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Oncology Clinical Trials and Research Update from UPMC Children’s

March 21, 2019

The Division of Pediatric Hematology/Oncology at UPMC Children’s Hospital of Pittsburgh has well over 100 open clinical trials in progress covering numerous aspects of the oncologic spectrum.

Many of these trials are novel, investigator-initiated studies adding to the breadth of the numerous multicenter trials in progress at UPMC Children’s as a part of the Children’s Oncology Group.

In this clinical trials update, we focus on several new studies led by Alberto Broniscer, MD, director of neuro-oncology. Also profiled are investigations being conducted by Kelly Bailey, MD,PhD, a sarcoma specialist in the Division who is investigating the Ewing sarcoma tumor microenvironment.

Neuro-Oncology Trials – Chipping Away At Intractable Illnesses

Dr. Broniscer, director of the nationally recognized neuro-oncology program at UPMC Children’s Hospital of Pittsburgh, leads a robust multidisciplinary clinical apparatus and research program engaged in a diverse array of clinical, translational, and basic science investigations. Dr. Broniscer has more than two decades of clinical and research experience studying brain and spinal cord tumors in children with an emphasis on diffuse intrinsic pontine glioma (DIPG).

A selection of in-progress trials includes the following institutional experimental vaccine studies for low-grade gliomas and ependymomas. For these studies, Dr. Broniscer is collaborating with Ian Pollack MD, chief of pediatric neurosurgery at UPMC Children’s Hospital of Pittsburgh.

  • A Pilot Study to Evaluate the Effects of Vaccinations with HLA-A2 Restricted Tumor Antigen-Peptides in Combination with Imiquimod for Children with Recurrent Ependymomas. Identifier: NCT01795313. The purpose of this study is to determine if vaccination with HLA-A2 restricted peptides, combined with the immunoadjuvant imiquimod is safe and can induce immune responses in children who have recurrent ependymomas.
  • A Pilot Study to Evaluate the Effects of Vaccinations with HLA-A2-Restricted Glioma Antigen-Peptides in Combination with Poly-ICLC for Children with Newly Diagnosed Malignant or Intrinsic Brain Stem Gliomas (BSG), High-Grade Gliomas (HGG), or Low-Grade Gliomas (LGG). Identifier: NCT01130077. The overall objective of this pilot study is to collect immunological and safety data following administration of vaccinations with HLA-A2. These data will be used to determine whether a larger study of clinical efficacy is warranted.
  •  A Phase II Study of Vaccinations with HLA-A2 Restricted Glioma Peptides in Combination with Poly-ICLC for Children with Recurrent Unresectable Low-Grade Gliomas. Identifier: NCT02358187. This study will assess the immunogenicity, safety, and preliminary clinical efficacy of the GAA/TT-peptide vaccine and poly-ICLC in HLA-A2+ children with unresectable low-grade gliomas who have received at least two chemotherapy/biologic regimens. Radiation therapy can be one of the biologic regimens, but patients may not have received radiation to the index lesion within one year of trial enrollment.