A Phase I open-label, multicenter study, to evaluate the safety, feasibility, and maximum tolerated dose (MTD) of treating children with newly diagnosed DIPG or recurrent neuroblastoma with molecular targeted therapy in combination with adoptive cell therapy (Total tumor mRNA-pulsed autologous Dendritic Cells (DCs) (TTRNA-DCs), Tumor-specific ex vivo expanded autologous lymphocyte transfer (TTRNA-xALT) and Autologous G-CSF mobilized Hematopoietic Stem Cells (HSCs)).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
24
There will be two immunotherapy products manufactured and administered to subjects enrolled on this trial. The first product will be autologous dendritic cells (DCs) loaded with total tumor messenger ribonucleic acid (mRNA) (TTRNA) derived from malignant tumors. The second product will be autologous T lymphocytes stimulated ex vivo against TTRNA antigens for autologous transfer (TTRNA-xALT). DCs are professional antigen-presenting cells critical for the initiation of B and T-cell responses in vivo.
University of Florida
Gainesville, Florida, United States
RECRUITINGLevine Children's Hospital
Charlotte, North Carolina, United States
ACTIVE_NOT_RECRUITINGPenn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, United States
RECRUITINGNumber of Participants with Dose Limiting Toxicities as a Measure of Safety and Tolerability
To evaluate the dose-limiting toxicities (DLTs) and to establish the maximum tolerated dose (MTD) of treating children with molecular targeted therapy in combination with adoptive cellular therapy
Time frame: 2 years
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
To evaluate the overall safety profile of study treatment
Time frame: 2 years plus 30 days
Number of Participants that are able to have vaccine produced and delivered
To evaluable the feasibility of producing and administering the protocol directed therapy
Time frame: 2 years
Number of participants with progression free survival (PFS) during study
To determine the activity of treatments chosen based on Progression free survival (PFS)
Time frame: 7 years
Number of participants with overall survival (OS) during study
To determine the activity of treatments chosen based on Overall Survival (OS)
Time frame: 7 years
Determine the Overall Response Rate (ORR) of Participants using INSS Response Evaluation Criteria for NB and RANO criteria for DIPG
To determine the activity of treatments chosen based on Overall Response Rate (ORR)
Time frame: 2 years
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