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HLA-A2 Restricted Peptide Vaccine and Imiquimod in Treating Children with Recurrent Ependymoma

Trial Status: Active

This pilot phase I trial studies the side effects and to see how well human leukocyte antigen (HLA)-A2 restricted peptide vaccine and imiquimod work in treating children with ependymoma that has come back after a period of improvement. Vaccines made from HLA-A2 restricted tumor antigen peptides and imiquimod may help the body build an effective immune response to kill tumor cells.

Inclusion Criteria

  • Tumor types – tumor type/location: * Stratum A: Patients with a recurrent ependymoma with the primary site in the posterior fossa; patients may have non-bulky asymptomatic, metastatic disease; patients may have undergone surgical resection or debulking prior to enrollment Stratum B: Patients with a recurrent ependymoma with the primary site outside the posterior fossa; patients may have non-bulky asymptomatic, metastatic disease; patients may have undergone surgical resection or debulking prior to enrollment
  • HLA-A2 positive based on flow cytometry
  • Patients must have previously received standard initial therapy, including attempted gross total resection, where safely feasible, and in appropriate circumstances (e.g., those older than one year at initial diagnosis, with non- metastatic tumors and at least microscopic residual disease) involved field fractionated radiation therapy (RT); patients may have received re-irradiation but not to the index lesion within 4 weeks
  • Patients must be clinically stable and off or on low-dose (no more than 0.1 mg/kg/day, max 4 mg/day dexamethasone) corticosteroid for at least one week prior to study registration
  • All patients must sign an Institutional Review Board (IRB)-approved informed consent document
  • Patients must have a performance status of >= 70; (Karnofsky if > 16 years and Lansky if =< 16 years of age
  • Males and females must agree to use effective birth control methods during the course of vaccination (from the first vaccine to two weeks after the last vaccine)
  • Patients must be free of systemic infection requiring intravenous (IV) antibiotics at the time of registration; patients must be off IV antibiotics for at least 7 days prior to registration
  • Absolute neutrophil count (ANC) > 1,000/ul
  • Platelets > 100,000/ul (transfusion independent)
  • Absolute lymphocyte count of >= 500/uL
  • Hemoglobin > 8 g/dl (may be transfused)
  • Bilirubin =< 1.5 x institutional normal for age
  • Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 3 x institutional normal
  • Serum creatinine based on age or creatinine clearance or radioisotope glomerular filtration rate (GFR) > 70 ml/min/ml/min/1.73 m^2 * Age 1 to < 2 male 0.6 female 0.6 * Age 2 to < 6 male 0.8 female 0.8 * Age 6 to < 10 male 1 female 1 * Age 10 to < 13 male 1.2 female 1.2 * Age 13 to < 16 male 1.5 female 1.4 * Age > 16 male 1.7 female 1.4
  • Patients must have recovered from the toxic effects of prior therapy and be at least 3 weeks from the last dose of standard cytotoxic chemotherapy or myelosuppressive biological therapy and at least 1 week from the last dose of non-myelosuppressive biologic therapy; any questions related to the definition of non-cytotoxic agents should be directed to the principal investigators; for those who have received radiation, 4 weeks must have elapsed before beginning vaccination
  • Patients must have no overt cardiac, gastrointestinal, pulmonary or psychiatric disease

Exclusion Criteria

  • Concurrent treatment or medications (must be off for at least 1 week) including: * Interferon (e.g. Intron-A) * Allergy desensitization injections * Growth factors (e.g. Procrit, Aranesp, Neulasta) * Interleukins (e.g. Proleukin) * Any investigational therapeutic medication
  • Patients must not have a history of any immune system disorder, or laboratory abnormality or any condition that could potentially alter immune function
  • Use of immunosuppressives within four weeks prior to study entry or anticipated use of immunosuppressive agents; patients must be on no more than 0.1 mg/kg/day, max 4 mg/day dexamethasone for at least one week before study registration; topical corticosteroids are acceptable
  • Patients who in the opinion of the investigator may not be able to comply with all the study requirements
  • Patients with a known immune deficiency
  • Pregnancy or breastfeeding; female patients who are post-menarchal must have a documented negative pregnancy test
  • Tetanus vaccine during therapy or within 1 week prior to enrollment
  • Patients who have received prior immunotherapy


Children's Hospital of Pittsburgh of UPMC
Status: ACTIVE
Contact: Scott Henry Maurer
Phone: 412-692-5056


I. To determine safety and tolerability of vaccination with tumor-associated antigen (TAA) peptides for children with recurrent ependymomas.

II. To define the rate and magnitude of immune response in post-vaccine peripheral blood mononuclear cells against vaccine peptides, in response to peptide-based vaccine therapy, using IFN-gamma-enzyme-linked immuno-spot (ELISPOT) and tetramer assays, and to determine whether immunological reactivity is associated with clinical response.


I. Clinical Response: radiological response will be determined using the standard World Health Organization (WHO) response criteria. Overall survival (OS) and progression-free survival will be evaluated.

II. Evaluation of tumor tissues for biological correlates: archived tissue from the time of diagnosis and any subsequent surgeries, as well for patients who develop progression or pseudoprogression on study, and undergo biopsy/tumor debulking, where clinically appropriate, will be analyzed for TAA expression status and infiltration of TAA-specific T-cells.


Patients receive HLA-A2 restricted peptide vaccine subcutaneously (SC) on weeks 0, 3, 6, 9, 12, 15, 18, and 21 for a total of 8 doses. Patients also receive imiquimod topically to the injection site for 8 hours after each vaccine. Beginning at week 24, patients with at least stable disease without regimen limiting toxicities may receive additional HLA-A2 restricted peptide vaccine SC and imiquimod topically every 6 weeks for up to 2 years in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 6 weeks.

Trial Phase Phase I

Trial Type Treatment

Lead Organization
Children's Hospital of Pittsburgh of UPMC

Principal Investigator
Scott Henry Maurer

  • Primary ID Ependymoma Vaccine
  • Secondary IDs NCI-2016-01096
  • ID NCT01795313