Pediatric Recurrent Central Nervous System TumorsPhase 1NCT03988283
Neoepitope-based Personalized DNA Vaccine Approach in Pediatric Patients With Recurrent Central Nervous System Tumors
A Phase 1 study testing Personalized neoantigen DNA vaccine, Papivax Biotech TDS-IM v2.0 and Peripheral blood draw in people with Pediatric Recurrent Central Nervous System Tumors, run by Washington University School of Medicine at 1 site in United States.
Trial summary
The purpose of this research study is to learn about the safety and feasibility of giving a personalized DNA vaccine to people with central nervous system tumors that have returned or have been resistant to treatment. Read more about this study
- Who is it for?
- Ages Up to 25 years
- Conditions the study is for
- Pediatric Recurrent Central Nervous System Tumors
- What is being tested?
- Personalized neoantigen DNA vaccine, Papivax Biotech TDS-IM v2.0 and Peripheral blood draw
- Type of treatment
- Vaccines, Injection or infusion, Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Oct 2024to 28 Feb 2031
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 Up to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Any patient between the ages of 12 and 25 years of age (inclusive) who was diagnosed with any of the following:
- High-grade glioma (HGG); newly diagnosed patients
- Diffuse midline glioma (DMG); newly diagnosed patients
- A pediatric CNS (brain or spine) tumor of any histologic subtype, who has now developed recurrent or refractory disease.
- All patients enrolled in this trial will receive treatment for pediatric CNS tumors, including systemic agents, investigational agents, or radiation therapy, prior to receiving the neoantigen DNA vaccine. Co-enrollment to another interventional clinical trial is permitted during the vaccine manufacture period.
- Availability of tissue for sequencing to determine presence of targetable neoantigen. This may be fresh tissue collected as part of routine care, another research project, or banked fresh frozen samples from tissue obtained at time of progression from a previous biopsy, subtotal resection, total gross resection, or re-resection, with the following disease specific restrictions:
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- For HGG and DMG patients, tissue may be collected either at diagnosis or after tumor recurrence or progression.
- For pediatric CNS tumor patients, tissue must be collected after tumor recurrence or progression.
- Life expectancy > 24 weeks.
- Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable) or patient has a guardian who has the ability to understand and willingness to sign an IRB approved written informed consent document.
- Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, or should a man suspect he has fathered a child, s/he must inform her treating physician immediately.
- Step 1 Exclusion Criteria:
- A history of other malignancy ≤ 3 years previous with the exception of non-melanoma skin cancer, any in situ cancer that has been successfully resected and cured, treated superficial bladder cancer, or any early-stage solid tumor that was successfully resected without need for adjuvant radiation or chemotherapy.
- Known allergy, or history of serious adverse reaction to, vaccines such as anaphylaxis, hives, or respiratory difficulty.
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- History of immunodeficiency disorder or autoimmune condition requiring active immunosuppressive therapy. This includes inflammatory bowel disease, ulcerative colitis, Crohn's disease, systemic vasculitis, scleroderma, psoriasis, multiple sclerosis, hemolytic anemia, immune-mediated thrombocytopenia, rheumatoid arthritis, systemic lupus erythematosus, Sjögren's syndrome, sarcoidosis, or other rheumatologic disease or any other medical condition or use of medication which might make it difficult for the patient to complete the full course of treatments or to generate an immune response to vaccines.
- Patients with HIV are eligible unless their CD4+ T-cell counts are < 350 cells/mcL or they have a history of AIDS-defining opportunistic infection within the 12 months prior to registration. Concurrent treatment with effective ART according to DHHS treatment guidelines is recommended.
- Presence of clinically significant increased intracranial pressure (e.g. impending herniation) or hemorrhage, uncontrolled seizures, or requirement for immediate palliative treatment.
- Individuals in whom a measurement of the circumference of the thigh at the midpoint between the hip and knee is < 35 cm.
- Individuals in whom a skinfold measurement of the cutaneous and subcutaneous tissue for eligible injection sites (left and right vastus laterals muscles) exceeds 50 mm.
