Pediatric Clinical Trials

We believe ending cancer will require a weapon as adaptable and multiform as the disease itself.
We Believe

We believe ending cancer will require a weapon as adaptable and multiform as the disease itself.

  • PEDIATRIC TRIALS

    Study:  Developing evidence-based criteria for initiating treatment for neurofibromatosis type I-associated optic pathway glioma
    PI:  David Van Mater, MD, PhD 
    Phase:  N/A (Observational) 
    Category:  Pediatric Neuro-Oncology 
    Purpose:  To understand the natural history of vision in NF1 patients with an optic pathway glioma and to determine predictors of future vision loss or change in tumor size. For detailed information, please view this trial at ClinicalTrials.gov 
    IRB number:  Pro00066558 
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288

     

    Study:  The collection of blood samples from patients with NF1 for research purposes
    PI:  
    David Van Mater, MD, PhD 
    Phase:  N/A (Biorepository) 
    Category:  Pediatric Neuro-Oncology 
    Purpose:  To augment an existing biobank resource for neurofibromatosis type 1 (NF1) research. For detailed information, please view this trial at ClinicalTrials.gov 
    IRB number:  Pro00068686 
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288

     

    Study: CONNECT1702: A Phase 1b Study of PTC596 in Children with Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
    PI:  David Ashley, PhD
    Phase: 1b
    Category: Pediatric Neuro-Oncology
    Purpose: In this research study the investigators want to learn more about the safety of the study drug, PTC596 has when taken during radiation. The investigators also want to learn about the effects, if any, these drugs have on children and young adults with brain tumors. The investigators are asking people to be in this research study who have been diagnosed with a high grade glioma (HGG) including diffuse intrinsic pontine glioma (DIPG) to be in the research, because they have scheduled to have radiation to treat their cancer. The study is divided into two parts. The goal of the first part is to find the dose of PTC596 that can be given with radiation without causing serious side effects. The purpose of this surgical study is to test the amount of a study drug that may be found in the tumor and blood when given prior to and during a planned surgery for removal of the recurrent tumor. For more information, please visit this trial at ClinicalTrials.gov at NCT03605550
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288

     

    Study: CONNECT1903: A Pilot and Surgical Study of Larotrectinib for Treatment of Children with Newly-Diagnosed High-Grade Glioma with NTRK Fusion
    PI:  David Ashley, PhD
    Phase: 1
    Category: Pediatric Neuro-Oncology
    Purpose: This is a pilot study that will evaluate disease status in children that have been newly diagnosed high-grade glioma with TRK fusion. The evaluation will occur after 2 cycles of the medication (Larotrectinib) have been given. The study will also evaluate the safety of larotrectinib when given with chemotherapy in your children; as well as the safety larotrectinib when given post-focal radiation therapy. For more information, please visit this trial at ClinicalTrials.gov at NCT04655404
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288

     

    Study: CONNECT1906: Phase 2 Trial of a Novel Peptide Vaccine (PEP-CMV) Targeting CMV Antigen for Newly Diagnosed Pediatric High-grade Glioma and Diffuse Intrinsic Pontine Glioma and Recurrent Medulloblastoma
    PI:  Dan Landi, MD, and Eric Thompson, MD
    Phase: 2 (Not yet recruiting)
    Category: Pediatric Neuro-Oncology
    Purpose: This study will address the question of whether targeting CMV antigens with PEP-CMV can serve as a novel immunotherapeutic approach in pediatric patients with newly-diagnosed high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG) as well as recurrent medulloblastoma (MB). PEP-CMV is a vaccine mixture of a peptide referred to as Component A. Component A is a synthetic long peptide (SLP) of 26 amino acid residues from human pp65. The SLPs encode multiple potential class I, class II, and antibody epitopes across several haplotypes. Component A will be administered as a stable water:oil emulsion in Montanide ISA 51. For more information, please visit this trial at ClinicalTrials.gov at NCT05096481
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288

