Clinical Trial

PNOC008: A Pilot Trial Testing the Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan in Children and Young Adults with High Grade Glioma (Excluding Diffuse Intrinsic Pontine Glioma)

Please note, this study has closed to accrual and is no longer accepting patients. || Patients with newly diagnosed high grade glioma (including midline and excluding DIPG) who have undergone surgery as part of their standard of care and can provide the necessary tumor tissue for analysis will be eligible for this study. The tissue will be sent for detailed profiling. After the surgery, patients will be treated with focal irradiation per institutional guidelines. After completion of radiation therapy, patients will follow the specialized tumor board recommendation. The treatment will be based on each child’s individual tumor profile and will include up to 4 medications. Depending on the individual treatment plan, the tumor board will also recommend required study observations to assure safety for the patient.

Despite years of research and multiple clinical trials in patients with High Grade Gliomas, no significant survival advantage has been achieved. This study will use an individualized treatment approach based on whole exome sequencing (WES), a targeted panel approach (UCSF 500 gene panel), and transcriptome profile as determined by gene expression analysis (RNA sequencing). The transcriptomic and genomic profiling will be performed in collaboration with the genomics team at UCSF as well as NantOmics. We have shown that it is feasible to perform detailed profiling of DIPG specimens (PNOC003; NCT02274987) and that a therapy recommendation can be issued within 21 business days. We have also shown that there are no major safety concerns with regard to being treated with an individualized therapy plan with up to 4 FDA approved drugs in combination.

PRIMARY OBJECTIVES

  • To determine the progression free survival at 12 months of children and young adults with newly diagnosed High Grade Glioma who underwent maximal safe surgical resection followed by focal radiation therapy who are being treated according to a specialized tumor board recommendation that is based on WES, targeted panel (UCSF500 gene panel), and RNA-Seq analysis of the tumor.
  • To determine the overall survival at 12 months in children with newly diagnosed midline High Grade Glioma (excluding DIPG) who underwent maximal safe surgical resection followed by focal radiation therapy who are being treated according to a personalized treatment recommendation that is based on WES, targeted panel (UCSF 500 gene panel), and RNA seq analysis of the tumor.

 

SECONDARY OBJECTIVES

  • To determine the safety and describe the toxicity of treating children and young adults with newly diagnosed High Grade Glioma who underwent maximal safe surgical resection followed by focal radiation therapy who are being treated) according to a specialized tumor board recommendation that is based on WES, targeted panel (UCSF500 gene panel), and RNA-Seq analysis of the tumor.

Funding is provided by The V Foundation.

Please note, this study has closed to accrual and is no longer accepting patients. || Patients with newly diagnosed high grade glioma (including midline and excluding DIPG) who have undergone surgery as part of their standard of care and can provide the necessary tumor tissue for analysis will be eligible for this study. The tissue will be sent for detailed profiling. After the surgery, patients will be treated with focal irradiation per institutional guidelines. After completion of radiation therapy, patients will follow the specialized tumor board recommendation. The treatment will be based on each child’s individual tumor profile and will include up to 4 medications. Depending on the individual treatment plan, the tumor board will also recommend required study observations to assure safety for the patient.

Despite years of research and multiple clinical trials in patients with High Grade Gliomas, no significant survival advantage has been achieved. This study will use an individualized treatment approach based on whole exome sequencing (WES), a targeted panel approach (UCSF 500 gene panel), and transcriptome profile as determined by gene expression analysis (RNA sequencing). The transcriptomic and genomic profiling will be performed in collaboration with the genomics team at UCSF as well as NantOmics. We have shown that it is feasible to perform detailed profiling of DIPG specimens (PNOC003; NCT02274987) and that a therapy recommendation can be issued within 21 business days. We have also shown that there are no major safety concerns with regard to being treated with an individualized therapy plan with up to 4 FDA approved drugs in combination.

PRIMARY OBJECTIVES

  • To determine the progression free survival at 12 months of children and young adults with newly diagnosed High Grade Glioma who underwent maximal safe surgical resection followed by focal radiation therapy who are being treated according to a specialized tumor board recommendation that is based on WES, targeted panel (UCSF500 gene panel), and RNA-Seq analysis of the tumor.
  • To determine the overall survival at 12 months in children with newly diagnosed midline High Grade Glioma (excluding DIPG) who underwent maximal safe surgical resection followed by focal radiation therapy who are being treated according to a personalized treatment recommendation that is based on WES, targeted panel (UCSF 500 gene panel), and RNA seq analysis of the tumor.

 

SECONDARY OBJECTIVES

  • To determine the safety and describe the toxicity of treating children and young adults with newly diagnosed High Grade Glioma who underwent maximal safe surgical resection followed by focal radiation therapy who are being treated) according to a specialized tumor board recommendation that is based on WES, targeted panel (UCSF500 gene panel), and RNA-Seq analysis of the tumor.

Funding is provided by The V Foundation.

How to Enroll

If you believe your child or patient is eligible for this trial, contact the closest participating site or email us for more information.