Pediatric Neuro-Oncology Clinical Trials

ACNS1831: A Phase 3 Randomized Study of Selumetinib (IND #77782) Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Diagnosis: Low-Grade Glioma (LGG)

ACNS1833: A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
Diagnosis: Low-Grade Glioma (LGG)

NF1-OPG Natural History Study: Developing Evidence-Based Criteria for Initiating Treatment for Neurofibromatosis Type 1 Associated Optic Pathway Glioma/CTF 004: The Collection of Blood Samples from Patients with NF1 for Research Purposes
Diagnosis: Optic pathway glioma/NF1 (LGG)

Frameshift Peptides of Children with Neurofibromatosis Type 1 (NF1) and Either Low-Grade Gliomas or Plexiform Neurofibromas
Diagnosis: NF1 & LGG or Plexiform Neurofibromas

PBTC-029B: Phase I and Phase II of AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma
Diagnosis: Low Grade Glioma (LGG)

PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: LGG/HGG & LGG with NF1

Frameshift Peptides of Children with Neurofibromatosis Type 1 (NF1) and Either Low-Grade Gliomas or Plexiform Neurofibromas
Diagnosis: NF1 & LGG or Plexiform Neurofibromas

GD2 CAR-T: Phase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor (CAR) T cells (GD2CART) for Diffuse Intrinsic Pontine Gliomas (DIPG) and Spinal Diffuse Midline Glioma (DMG)
Diagnosis: Diffuse Intrinsic Pontine Glioma (DIPG) and Spinal Cord Diffuse Midline Glioma (DMG) that is H3K27 mutated

PBTC-047: Phase 1 Trial of Panobinostat in Children with Diffuse Intrinsic Pontine Glioma
Diagnosis: Diffuse Intrinsic Pontine Glioma (DIPG)

PBTC-051: Phase I Study to Evaluate the Safety and Tolerability of the CD40 Agonistic Monoclonal Antibody APX005M in Pediatric Subjects with Recurrent/Refractory Brain Tumors and Newly Diagnosed Brain Stem Glioma
Diagnosis: Stratum I Recurrent or Refractory Primary Malignant CNS tumor patients; Stratum II newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)

COG-ACNS-1721: A Phase 2 Study of Veliparib and Local Irradiation, Followed by Maintenance Veliparib and Temozolomide, in patients with Newly Diagnosed High-Grade Glioma (HGG) without H3 K27M or BRAFV600E Mutations
Diagnosis: High Grade Glioma (HGG)

COG-ACNS-1723: A Phase 2 Study of Dabrafenib (NSC# 763760) with Trametinib (NSC# 763093) after Local Irradiation in Newly-Diagnosed BRAFV600-Mutant High-Grade Glioma (HGG) (IND# 145355)
Diagnosis: High Grade Glioma (HGG)

PBTC-045: A Safety and Preliminary Efficacy trial of MK-3475 (pembrolizumab; anti-PD-1) in children with recurrent, progressive or refractory high-grade gliomas (HGG), DIPGs and hypermutated brain tumors
Diagnosis: Non brainstem-HGG, Hypermutated Tumors, Ependymoma, and Medulloblastoma

PBTC-048: Feasibility Trial of Optune for Children with Recurrent or Progressive Supratentorial High-Grade Glioma and Ependymoma
Diagnosis: High Grade Glioma (HGG); Ependymoma

PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Medulloblastoma, DIPG, and HGG

PBTC-051: Phase I Study to Evaluate the Safety and Tolerability of the CD40 Agonistic Monoclonal Antibody APX005M in Pediatric Subjects with Recurrent/Refractory Brain Tumors and Newly Diagnosed Brain Stem Glioma
Diagnosis: Stratum I Recurrent or Refractory Primary Malignant CNS tumor patients; Stratum II newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)

PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: LGG/HGG & LGG with NF1

PBTC-056: A Phase I Study Of The Adam-10 Inhibitor, INCB7839 In Children With Recurrent/Progressive High-Grade Gliomas To Target Microenvironmental Neuroligin-3
Diagnosis: High Grade Glioma (HGG)/DIPG, H3K27-Mutant DMG, Glioblastoma Multiforme, Anaplastic Astrocytoma and Anaplastic Oligodendroglioma

There are currently no trials for Newly Diagnosed Ependymomas.  

PBTC-045: A Safety and Preliminary Efficacy trial of MK-3475 (pembrolizumab; anti-PD-1) in children with recurrent, progressive or refractory high-grade gliomas (HGG), DIPGs and hypermutated brain tumors
Diagnosis: Non brainstem-HGG, Hypermutated Tumors, Ependymoma, and Medulloblastoma

PBTC-048: Feasibility Trial of Optune for Children with Recurrent or Progressive Supratentorial High-Grade Glioma and Ependymoma
Diagnosis: High Grade Glioma (HGG); Ependymoma

SJMB12: A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma
Diagnosis: Medulloblastoma

ACNS1422: A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients
Diagnosis: Medulloblastoma

