Pediatric Brain Tumor Clinical Trials

Doctors looking at medical imaging of a brain

A clinical trial is research designed to determine the most effective and safest ways to treat a disease and improve survival rates.  Clinical trials often provide children with rare or hard-to-treat conditions like brain tumors with access to innovative treatments that may help where traditional options fail. 

C.S. Mott Children’s Hospital is home to the largest cutting-edge pediatric cancer research effort in the state of Michigan.  Our pediatric oncology program currently offers dozens of clinical trials for children’s cancers, many of which were developed here at U-M.

We encourage patients and families to contact CCPBTC-CRA@med.umich.edu for more information and a current list of trials currently available for U-M patients.

U-M Investigator Initiated Trials for Brain Tumor Patients

Pediatric MI-ONCOSEQ: Personalized Medicine based on Molecular Profiling of Pediatric and Young Adult Patients with Cancer
Principal Investigators: Rajen Mody, MD, MS, and Arul Chinnaiyan, MD, PhD
Currently Enrolling

A Phase II Study of Dasatinib in Combination with Everolimus for Children with Gliomas Harboring PDGFR/FGFR Alterations
Principal Investigator: Carl Koschmann, MD
Currently Enrolling

Pediatric Brain Tumor Cerebrospinal Fluid (CSF) Fluid Registry
Principal Investigator: Carl Koschmann, MD
Currently Enrolling

Non-U-M Initiated Clinical Trials Available at U-M for Brain Tumor Patients

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

Principal Investigator: Andrea Franson, MD
Sponsor: SpringWorks Therapeutics
Currently Enrolling

A Study Comparing Two Carboplatin Containing Regimens for Children and Young Adults With Previously Untreated Low Grade Glioma
U-M Site Principal Investigator: Patricia Robertson, MD
Host Site: Lurie Children’s Hospital
Currently Enrolling

Combination Chemotherapy With or Without Etoposide Followed By an Autologous Stem Cell Transplant in Treating Young Patients With Previously Untreated Malignant Brain Tumors
U-M Site Principal Investigator: Carl Koschmann, MD
Host Site: Nationwide Children’s Hospital
Currently Enrolling

DIPG Registry and Repository
U-M Site Principal Investigator: Carl Koschmann, MD
Host Site: Cincinnati Children’s Hospital
Currently Enrolling

ONC201 in Newly Diagnosed Diffuse Intrinsic Pontine Glioma and Recurrent/Refractory Pediatric H3 K27M Gliomas
Principal Investigator: Carl Koschmann, MD
Host Site: Oncoceutics
Currently Enrolling

PNOC Trials

PNOC022: A Combination Therapy Trial using an Adaptive Platform Design for Children and Young Adults with Diffuse Midline Gliomas (DMGs) including Diffuse Intrinsic Pontine Gliomas (DIPGs) at Initial Diagnosis, Post-Radiation Therapy and at Time of Progression
Andrea Franson, MD and Carl Koschmann, MD are two of the study co-chairs nationally
Estimated enrollment start in Summer or Fall 2021

PNOC023: Open label Phase 1 and Target Validation study of ONC206 in Children and Young Adults with Newly Diagnosed or Recurrent Diffuse Midline Glioma (DMG), and Other Recurrent Primary Malignant Brain Tumors
Site PI: Carl Koschmann, MD
Estimated enrollment start in Summer or Fall 2021

PNOC026/DAY101-001/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
Site PI: Andrea Franson, MD
Currently Enrolling

PNOC016:  A Target Validation Study of Fimepinostat in Children and Young Adults with Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG), Recurrent Medulloblastoma, or Recurrent High-Grade Glioma (HGG)
U-M Site Principal Investigator: Andrea Franson, MD
Currently Enrolling

PNOC013  A Safety and Pharmacokinetic Study of Single Agent REGN2810 in Pediatric Patients with Relapsed or Refractory Solid or Central Nervous System (CNS) Tumors and a Safety and Efficacy Trial of REGN2810
U-M Site Principal Investigator: Andrea Franson, MD
Currently Enrolling

COG Phase I Trials for Brain Tumor Patients 

ADVL1823: Larotrectinib (LOXO-101, NSC# 788607, IND# 141824) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias
U-M Site Principal Investigator: Rajen Mody, MD, MS
Currently Enrolling

ADVL1921: Phase 1 study to evaluate the safety and pharmacokinetics of Palbociclib Ibrance® in combination with Irinotecan and Temozolomide in pediatric patients with recurrent or refractory solid tumors
U-M Site Principal Investigator: Rajen Mody, MD, MS
Currently Enrolling

LOXO-195: A Phase 1/ 2 Study of the TRK Inhibitor LOXO-195 in Adult and Pediatric Subjects with Previously Treated NTRK Fusion Cancers 
U-M Site Principal Investigator: Rajen Mody, MD, MS
Currently Enrolling

ADVL1711: A Phase 1/2 Study of Lenvatinib in Combination with Everolimus in Recurrent and Refractory Pediatric Solid Tumors, including CNS Tumors
U-M Site Principal Investigator: Rajen Mody, MD, MS
Currently Enrolling

COG Phase II-III Trials for Brain Tumor Patients

ALTE07C1 - Neuropsychological, Social, Emotional and Behavioral Outcomes in Children with Cancer
U-M Site Principal Investigator: Patricia Robertson, MD
Currently Enrolling

ACNS1422 - A Phase II Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients
U-M Site Principal Investigator: Andrea Franson, MD
Currently Enrolling

ACNS1721 - 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
U-M Site Principal Investigator: Carl Koschmann, MD
Currently Enrolling

ACNS1723 - 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)
U-M Site Principal Investigator: Andrea Franson, MD
Currently Enrolling

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)
U-M Site Principal Investigator: Andrea Franson, MD
Currently Enrolling

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)
Currently Enrolling
PI: Andrea Franson, MD

ACNS1931: “A Phase 3 Study of Selumetinib (NSC# 748727, IND# 77782) or Selumetinib in Combination with Vinblastine for non-NF1, non-TSC Patients with Recurrent or Progressive Low-Grade Gliomas (LGGs) Lacking BRAFV600E or IDH1 Mutations”
Enrollment at U-M expected Summer 2021
PI: Andrea Franson, MD