  • Recurrent 

     
    Study: PEP-CMV in Recurrent Medulloblastoma/Malignant Glioma (PRiME)
    PI:  Gary Archer, PhD
    Phase: 1
    Category: Pediatric Neuro-Oncology
    Purpose: The primary goal of this prospective clinical trial is to evaluate the safety of PEP-CMV in patients with recurrent medulloblastoma and malignant glioma. Patients with histologically-proven medulloblastoma or malignant glioma who had received definitive chemo-radiotherapy and subsequently had tumor recurrence/progression may be enrolled any time after recurrence/progression regardless of prior adjuvant therapy. PEP-CMV is a vaccine mixture of 2 peptides comprised in two components (referred to as Component A and Component B). Component A is a synthetic long peptide (SLP) of 26 amino acid residues from human pp65. Component B consists of a neutralizing antibody epitope from human CMV glycoprotein B (gB) conjugated to KLH. For more information, please visit this trial at ClinicalTrials.gov at NCT03299309
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288

     

    Study: FIREFLY-1: A Phase 2, Open-Label, Multicenter Study to Evaluate the Safety and Efficacy of the Oral Pan-RAF Inhibitor DAY101 in Pediatric Patients with BRAF-Altered, Recurrent or Progressive Low-Grade Glioma (DAY101-001)
    PI:
      Dan Landi, MD
    Phase: 2
    Category: Pediatric Neuro-Oncology
    Purpose: FIREFLY-1 is a Phase 2, multi center, open-label study to evaluate the safety and efficacy of oral pan-RAF inhibitor DAY101 in pediatric, adolescent, and young adult patients with recurrent or progressive low-grade glioma harboring a known BRAF alteration. For more information, please visit this trial at ClinicalTrials.gov at NCT04775485
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288

     

    Study: NORTH: A Phase II Study of Panobinostat in Paediatric, Adolescent and Young Adult Patients with Solid Tumours Including Osteosarcoma, Malignant Rhabdoid Tumour/Atypical Teratoid Rhabdoid Tumours and Neuroblastoma
    PI:
      David Ashley, PhD
    Phase: 2
    Category: Pediatric Neuro-Oncology
    Purpose: This is an open label, phase II, multi-centre study evaluating the anti-tumor activity of continuous, low dose of panobinostat in patients with recurrent or refractory solid tumors stratified by primary histology into osteosarcoma, malignant rhabdoid tumor/atypical teratoid rhabdoid tumor (MRT/ATRT), and neuroblastoma. Patients will be stratified at study entry by tumor type into three strata: osteosarcoma, MRT/ATRT and neuroblastoma [osteosarcoma and neuroblastoma arms are closed to enrolment]. Patients will be enrolled onto the study following completion of their conventional therapy including chemotherapy and/or radiation treatment and completion of a three-week wash out period. Panobinostat will then be administered as a continuous oral dose (starting at a de-escalated dose of 8mg/m2 per day), for up to 12 courses, a total of 48 weeks. The minimum dose is 2mg/m2 per day. Dosing will follow a dose de-escalation or escalation scheme for each stratum which will be determined by biological effect of the drug (measured in patient peripheral blood samples) and levels of toxicity (measured by dose limiting toxicity and adverse events observed). Dose levels for subsequent enrolments in each strata will be based on the de-escalated or escalated dose in each cohort. The final dose per strata will be that which achieves significant biological effect with acceptable toxicity that is maintained for a 4 week period. Patients or their parents/guardians will be required to maintain a drug diary to monitor drug usage throughout the trial. Patients will be followed for up to 2 years from completion of study therapy. For more information, please visit this trial at ClinicalTrials.gov at NCT04897880
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288

  • Consortiums

    Pacific Pediatric Neuro-Oncology Consortium (PNOC): The Pacific Pediatric Neuro-Oncology Consortium (PNOC) is an international consortium with centers within the United States, Europe, Asia and Australia. We are dedicated to bringing new therapies to children and young adults with brain tumors. Our goal is to improve outcomes by translating the latest findings in brain tumor biology into better treatments for these children. For more information, please visit: https://pnoc.us/

    COllaborative Network for NEuro-oncology Clinical Trials (CONNECT) Consortium: The COllaborative Network for NEuro-oncology Clinical Trials (CONNECT) conducts clinical trials in high-risk pediatric brain tumors (such as DIPG) to investigate combinations of novel drugs with traditional therapies. We seek to improve outcomes in the worst childhood brain tumors by combining innovative discoveries with “tried and true” therapy regimens through science, research, collaboration, and service. For more information, please visit: https://connectconsortium.org/