PBTC-045: A Safety and Preliminary Efficacy trial of MK-3475 (pembrolizumab; anti-PD-1) in children with recurrent, progressive or refractory high-grade gliomas (HGG), DIPGs and hypermutated brain tumors
Diagnosis: Non brainstem-HGG, Hypermutated Tumors, Ependymoma, and Medulloblastoma

PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Medulloblastoma, DIPG, and HGG

PBTC-053: A Pediatric Brain Tumor Consortium Phase I/ II and Surgical Study of CX-4945 in Patients with Recurrent SHH Medulloblastoma
Diagnosis: Medulloblastoma

SJATRT: Phase 2 Study of Alisertib as a single agent in Recurrent or Progressive Central Nervous System (CNS) Atypical Teratoid Rhabdoid Tumors (ATRT) and Extra-CNS Malignant Rhabdoid Tumors (MRT) and in Combination Therapy in Newly Diagnoses ATRT
Diagnosis: Atypical Teratoid Rhabdoid Tumors (ATRT)

 PBTC-051: Phase I Study to Evaluate the Safety and Tolerability of the CD40 Agonistic Monoclonal Antibody APX005M in Pediatric Subjects with Recurrent/Refractory Brain Tumors and Newly Diagnosed Brain Stem Glioma
Diagnosis: Stratum I Recurrent or Refractory Primary Malignant CNS tumor patients; Stratum II newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)

PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: LGG/HGG & LGG with NF1

Frameshift Peptides of Children with Neurofibromatosis Type 1 (NF1) and Either Low-Grade Gliomas or Plexiform Neurofibromas
Diagnosis: NF1 & LGG or Plexiform Neurofibromas

NF1-OPG Natural History Study: Developing Evidence-Based Criteria for Initiating Treatment for Neurofibromatosis Type 1 Associated Optic Pathway Glioma/CTF 004: The Collection of Blood Samples from Patients with NF1 for Research Purposes
Diagnosis: Optic pathway glioma

SpringWorks: MEK-NF-201: A Phase 2b Trial of the MEK 1/2 Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients with Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) that are Causing Significant Morbidity
Diagnosis: Neurofibroma

GD2 CAR-T: Phase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor (CAR) T cells (GD2CART) for Diffuse Intrinsic Pontine Gliomas (DIPG) and Spinal Diffuse Midline Glioma (DMG)
Diagnosis: Diffuse Intrinsic Pontine Glioma (DIPG) and Spinal Cord Diffuse Midline Glioma (DMG) – H3K27 mutated

POE16-01: A Phase I/II Study of Neratinib in Pediatric Patients with Relapsed/ Refractory Solid Tumors or Hematologic Malignancies
Diagnosis: Solid & Central Nervous System (CNS) Tumor, Lymphoma, or Leukemia

PBTC-053: A Pediatric Brain Tumor Consortium Phase I/ II and Surgical Study of CX-4945 in Patients with Recurrent SHH Medulloblastoma
Diagnosis: Medulloblastoma

ADVL1622: Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases, in Children and Young Adults with Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors
Diagnosis: MET aberration, RET mutations, or AXL aberrations

ALTE07C1: Neuropsychological, Social, Emotional and Behavioral Outcomes in Children with Cancer
Diagnosis: Non-disease specific

APEC14B1: The Project: Everychild Protocol: A Registry, Eligibility, Screening, Biology, and Outcome Study
Diagnosis: Neoplasm

APEC1621A:  LOXO-101 in patients with tumors harboring actionable NTRK fusions
Diagnosis: Solid Tumors

APEC1621: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)
Diagnosis: CNS diagnosis with specific mutation

• APEC1621A: Phase 2 study of LOXO-101 (Larotrectinib) in patients with tumors harboring NTRK fusions
• APEC1621B: Phase 2 study of JNJ-42756493 (Erdafitinib) in patients with tumors harboring FGFR alterations
• APEC1621C: Phase 2 study of Tazemetostat in patients with tumors harboring alterations in EZH2 or members of the SWI/SNF complex
• APEC1621D: Phase 2 study of LY3023414 in patients with solid tumors
• APEC1621E: Phase 2 study of Selumetinib in patients with tumors harboring activating MAPK pathway mutations
• APEC1621F: Phase 2 study of Ensartinib in patients with tumors harboring ALK or ROS1 genomic alterations
• APEC1621G: Phase 2 study of Vemurafenib in patients with tumors harboring BRAF V600 mutations
• APEC1621H: Phase 2 study of Olaparib in patients with tumors harboring defects in DNA damage repair genes
• APEC1621I: Phase 2 subprotocol of Palbociclib in Patients with Tumors Harboring Activating Alterations in Cell Cycle Genes
• APEC1621J: Phase 2 subprotocol of BVD-523FB (ulixertinib) in patients with tumors harboring activating MAPK Pathway Mutations

UCSC Treehouse: California Initiative to Advance Precision Medicine: California Kids Cancer Comparison, and Treehouse Childhood Cancer Initiative
Diagnosis: All relapsed/refractory solid tumors

Pediatric Brain Tumor Consortium

The Children's Oncology Group

SpringWorks Therapeutics