Central Nervous System Tumors

A central nervous system (CNS) tumor begins when healthy cells in the brain or the spinal cord change and grow out of control, forming a mass, or tumor. If the tumor is malignant, it can spread to other parts of the body or brain, effecting a child’s thought processes and movements.

Our neuro-oncology team leads many different CNS tumor trials in coordination with sites around the world testing combinations of existing treatments, new approaches to radiation therapy or surgery, and new ways to relieve symptoms and side effects during treatment.

A Multicenter Phase 2/3 Trial of the Efficacy and Safety of Intracerebroventricular Radioimmunotherapy using 131I- omburtamab for Neuroblastoma Central Nervous System/Leptomeningeal Metastases.

Protocol ID: Y-mAbs 101

Disease: NBL

Who can participate?: Patients must be b/t the ages of birth and 18 yr at the time of screening. Life expectancy of at least 3 months. Must have a histologically confirmed dx of NB w/ relapse in the CNS or LM. Must have stable systemic disease not requiring chemo/ immunotherapy as judged by the investigator. 

Lead Researcher: Keri A. Streby
A Phase 1 Open Label, Multi Center Study to Evaluate the Safety and Tolerability of BXQ-350 in Children with Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) and Diffuse Midline Glioma (DMG)

Protocol ID: BXQ-350.AD

Diseases: Diffuse Midline Glioma and Diffuse intrinsic pontine glioma (DIPG)

Who Can Participate?: New diagnosis, ages 1 to 30

Lead Researcher: Maryam Fouladi, MD

A Phase 1b Study of PTC596 in Children with Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma

Protocol ID: CONNECT1702

Diseases: Diffuse intrinsic pontine glioma (DIPG) and High Grade Glioma (HGG)

Who Can Participate?: New diagnosis, ages 1 to 21

Lead Researcher: Maryam Foudladi, MD

A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neuroblastoma

Protocol ID: ANBL1821

Disease: NBL

Who can participate?: Patients ≥ 1 year of age at the time of enrollment are eligible for this study. Patients must have had histologic verification of neuroblastoma or ganglioneuroblastoma or demonstration of neuroblastoma cells in the bone marrow with elevated urinary catecholamines at the time of initial diagnosis. Patients must have ONE of the following:

  1. First episode of recurrent disease following completion of aggressive multi-drug frontline therapy.
  2. First episode of progressive disease during aggressive multi-drug frontline therapy.
  3. Primary resistant/refractory disease

Lead Researcher: Mark A. Ranalli

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)

Protocol ID: ACNS1723

Diseases: CNS Tumors High Grade Glioma (HGG)

Who Can Participate?: New diagnosis, ages 3 to 21

Lead Researcher: Mark A. Ranalli, MD

A Phase 2 Study of Veliparib (ABT-888, IND # 139199) and Local Irradiation, Followed by Maintenance Veliparib and Temozolomide, in Patients with Newly Diagnosed High-Grade Glioma (HGG) without H3 K27M or BRAFV600 Mutations

Protocol ID: ACNS1721

Disease: CNS

Who can participate?: Stratum 2 (IDH mutant): Patients must be ≥ 3 years of age and ≤ 25 years of age at the time of enrollment. Patients must have eligibility confirmed by rapid central pathology and central molecular screening reviews performed on APEC14B1 (see Section 3.1): Newly-diagnosed high-grade glioma such as anaplastic astrocytoma or glioblastoma, Negative results for H3 K27M by immunohistochemistry (IHC), Negative results for BRAFV600 mutation by Next-Generation Sequencing (NGS)."

