EpendymomaPhase 1NCT01795313
Immunotherapy for Recurrent Ependymomas in Children Using Tumor Antigen Peptides With Imiquimod
A Phase 1 study testing HLA-A2 restricted synthetic tumor antigen, Imiquimod and enzyme-linked immunosorbent assay in people with Ependymoma, run by James Felker at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 months to 21 years
- Conditions the study is for
- Ependymoma
- What is being tested?
- HLA-A2 restricted synthetic tumor antigen, Imiquimod and enzyme-linked immunosorbent assay
- Type of treatment
- Vaccines
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2012to 3 Dec 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 months to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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).
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- 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.
- Patients must be willing to travel to Pittsburgh to receive the vaccine. Visits: Every 3 weeks x 9, then every 6 weeks x 12 depending on response/side effects
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
About this study
Official title: Immunotherapy for Recurrent Ependymomas in Children Using Human Leukocyte Antigen (HLA)-A2 Restricted Tumor Antigen Peptides in Combination With Imiquimod
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
- Being tested
- HLA-A2 restricted synthetic tumor antigen, Imiquimod, enzyme-linked immunosorbent assay, flow cytometry, immunohistochemistry staining method
- Medical subject headings
- Ependymoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HLA-A2 restricted synthetic tumor antigenBiological
ImiquimodDrug
enzyme-linked immunosorbent assayOther
flow cytometryOther
laboratory biomarker analysisOther
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with unacceptable toxicity
Grade 3 or 4 non-hematological toxicities.
Measured: 2 years
Secondary outcomes (1)
Tumor-associated antigen-specific T-cell
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2013 (estimated)
- Study startAug 2012
- Primary completion3 Dec 2025
- Study completion3 Dec 2025
Why it stopped: Closed supratentorial arm prematurely secondary to lack of enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for EpendymomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is a pilot study to assess tolerability of our vaccine regimen in children with ependymomas initially arising above or below the tentorium
- Masking
- None (open label)
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- James Felker
- Sponsor type
- University, hospital or other
- Collaborators
- Solving Kids' Cancer, National Cancer Institute (NCI)
- Responsible party
- Sponsor-investigator, James Felker, University of Pittsburgh
- Investigators
- James Felker, MD, Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01795313
- Sponsor's protocol ID
- STUDY19100001
- Other IDs
- R01CA174858, PRO12050422
- Status last verified
- Feb 2026
- First submitted
- 12 Feb 2013
- Last updated
- 5 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Immunotherapy for Recurrent Ependymomas in Children Using Human Leukocyte Antigen (HLA)-A2 Restricted Tumor Antigen Peptides in Combination With Imiquimod
- ClinicalTrials.gov ID
- NCT01795313
- Acronym / sponsor ID
- STUDY19100001
- Conditions
- Ependymoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Closed supratentorial arm prematurely secondary to lack of enrollment
- Enrolment
- 23 participants (actual)
- Lead sponsor
- James FelkerUniversity, hospital or other
- Collaborators
- Solving Kids' Cancer, National Cancer Institute (NCI)
- Study start
- Aug 2012
- Primary completion
- 3 Dec 2025 (actual)
- Study completion
- 3 Dec 2025 (actual)
- First posted
- 20 Feb 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a pilot study to assess tolerability of our vaccine regimen in children with ependymomas initially arising above or below the tentorium
| Arm | Type | Receives |
|---|---|---|
| HLA-A2 restricted tumor antigen vaccine | Experimental | Biological: HLA-A2 restricted synthetic tumor antigen, Drug: Imiquimod, Other: enzyme-linked immunosorbent assay, Other: flow cytometry, Other: immunohistochemistry staining method, Other: laboratory biomarker analysisThis is a single-arm study of a HLA-A2 restricted tumor antigen peptide vaccine, administered in conjunction with imiquimod |
Who may qualify
- Ages
- 12 months to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (16 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 |
Primary outcome
Number of Participants with unacceptable toxicity
Time frame: 2 years
Grade 3 or 4 non-hematological toxicities.
1 secondary outcome
Tumor-associated antigen-specific T-cell
Time frame: 2 years
Results and safety
The study was stopped early (reason given: Closed supratentorial arm prematurely secondary to lack of enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James Felker, MD principal investigator
University of Pittsburgh
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing HLA-A2 restricted synthetic tumor antigen, Imiquimod and enzyme-linked immunosorbent assay in people with Ependymoma, run by James Felker at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 5 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Closed supratentorial arm prematurely secondary to lack of enrollment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with unacceptable toxicity (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2026.