemotherapy and a tetanus booster to boost the vaccine's effect. The main goal is to check safety, not yet to prove the vaccine works against the cancer.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- PEP-CMV vaccine (a peptide vaccine targeting CMV protein pp65, given with temozolomide and a tetanus-diphtheria booster)
- What this could lead to
- If this vaccine proves safe and triggers an immune response, it could point toward a new way to fight recurrent brain tumors in children and young adults.
- What could go wrong
- This is a very early (Phase 1) safety study with only 30 participants. The vaccine may not shrink tumors or improve survival, and there is a risk of side effects from the vaccine or chemotherapy.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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30 people
The number who actually took part.
- Started
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Jun 2018
- Expected to finish
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May 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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3 to 35 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Patients who are 3 - 35 years old 2. Histopathologically proven previous diagnosis of medulloblastoma or Grade III or IV glioma. 3. Radiology evidence of recurrent medulloblastoma (reMB) or recurrent Grade III and IV glioma. Patients will be considered for a biopsy or resection of the recurrent/progressive tumor at the discretion of the treating neurosurgeon and neuro-oncologist. 4. Brain MRI within one month prior to enrollment. 5. Received prior therapy for their initial diagnosis prior to recurrence/progression or who are unable to receive radiation therapy due to genetic disorders that put them at significant risk for radiation-induced secondary malignancies (i.e. Gorlin's syndrome or NF1 mutation). 6. Patients with neurological deficits should have deficits that are stable for a minimum of 2 weeks prior to registration. 7. Karnofsky Performance Status (KPS) of ≥ 60% (KPS for \> 10 years of age) or Lansky performance Score (LPS) of ≥ 60 (LPS for ≤ 10 years of age) assessed within 2 weeks prior to registration. Patients who are unable to walk because of paralysis but who are up in a wheel chair will be considered ambulatory for the purposes of the performance score. 8. Bone Marrow: * ANC (Absolute neutrophil count) ≥ 1000/µl (unsupported)\*. * Platelets ≥ 100,000/µl (unsupported)\*. * Hemoglobin \> 8 g/dL (may be supported). 9. Renal: • Serum creatinine ≤ upper limit of institutional normal. 10. Hepatic: * Bilirubin ≤ 1.5 times upper limit of normal for age. * SGPT (ALT) ≤ 3 times institutional upper limit of normal for age. * SGOT (AST) ≤ 3 times institutional upper limit of normal for age. 11. Patients of childbearing or child-fathering potential must be willing to use a medically acceptable form of birth control, which includes abstinence, while being treated on this study. 12. Signed informed consent according to institutional guidelines must be obtained prior to registration. 13. Any prior chemoradiotherapy is allowed. Exclusion Criteria: 1. Pregnant or need to breast feed during the study period (Negative serum pregnancy test required). 2. Active infection requiring treatment or an unexplained febrile (\> 101.5 degrees F) illness. 3. Known immunosuppressive disease or human immunodeficiency virus infection. 4. Patients with active renal, cardiac (congestive cardiac failure, myocardial infarction, myocarditis), or pulmonary disease. 5. Patients receiving concomitant immunosuppressive agents for medical condition. 6. Patients who need definitive radiotherapy for treatment of recurrent MB or recurrent Grade III or IV glioma. 7. Patients receiving any other investigational drug therapy. 8. Patients on corticosteroids \> 0.1 mg/Kg/day (i.e. \> the maximum dose of 4 mg/day). 9. Patients with any clinically significant unrelated systemic illness (serious infections or significant cardiac, pulmonary, hepatic or other organ dysfunction). 10. Patients with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Duke University Medical Center
Durham, North Carolina, 27710, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Targeted radiation zaps childhood brain tumors in early trial
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- Parental distress may undermine treatment in children with recurrent brain tumors