Lead Researcher: Mark A. Ranalli

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)

Protocol ID: ACNS1831

Diseases: CNS Tumors, Low Grade Glioma (LGG), and Neurofibromatosis

Who Can Participate?: New diagnosis, ages 2 to 21

Lead Researcher: Mark A. Ranalli, MD

A Phase I Dose Escalation Study of Autologous Expanded Natural Killer (NK) Cells for Immunotherapy of Relapsed Refractory Neuroblastoma with Dinutuximab +/- Lenalidomide

Protocol ID: NANT 2013-01

Disease: NBL

Who can participate?: Must be ≤ 30 yrs of age. Diagnosis of HR neuroblastoma either by histologic verification of neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines. Recurrent/progressive, refractory or persistent disease based upon the response to prior therapy according to the protocol's guidelines.

Lead Researcher: Keri A. Streby

A Safety and Pharmacokinetic Study of Single Agent REGN2810 in Pediatric Patients with Relapsed or Refractory Solid or CNS Tumors and a Safety and Efficacy Trial of REGN2810 in Combiantion with Radiotherapy in Pediatric Patients with ndDIPG, ndHGG and rHGG

Protocol ID: PNOC013

Diseases: Diffuse intrinsic pontine glioma (DIPG) and High Grade Glioma (HGG)

Who Can Participate?: New Diagnosis, Relapse/Refractory, ages 3 to 25

Lead Researcher: Diana S. Osorio, MD

A Target Validation Study of Fimepinostat in Children and Young Adults with Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG), Recurrent Medulloblastoma, or Recurrent HGG

Protocol ID: c cc

Diseases: Diffuse intrinsic pontine glioma (DIPG), High Grade Glioma (HGG), and Medulloblastoma

Who Can Participate?: New Diagnosis, Relapse/Refractory, ages 3 to 39

Lead Researcher: Diana S. Osorio, MD

MIBG or Crizotinib add to Intensive Therapy for children with newly diagnosed with high risk neuroblastoma

Protocol ID: ANBL1531

Disease: NBL

Who can participate?: Enrollment ANBL00B1 or APEC14B1; less than 30; dx NBL high risk with no prior systemic therapy.

Lead Researcher: Mark A. Ranalli

NCH Protocol PBTB: Pediatric Brain Tumor Bank

Protocol ID: PBTB

Diseases: CNS Tumors

Who Can Participate?: Ages 0 to 21

Lead Researcher: Diana S. Osorio, MD

Neuroblastoma Classification Biology Studies

Protocol ID: ANBL00B1

Disease: NBL

Who can participate?: All newly diagnosed patients with suspected neuroblastoma, suspected ganglioneuroblastoma, or suspected ganglioneuroma/maturing subtype seen at COG institutions are eligible for this study.

Lead Researcher: Mark A. Ranalli

PHASE 2 SUBPROTOCOL OF AG-120 (IVOSIDENIB) IN PATIENTS WITH TUMORS HARBORING IDH1 MUTATIONS

Protocol ID: APEC1621K

Diseases: CNS Tumors, Langerhans cell histiocytosis (LCH), Neuroblastoma (NBL), Non-Hodgkin lymphoma (NHL), and Sarcoma

Who Can Participate?: Replapse/Refractory, age 1 to 21

Lead Researcher: Mark A. Ranalli, MD

PHASE 2 SUBPROTOCOL OF TIPIFARNIB IN PATIENTS WITH TUMORS HARBORING HRAS GENOMIC ALTERATIONS

Protocol ID: APEC1621M

Diseases: CNS Tumors, Carcinoma, Diffuse intrinsic pontine glioma (DIPG), Ewing Sarcoma (EWS), Ganglioneuroblastoma (GBN), Germ cell tumor, High Grade Glioma (HGG), Low Grade Glioma (LGG), Medulloblastoma, Neuroblastoma (NBL), Non-rhabdomyosarcoma soft tissue sarcoma (NRSTS), Osteosarcoma (OS), Retinoblastoma, Rhabdomyosarcoma, Sarcoma, Synovial sarcoma, and Wilms tumor

Who Can Participate?: Ages 1 to 21

Lead Researcher: Mark A. Ranalli, MD

PNOC 005: A Phase 1 Study of Modified Measles Virus (MV-NIS) for the Treatment of Children and Young Adults with Recurrent Medulloblastoma or Recurrent ATRT