- Individuals in whom the ability to observe possible local reactions at the eligible injection sites is, in the opinion of the investigator, unacceptably obscured due to a physical condition (e.g. hypertrophic skin patches, keloids, or other conditions which could interfere with the administration procedure or subsequent assessment of local reactogenicity) or permanent body art.
- Has a metal implant or implantable device within the area of the electroporation injection or has any non-removable electronic stimulation device, such as cardiac demand pacemaker, automatic implantable cardiac defibrillator, nerve stimulator, or deep brain stimulator.
- Acute or chronic, clinically significant hematologic, pulmonary, cardiovascular, or hepatic or renal functional abnormality as determined by the investigator based on medical history, physical examination, EKG, and/or laboratory screening test.
- Participants with seizure disorder may be enrolled if seizures are well-controlled. Patients with neurologic deficits should have deficits that are stable for a minimum of 1 week prior to enrollment
- Step 2 Eligibility Criteria for Treatment Administration
- Personalized neoantigen DNA vaccine manufactured for administration.
- HGG or DMG patients ONLY: must have developed recurrent or refractory disease after receiving at least one line of prior treatment.
- Karnofsky/Lansky performance status ≥ 50%
- Life expectancy > 12 weeks.
- Prior therapy washout requirements (with exception of bevacizumab):
- Myelosuppressive chemotherapy: Participants must have received their last dose of known myelosuppressive anticancer chemotherapy at least 3 weeks prior to first dose of vaccine or at least 6 weeks prior if nitrosurea.
- Biologic agent: Participant must have received their last dose of the biologic agent at least 7 days prior to first dose of vaccine.
- For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended to beyond the time during which adverse events are known to occur. The duration of this interval should be discussed with the study PI.
- For biologic agents that have a prolonged half-life, the appropriate interval since last treatment should be discussed with the PI prior to first dose of vaccine.
- Monoclonal antibody treatment: At least three half-lives must have elapsed prior to first dose of vaccine. Such participants should be discussed with the PI prior.
- Bone Marrow Transplant: Participant must be:
- ≥ 6 months since allogeneic bone marrow transplant prior to first dose of vaccine.
- ≥ 3 months since autologous bone marrow/stem cell prior to first dose of vaccine.
- Radiotherapy (in instances of lesions amenable to radiotherapy, such as bone metastases or metastases causing nerve impingement): The last fraction must have been at least 4 weeks prior to first dose of vaccine.
- Investigational agents: The last dose must have been at least 4 weeks prior to first dose of vaccine.
- Adequate bone marrow and organ function as defined below:
- Absolute neutrophil count ≥ 1.5 K/cumm
- Platelets ≥ 100 K/cumm
- Hemoglobin level ≥ 8 g/dL
- Total bilirubin ≤ 1.5 x institutional upper limit of normal (IULN)
- AST(SGOT)/ALT(SGPT) ≤ 3.0 x IULN
- Creatinine ≤ IULN OR creatinine clearance ≥ 60 mL/min/1.73 m^2 for patients with creatinine levels above institutional normal
- Any adverse event patients may have experienced during prior therapy must have resolved to ≤ grade 1 CTCAE v5.
- Systemic corticosteroid therapy is permitted provided dosing is minimal based on age, defined as 0.1mg/kg/day with a max of 4mg daily (dexamethasone or equivalent) on the day of vaccine administration. Participants using topical, ocular, intra-articular, or intranasal/inhaled steroids may participate. Brief courses of corticosteroids for prophylaxis (eg, contrast dye allergy) or study treatment-related standard premedication are permitted.
- Bevacizumab will be allowed if given for symptomatic control of vasogenic edema and to avoid high dose of corticosteroids.
- Step 2 Exclusion Criteria:
- No candidate neoantigen identified during the vaccine evaluation process.
- Ongoing or active infection requiring systemic therapy (e.g. active HBV or HCV infection that requires treatment) or causing fever (temperature > 38.1˚C) or subjects with unexplained fever (temperature > 38.1˚C) within 7 days prior to the first vaccine dose.
- Administration of live vaccine within 30 days prior to first dose of vaccine. Participants may receive the COVID-19 vaccine within this time period as it is not a live vaccine.
- Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 7 days of first dose of vaccine.
- Syncopal episode within 12 months of first dose of vaccine.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Washington University School of Medicine
St Louis, Missouri, 63110
Michael A Huang, M.D., 314-454-4146, huangm@wustl.edu
Michael A Huang, M.D.
About this study
Official title: A Pilot Study to Assess the Safety, Feasibility, and Preliminary Efficacy of a Neoepitope-based Personalized DNA Vaccine Approach in Pediatric Patients With Recurrent Central Nervous System Tumors
The purpose of this research study is to learn about the safety and feasibility of giving a personalized DNA vaccine to people with central nervous system tumors that have returned or have been resistant to treatment.
- Being tested
- Personalized neoantigen DNA vaccine, Papivax Biotech TDS-IM v2.0, Peripheral blood draw
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Personalized neoantigen DNA vaccineBiological
At each vaccination time point, patients will receive one injection of the neoantigen DNA vaccine, one injection into the vastus lateralis.
Papivax Biotech TDS-IM v2.0Device
All study injections will be given intramuscularly using an integrated electroporation device (TDS-IM v2.0 device - Papivax Biotech).
Peripheral blood drawProcedure or surgery
-After trial enrollment and up to 7 days after the first vaccine dose (baseline); no more than 2 weeks after the 3rd vaccine dose; no more than 2 weeks after the 6th vaccine dose; two weeks after the last dose; time of progression or discontinuation (optional)
| Study group | Role | What they receive |
|---|---|---|
| Personalized neoantigen DNA vaccine | Experimental | Patients will receive the vaccine monthly (+/- 3 days) for 6 doses as a priming phase followed by booster injections quarterly Q3mo thereafter. If sufficient quantities of vaccine are available, vaccination may continue until development of intolerance or disease progression in the case of fatal high grade neoplasms or for up to one year. Additionally, patients with non-fatal tumors who complete one year of vaccinations and have stable disease at the completion of treatment will be given the option of resuming vaccinations if they develop subsequent progression. Receives: Biological: Personalized neoantigen DNA vaccine; Device: Papivax Biotech TDS-IM v2.0; Procedure: Peripheral blood draw |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of adjuvant personalized neoantigen DNA vaccine as measured by the number of grade 3 and 4 adverse events
Measured: Through 30 days after completion of treatment (estimated to be 13 months)
Feasibility of adjuvant personalized neoantigen DNA vaccine as measured the number of participants that had a neoantigen-specific DNA vaccine generated
Measured: From time of resection to time of initiation (approximately 12-14 weeks)
Secondary outcomes (2)
Median Progression Free Survival (PFS)
- PFS is defined as the duration of time from date of first dose of vaccine to date of progression or evidence of last progression free time.
- Progressive Disease (PD): At least a 25% increase in the sum of products of perpendicular diameters of at least 1 target lesion, taking as reference the smallest sum of products of perpendicular diameters on study (this includes the baseline sum if that is the smallest on study). The absolute increase in any dimension must be at least 5mm when calculating the products of perpendicular diameters. Appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions on stable or increasing doses of corticosteroids compared with baseline scan or best response after initiation of therapy* not caused by comorbid events.
Measured: 2 years
Median Overall Survival
-OS is defined as date of first dose of vaccine to date of death or date of last known alive.
