Immunotherapy for Recurrent Ependymomas in Children Using Tumor Antigen Peptides With Imiquimod

NCT01795313 | Terminated Phase 1 DMC FDA Drug

• 3 other identifiers: STUDY19100001R01CA174858PRO12050422
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to see if vaccination with HLA-A2 restricted peptides, combined with the immunoadjuvant imiquimod is safe and can induce immune responses in children with recurrent ependymomas. Eligible patients are stratified by primary tumor location.

Conditions

Ependymoma

Outcome Measures

Primary Outcomes (1)

• Number of Participants with unacceptable toxicity

  Grade 3 or 4 non-hematological toxicities.

  2 years

Secondary Outcomes (1)

• Tumor-associated antigen-specific T-cell

  2 years

Study Arms (1)

HLA-A2 restricted tumor antigen vaccine

EXPERIMENTAL

This is a single-arm study of a HLA-A2 restricted tumor antigen peptide vaccine, administered in conjunction with imiquimod

Biological: HLA-A2 restricted synthetic tumor antigen; Drug: Imiquimod; Other: enzyme-linked immunosorbent assay; Other: flow cytometry; Other: immunohistochemistry staining method; Other: laboratory biomarker analysis

Interventions

HLA-A2 restricted synthetic tumor antigen BIOLOGICAL

HLA-A2 restricted tumor antigen vaccine

Imiquimod DRUG

HLA-A2 restricted tumor antigen vaccine

enzyme-linked immunosorbent assay OTHER

HLA-A2 restricted tumor antigen vaccine

flow cytometry OTHER

HLA-A2 restricted tumor antigen vaccine

immunohistochemistry staining method OTHER

HLA-A2 restricted tumor antigen vaccine

laboratory biomarker analysis OTHER

HLA-A2 restricted tumor antigen vaccine

Eligibility Criteria

• Age: 12 Months - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients must have recurrent/progressive ependymoma that has progressed or recurred after initial adjuvant therapy.
• HLA-A2 positive based on flow cytometry performed at the University of Pittsburgh.
• 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.
• Patients must be ≥ 12 months and \<22 years of age at the time of study registration.
• Patients must have a performance status of ≥ 70; (Karnofsky if \> 16 years and Lansky if ≤ 16 years of age).
• Patients may have non-bulky, asymptomatic metastatic disease.
• 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 IV antibiotics at the time of registration and off IV antibiotics for at least 7 days prior to registration.
• Patients must have adequate organ function as measured by:
• Bone marrow: Absolute neutrophil count (ANC) \> 1,000/µl; Platelets \> 100,000/µl (transfusion independent); Absolute lymphocyte count (ALC) ≥ 500/µl; Hemoglobin \>8 g/dl (may be transfused).
• Hepatic: bilirubin ≤ 1.5x institutional normal for age; serum glutamate pyruvate transaminase (SGPT) \< 3x institutional normal
• Renal: Serum creatinine based on age or creatinine clearance or radioisotope glomerular filtration rate (GFR) \> 70 ml/min/1.73 m²
• 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, at least one week from the last dose of non-myelosuppressive biological therapy and at least 4 weeks from the completion of radiation therapy.
• Patients must have no overt cardiac, gastrointestinal, pulmonary, or psychiatric disease.

You may not qualify if:

• Patients living outside of North America are not eligible.
• Patients must be off concurrent treatment or medications 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®), and 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 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.

Sponsors & Collaborators

• James Felker: lead
• Solving Kids' Cancer: collaborator
• National Cancer Institute (NCI): collaborator

Study Sites (1)

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

MeSH Terms

Conditions

Ependymoma

Interventions

Imiquimod; Enzyme-Linked Immunosorbent Assay; Flow Cytometry; Immunohistochemistry

Condition Hierarchy (Ancestors)

Glioma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Aminoquinolines; Quinolines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Immunoenzyme Techniques; Immunoassay; Immunologic Techniques; Investigative Techniques; Immunosorbent Techniques; Molecular Probe Techniques; Cell Separation; Cytological Techniques; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Cytophotometry; Fluorometry; Luminescent Measurements; Photometry; Chemistry Techniques, Analytical; Histocytochemistry; Histological Techniques

Study Officials

• James Felker, MD

  University of Pittsburgh

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor

Model Details: This is a pilot study to assess tolerability of our vaccine regimen in children with ependymomas initially arising above or below the tentorium

Study Record Dates

• First Submitted: February 12, 2013
• First Posted: February 20, 2013
• Study Start: August 1, 2012
• Primary Completion: December 3, 2025
• Study Completion: December 3, 2025
• Last Updated: February 5, 2026

Record last verified: 2026-02

Locations

US (1)