Table 1.
Actively recruiting immunotherapy trials for pHGGs as of March 2021
| NCT No. | Title of study | Therapeutic strategy | Phase | Objectives |
|---|---|---|---|---|
| NCT04196413 | GD2CART for the treatment of H3K27M mutated diffuse intrinsic pontine glioma or spinal diffuse midline glioma | GD2 CAR T cells | I | Determine feasibility of manufacturing autologous GD2 CAR T cells. Assess safety, determine maximum tolerated dose, and establish phase II dosing. Assess expansion, persistence and phenotype of GD2 CAR T cells. |
| NCT04185038 | Genetically engineered cells (B7-H3-specific CAR T cells) in treating pediatric patients with diffuse intrinsic pontine glioma, diffuse midline glioma, or recurrent or refractory central nervous system tumors | B7-H3 CAR T cells | I | Assess feasibility and safety of B7-H3 CAR T cells. Establish dose tolerability and define maximum tolerated dose. Assess B7-H3 CAR T-cell distribution within CSF. Assess disease response to B7-H3 CAR T cells. |
| NCT02442297 | HER2-Specific T cells in treating participants with recurrent or refractory HER2-positive glioblastoma | HER2 CAR T cells | I | Evaluate safety, persistence, expansion, and function of HER2 CAR T cells. |
| NCT03500991 | HER2-specific CAR T cell locoregional immunotherapy for HER2-positive recurrent/refractory pediatric CNS tumors | HER2 CAR T cells | I | Establish safety and feasibility of HER2-specific CAR T-cell infusions. Assess HER2 CAR T-cell distribution and function in CSF and peripheral blood. |
| NCT03638167 | EGFR806-specific CAR T cell locoregional immunotherapy for EGFR-positive recurrent or refractory pediatric CNS tumors | EGFR806-specific CAR T cells | I | Establish safety and feasibility of EGFR806-specific CAR T-cell infusions. Assess EGFR806-specific CAR T-cell distribution and function in CSF and peripheral blood. Assess expression of EGFR in relapsed treated tumours. |
| NCT02208362 | Genetically modified T-cells in treating patients with recurrent or refractory malignant glioma | IL13RA2 CAR T cells | I | Feasibility and safety of ex vivo expanded CAR T cells. Determine maximum tolerated dose schedule and phase II dosing. Assess post-immunotherapy responses. |
| NCT04099797 | Genetically engineered cells (C7R-GD2.CAR T cells) for the treatment of patients with GD2-expressing high grade glioma or diffuse intrinsic pontine glioma, The GAIL-B trial | GD2 CAR T cells with constitutively active IL7 receptor (C7R-GD2 CAR T cells) | I | Determine safety of escalating doses of CAR T cells. Estimate antitumour response. Evaluate fate and immunological effects of CAR T cells. |
| NCT03652545 | Multi-antigen T cell infusion against neuro-oncologic disease (REMIND) | TAA-T | I | Determine safety and feasibility of rapidly generated TAA-T. Determine optimal dosing schedule of TAA-T. |
| NCT03911388 | HSV G207 in children with recurrent or refractory cerebellar brain tumors | G207 HSV with or without radiotherapy | I | Determine safety and tolerability by frequency of grade 3 above adverse events. Assess immunological response via HSV-1 antibody titres. Assess progression free survival and overall survival. |
| NCT02960230 | H3.3K27M-specific Peptide Vaccine and Poly ICLC with or without Nivolumab in treating patients with newly diagnosed HLA-A2 positive, H3.3K27M positive diffuse intrinsic pontine glioma or other newly diagnosed gliomas | H3.3K27M-specific peptide vaccine and poly-ICLC with or without nivolumab (anti-PD1) | I | Assess safety of H3.3K27M-specific peptide vaccine with or without nivolumab in H3.3K27M+ DIPG or midline gliomas. Determine overall survival at 12 months. Assess H3.3K27M epitope-specific cytotoxic T lymphocyte response. |
| NCT03396575 | Brain stem gliomas treated with adoptive cellular therapy during focal radiotherapy recovery alone or with dose-intensified temozolomide (BRAVO) | TTRNA-DC with or without chemotherapy | I | Assess safety and feasibility of TTRNA-DC vaccines with or without chemotherapy. Determine the maximally achievable dose or maximum tolerated dose. Assess post-immunotherapy antitumour immune response in lymphocytes. |
| NCT01130077 | A pilot study of glioma associated antigen vaccines in conjunction with poly-ICLC in pediatric gliomas | HLA restricted glioma antigen peptides with poly-ICLC | I | Assess safety and tolerability. Determine glioma-associated antigen-specific T-cell responses via IFNγ activity. |
| NCT03389802 | CD40 agonistic monoclonal antibody APX005M in treating pediatric patients with recurrent or refractory brain tumors | CD40 agonistic monoclonal antibody | I | Evaluate safety of CD40 agonistic monoclonal antibody. Determine pharmacokinetics, maximum tolerated dose schedule, and phase II dosing. |
| NCT02359565 | Pembrolizumab in treating younger patients with recurrent, progressive, or refractory high-grade gliomas, diffuse intrinsic pontine gliomas, hypermutated brain tumors, ependymoma or medulloblastoma | Pembrolizumab (anti-PD1) | I | Establish safety and adverse effects with the administration of adult recommended dose. Estimate sustained objective response rate. Determine changes in immunophenotypic profile of CD8+ T cells. |
| NCT04323046 | Immunotherapy (Nivolumab and Ipilimumab) before and after surgery for the treatment of recurrent or progressive high grade glioma in children and young adults | Nivolumab (anti-PD1) and ipilimumab (anti-CTLA4) | I | Measure changes in the genetic signature of the tumour microenvironment after treatment. Safety and tolerability. |
| NCT03690869 | REGN2810 in pediatric patients with relapsed, refractory solid, or central nervous system (CNS) tumors and safety and efficacy of REGN2810 in combination with radiotherapy in pediatric patients with newly diagnosed or recurrent glioma | Cemiplimab (anti-PD1) with radiotherapy | I/II | Evaluate safety and anticipated phase II dose of cemiplimab. Determine pharmacokinetics of cemiplimab. Confirm safety and phase II dose of cemiplimab with radiotherapy. |
| NCT04049669 | Pediatric trial of indoximod with chemotherapy and radiation for relapsed brain tumors or newly diagnosed DIPG | Indoximod (indoleamine 2,3-dioxygenase inhibitor) with chemotherapy and/or radiation | II | Improve antitumour immune responses and measure outcomes according to iRANO criteria. |
DC = dendritic cell; iRANO = immune-related response assessment for neuro-oncology; poly-ICLC = polyinosinic-polycytidylic acid (poly[I:C]) stabilized by lysine and carboxymethylcellulose; TAA-T = tumour multi-antigen associated specific cytotoxic T lymphocytes; TTRNA-DC = total tumour RNA loaded dendritic cell vaccine.