Protocol ID: PNOC005

Diseases: Atypical Teratoid Rhabdoid Tumor, CNS Tumors, and Medulloblastoma

Who Can Participate?: Replase/Refractory, ages 1 to 39

Lead Researcher: Diana S. Osorio, MD

PNOC 007, H3.3K27M Specific Peptide Vaccine Combined with poly-ICLC for the Treatment of newly diagnosed HLA-A2+ H3.3K27M Positive Diffuse Intrinsic Pontine Glioma (DIPG) as well as other newly diagnosed HLA-A2+ H3.3K27M Positive Gliomas

Protocol ID: PNOC007

Diseases: Diffuse intrinsic pontine glioma (DIPG), High Grade Glioma (HGG), and Low Grade Glioma (LGG)

Who Can Participate?: New diagnosis, ages 3 to 21

Lead Researcher: Diana S. Osorio, MD

Study of 12Il-Metaiodabenzylguanidine (131i-MIBG) or Crizotinib added to intensive therapy for children with newly diagnosed HR NBL

Protocol ID: ANBL1531

Disease: NBL

Who can participate?: Must be enrolled on ANBL00B1 or APEC14B1; new diagnosed NBL or ganglioneuroblastoma; >1 y <30y age.

Lead Researcher: Mark A. Ranalli

Utilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients with Non-High-Risk Neuroblastoma *Patient must be enrolled on ANBL00B1 prior to enrollment

Protocol ID: ANBL1232

Disease: NBL

Who can participate?: < 12 months of age at diagnosis with INRG Stage L1 or < 18 months of age at diagnosis with INRG Stage L2 or Stage Ms neuroblastoma/ganglioneuroblastoma.

Lead Researcher: Mark A. Ranalli

Late Effects After High Risk Neuroblastoma Study

Protocol ID: ALTE15N2

Disease: NBL

Who can participate?: Study Patients must have been enrolled on COG Neuroblastoma Biology Study ANBL00B1.  Patients must be ≥ 5 and ≤ 50 years of age at study enrollment.  Patient must have been diagnosed with high-risk neuroblastoma per ANBL00B1 definition. Patient must have been diagnosed on or after January 1, 2000. At least 5 years must have elapsed since diagnosis.

Lead Researcher: Mark A. Ranalli, MD

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

Protocol ID: ACNS1422

Disease: CNS

Who can participate?: Patients must be greater than or equal to 3 years and less than 22 years of age at the time of enrollment. Patients must be newly diagnosed and have: Eligibility confirmed by rapid central pathology and molecular screening review on APEC14B1 (see Section 3.1): classical histologic type (non LC/A) WNT medulloblastoma, positive nuclear β-catenin by IHC, positive for CTNNB1 mutation, negative for MYC and MYCN by FISH.

Lead Researcher: Mark A. Ranalli, MD

A Phase III Study Comparing Two Carboplatin Containing Regimens for Children and Young Adults with Previously Untreated Low Grade Glioma

Protocol ID: LGG-1403 Untreated LGG

Disease: CNS

Who can participate?: Patients must be less than 21 years of age at study entry. Patients who are newly OR previously diagnosed with low grade glioma (LGG), who have not been treated with any modality besides surgery or corticosteroids. Untreated astrocytomas or other eligible tumors interpreted as low grade.

Lead Researcher: Diana S. Osorio, MD

A Phase II Study of 131I-Metaiodobenzylguanidine (131I - MIBG) Therapy for Patients with MIBG Avid Tumors

Protocol ID: MIBG

Disease: NBL

Who can participate?: Patients must be between 12 months and 65 years at the time of study enrollment.

Lead Researcher: Mark A. Ranalli, MD

NMTT - Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)

Protocol ID: NMTRC014

Disease: NBL

Who can participate?: ≤ 30.99 years of age and classified as high risk by the criteria used by COG or SIOPEN at the time of diagnosis.

Lead Researcher: Keri A. Streby, MD