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 posted17 Jun 2019
- Study start2 Oct 2024
- Primary completion31 Mar 2029 (estimated)
- Study completion28 Feb 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric Recurrent Central Nervous System TumorsStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- 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
- Masking
- None (open label)
- Enrolment
- 7 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Discovery Institute
- Responsible party
- Sponsor
- Investigators
- Michael A Huang, M.D., Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03988283
- Sponsor's protocol ID
- 202211187
- Status last verified
- May 2026
- First submitted
- 12 Jun 2019
- Last updated
- 7 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study to Assess the Safety, Feasibility, and Preliminary Efficacy of a Neoepitope-based Personalized DNA Vaccine Approach in Pediatric Patients With Recurrent Central Nervous System Tumors
- ClinicalTrials.gov ID
- NCT03988283
- Acronym / sponsor ID
- 202211187
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 7 participants (estimated)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Collaborators
- Children's Discovery Institute
- Study start
- 2 Oct 2024 (actual)
- Primary completion
- 31 Mar 2029 (estimated)
- Study completion
- 28 Feb 2031 (estimated)
- First posted
- 17 Jun 2019 (actual)
- 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 research study is to learn about the safety and feasibility of giving a personalized DNA vaccine to people with central nervous system tumors that have returned or have been resistant to treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Personalized neoantigen DNA vaccine | Experimental | Biological: Personalized neoantigen DNA vaccine, Device: Papivax Biotech TDS-IM v2.0, Procedure: Peripheral blood drawPatients will receive the vaccine monthly (+/- 3 days) for 6 doses as a priming phase followed by booster injections quarterly Q3mo thereafter. If sufficient quantities of vaccine are available, vaccination may continue until development of intolerance or disease progression in the case of fatal high grade neoplasms or for up to one year. Additionally, patients with non-fatal tumors who complete one year of vaccinations and have stable disease at the completion of treatment will be given the option of resuming vaccinations if they develop subsequent progression. |
Who may qualify
- Ages
- Up to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (56 listed)
- Any patient between the ages of 12 and 25 years of age (inclusive) who was diagnosed with any of the following:
- All patients enrolled in this trial will receive treatment for pediatric CNS tumors, including systemic agents, investigational agents, or radiation therapy, prior to receiving the neoantigen DNA vaccine. Co-enrollment to another interventional clinical trial is permitted during the vaccine manufacture period.
- Availability of tissue for sequencing to determine presence of targetable neoantigen. This may be fresh tissue collected as part of routine care, another research project, or banked fresh frozen samples from tissue obtained at time of progression from a previous biopsy, subtotal resection, total gross resection, or re-resection, with the following disease specific restrictions:
- Life expectancy > 24 weeks.
- Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable) or patient has a guardian who has the ability to understand and willingness to sign an IRB approved written informed consent document.
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| Personalized neoantigen DNA vaccineAt each vaccination time point, patients will receive one injection of the neoantigen DNA vaccine, one injection into the vastus lateralis. | Biological | Personalized neoantigen DNA vaccine |
| Papivax Biotech TDS-IM v2.0All study injections will be given intramuscularly using an integrated electroporation device (TDS-IM v2.0 device - Papivax Biotech). | Device | Personalized neoantigen DNA vaccine |
| Peripheral blood draw-After trial enrollment and up to 7 days after the first vaccine dose (baseline); no more than 2 weeks after the 3rd vaccine dose; no more than 2 weeks after the 6th vaccine dose; two weeks after the last dose; time of progression or discontinuation (optional) | Procedure or surgery | Personalized neoantigen DNA vaccine |
Primary outcome
Safety and tolerability of adjuvant personalized neoantigen DNA vaccine as measured by the number of grade 3 and 4 adverse events
Time frame: Through 30 days after completion of treatment (estimated to be 13 months)
Feasibility of adjuvant personalized neoantigen DNA vaccine as measured the number of participants that had a neoantigen-specific DNA vaccine generated
Time frame: From time of resection to time of initiation (approximately 12-14 weeks)
2 secondary outcomes
Median Progression Free Survival (PFS)
Time frame: 2 years
Median Overall Survival
Time frame: 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2026.
- Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of MedicineLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Michael A Huang, M.D. principal investigator
Washington University School of Medicine
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 Personalized neoantigen DNA vaccine, Papivax Biotech TDS-IM v2.0 and Peripheral blood draw in people with Pediatric Recurrent Central Nervous System Tumors, run by Washington University School of Medicine 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 56 inclusion and 0 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: looking for participants now, as of the sponsor's last update on 7 May 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Michael A Huang, M.D. (314-454-4146, huangm@wustl.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of adjuvant personalized neoantigen DNA vaccine as measured by the number of grade 3 and 4 adverse events (time frame: Through 30 days after completion of treatment (estimated to be 13 months)). The study lists 2 primary outcomes in all. 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 7 May 2026.