Personalized Synthetic Long Peptide Vaccine and Poly-ICLC in Treating Participants with Recurrent or Refractory Childhood Brain Tumors

Status: Approved


This phase I pilot trial studies the side effects and how well a personalized synthetic long peptide vaccine and poly-ICLC work in treating participants with childhood brain tumors that have come back or that aren't responding to treatment. The personalized synthetic long peptide vaccine is designed to target mutations specific to each person’s tumor that are discovered during genetic testing of the tumor. The vaccine may generate an immune response (the way the body fights viruses and other infections) to brain tumor cells. Poly-ICLC may have anti-tumor and antiviral effects. Giving a personalized synthetic long peptide vaccine with poly-ICLC may work better in treating participants with recurrent or refractory childhood brain tumors.

Eligibility Criteria

Inclusion Criteria

  • Any patient, regardless of current age, who was diagnosed between the ages of 0-21 years with a pediatric brain tumor of any histologic subtype, who has now developed recurrent or refractory disease.
  • 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 archived tissue from a previous craniotomy with biopsy, subtotal resection, total gross resection, or re-resection.
  • Karnofsky/Lansky performance status >= 60%.
  • Absolute neutrophil count >= 1,500/mcL.
  • Platelets >= 100,000/mcL.
  • Total bilirubin =< 1.5 x institutional upper limit of normal (IULN).
  • Aspartate aminotransferase (AST) serum glutamic oxaloacetic transaminase ([SGOT])/alanine aminotransferase (ALT) serum glutamate pyruvate transaminase ([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.
  • Systemic corticosteroid therapy is permitted provided dosing is minimal based on age 0.1mg/kg/day with a maximum (max) of 4mg daily (dexamethasone or equivalent) on the day of vaccine administration.
  • Bevacizumab will be allowed if given for symptomatic control of vasogenic edema and to avoid high dose of corticosteroids at the discretion of the treating physician.
  • 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, she must inform her treating physician immediately.
  • Ability to understand and willingness to sign an Institutional Review Board (IRB) approved written informed consent document (or that of legally authorized representative, if applicable).

Exclusion Criteria

  • As this is a safety and feasibility study, prior immunotherapy will be permitted. However, any prior immunotherapy must be discontinued at least 2 weeks before peptide vaccine administration. Non-immunologic therapy may be continued.
  • No candidate neoantigen identified during screening.
  • 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.
  • Currently receiving any other investigational agents.
  • Known allergy, or history of serious adverse reaction to, vaccines such as anaphylaxis, hives, or respiratory difficulty.
  • A history of allergic reactions attributed to compounds of similar chemical or biologic composition to poly-ICLC or other agents used in the study.
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  • History of pre-existing immunodeficiency disorder, autoimmune condition requiring immunosuppressive therapy, or chronic infection (i.e. hepatitis B, hepatitis C, human immunodeficiency virus [HIV]). 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, Sjogren’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.
  • Presence of clinically significant increased intracranial pressure (e.g. impending herniation) or hemorrhage, uncontrolled seizures, or requirement for immediate palliative treatment.
  • Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 7 days of first dose of vaccine.

Locations & Contacts


Saint Louis
Siteman Cancer Center at Washington University
Status: Approved
Contact: Karen M. Gauvain
Phone: 314-454-2002

Trial Objectives and Outline


I. To determine the safety and tolerability of adjuvant personalized neoantigen peptide vaccine administration with poly-ICLC in patients with recurrent or refractory pediatric brain tumors.

II. To determine the feasibility of adjuvant personalized neoantigen peptide vaccine administration with poly-ICLC in patients with recurrent or refractory pediatric brain tumors.


I. To characterize tumor-infiltrating lymphocytes (TIL) derived from tissue specimens from patients with recurrent or refractory pediatric brain tumors who have received a personalized neoantigen peptide vaccine with poly-ICLC before and after vaccination.

II. To determine the frequency of expressed neoantigens using patient-specific HLA class I prediction algorithms in patients with recurrent or refractory pediatric brain tumors.


I. To evaluate preliminary efficacy by determining progression-free survival rate and overall survival rate.

II. To identify pre- and post-vaccination biomarkers associated with response to personalized neoantigen vaccine.

III. To perform gene expression program analysis of TIL to determine activation states compared to controls and following vaccination.

IV. To evaluate antigen-specific cellular and humoral immune responses in the peripheral blood against non-immunized tumor-associated antigens (i.e., epitope spreading).

V. To characterize cell-free peripheral blood deoxyribonucleic acid (DNA) for use as a targeted circulating biomarker of antigenic maintenance or loss before and during treatment.


Participants receive personalized synthetic long peptide vaccine with poly-ICLC subcutaneously (SC) on days 1, 4, 8, 15, and 22 of course 1, and on day 1 of courses 2 and 3 and all subsequent courses. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, participants are followed up at 30 days and then annually for up to 24 months.

Trial Phase & Type

Trial Phase

Phase I

Trial Type


Lead Organization

Lead Organization
Siteman Cancer Center at Washington University

Principal Investigator
Karen M. Gauvain

Trial IDs

Primary ID 201807123
Secondary IDs NCI-2018-01578 ID NCT03068832