Brain and Central Nervous System Cancers Clinical Studies

Some of the many Cancer and Blood Disorders research opportunities available related to cancers of the brain and central nervous system (CNS) are listed below. Select a study to review a brief description and requirements. Parents or guardians of children who might be eligible are encouraged to inquire about enrollment. Contact information is provided at the end of each study.

Please note that Pilot and Phase I studies are the most experimental and should be considered only after other treatments or trials have been attempted without success.

Thank you for exploring these opportunities and helping us to find cures and prevent childhood disease.

Clinical Studies

Researchers

Central Nervous System Germ Cell Tumor Treatment with Chemotherapy and Radiation – Phase II

ACNS1123: Response-Based Radiation Therapy for Patients with Localized Central Nervous System Germ Cell Tumors

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Central Nervous System Immunotherapy Using MK-3475 – Phase I

PBTC-045: Trial of MK-3475 in Children with Recurrent, Progressive, or Refractory Diffuse Intrinsic Pontine Glioma, Non-brainstem High-grade Gliomas, Ependymoma, Medulloblastoma, or Hypermutated Brain Tumors

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Central Nervous System Tumor Treatment with APX005M – Phase I

PBTC-051: 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

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Central Nervous System Tumor Treatment with Chemotherapy followed by Response-Based Radiation – Phase II

ACNS2021: A Study of a New Way to Treat Children and Young Adults with a Brain Tumor Called NGGCT

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Central Nervous System Tumor Treatment with Savolitinib – Phase I

PBTC-049: Study of Savolitinib in Recurrent, Progressive, or Refractory Medulloblastoma, High-Grade Glioma, or Diffuse Intrinsic Pontine Glioma

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Children’s Oncology Group Long-Term Follow-up of Study Participants

ALTE05N1: Umbrella Long-Term Follow-Up Protocol for Participants with and without a History of Cancer

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Children’s Oncology Group’s Project: EveryChild

APEC14B1: Project:Every Child, A Registry, Eligibility Screening, Biology and Outcome Study

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DIPG Treatment with Panobinostat – Phase I

PBTC-047: Panobinostat in Children with Diffuse Intrinsic Pontine Glioma

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Ependymoma Treatment with Maintenance Chemotherapy – Phase III

ACNS0831: Trial of Post-Radiation Chemotherapy in Patients with Newly Diagnosed Ependymoma

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Ependymoma Vaccine Trial – Phase I

Treatment for Recurrent Ependymomas in Children Using HLA-A2 Restricted Tumor Antigen Peptides in Combination With Imiquimod

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Fitness Program for Adolescent and Young Adult Childhood Cancer Survivors: The StepByStep Study – Phase III

ALTE2031: StepByStep: A Trial of a Mobile Health and Social Media Physical Activity Intervention Among Adolescent and Young Adult Childhood Cancer Survivors

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Fitness Program for Children and Adolescents Following Cancer Therapy – Phase III

ALTE1631: A Web-based Physical Activity Intervention among Children and Adolescents

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Glioma (Low-Grade) Treatment using Selumetinib with Vinblastine and Selumetinib Alone – Phase III

ACNS1931: A Study to Compare Treatment with the Drug Selumetinib Alone versus Selumetinib and Vinblastine in Patients with Recurrent or Progressive Low-Grade Glioma

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Glioma (High-Grade and DIPG) Therapy with Selinexor and Radiation – Phase I/II

ACNS1821: A Study of the Drug Selinexor with Radiation Therapy in Patients with Newly Diagnosed DIPG and High-Grade Glioma

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Glioma (High-Grade) Treatment with Dabrafenib and Trametnib After Irradiation – Phase II

ACNS1723: A Study of the Drugs Dabrafenib and Trametinib after Radiation Therapy in Patients with Newly Diagnosed High-Grade Glioma

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Glioma (Low-Grade) Treatment using Selumetinib versus Standard Chemotherapy – Phase III

ACNS1833: A Study of the Drugs Selumetinib vs. Carboplatin/Vincristine in Patients with Low-Grade Glioma

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Gliomas (Low-Grade) Vaccine Study – Phase II

Vaccinations with HLA-A2-Restricted Glioma Antigen-Peptides in Combination with Poly-ICLC for Children with Recurrent Unresectable Low-Grade Gliomas (LGG)

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Glioma Treatment with AZD6244 – Phase I/II

PBTC-029B: Study of AZD6244 for Recurrent or Refractory Pediatric Low-Grade Glioma

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High-Risk Medulloblastoma Treatment with Chemotherapy and Radiation – Phase III

ACNS0322: Efficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients

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Medulloblastoma Therapy Reduction Following Surgery – Phase II

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

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Medulloblastoma Treatment with Carboplatin and Isotretinoin – Phase III

ACNS0332: Efficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients

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Neurofibromatosis and Glioma Therapy using Selumetinib – Phase III

ACNS1831: A Study of the Drugs Selumetinib versus Carboplatin/Vincristine in Patients with Neurofibromatosis and Low-Grade Glioma

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Pediatric Tumors and Specimen Study

ACNS02B3: A Children’s Oncology Group Protocol for Collecting and Banking Pediatric Specimens Including Rare Pediatric Tumors

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Pilocytic Astrocytoma and Optic Pathway Glioma Treatment with Lenalidomide – Phase II

ACNS1022: Trial of Lenalidomide in Pediatric Patients with Recurrent, Refractory or Progressive Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas

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SHH Medulloblastoma Treatment with CX-4945 – Phase I/II

PBTC-053: Surgical Study of CX-4945 in Patients with Recurrent SHH Medulloblastoma

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