Pediatric Brain Tumor Clinical Trials

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 [email protected] 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

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

A Phase 1 Study of a Combined Cytotoxic and Immune-stimulatory Therapy in Pediatric Patients with Recurrent, Primary Malignant Brain Tumors
Principal Investigator: Andrea Franson, MD
Currently Enrolling

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

A Study Comparing Abemaciclib Plus Temozolomide to Temozolomide Monotherapy in Children and Young Adults With High-grade Glioma Following Radiotherapy
UM Site Principal Investigator: Andrea Franson, MD
Currently Enrolling

CAR T Cells After Lymphodepletion for the Treatment of IL13Rα2 Positive Recurrent or Refractory Brain Tumors in Children
Sponsor: City of Hope Medical Center

A Study Comparing Two Carboplatin Containing Regimens for Children and Young Adults With Previously Untreated Low Grade Glioma
Host Site: Lurie Children’s Hospital
UM Site Principal Investigator: Carl Koschmann, M.D.
Currently Enrolling

DAY101 vs. Standard of Care Chemotherapy in Pediatric Patients With Low-Grade Glioma Requiring First-Line Systemic Therapy (LOGGIC/FIREFLY-2)
UM Site Principal Investigator: Andrea Franson, MD
Currently Enrolling

ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)
UM Site Principal Investigator: Carl Koschmann, MD
Currently Enrolling

SJIMB21: Molecular and Clinical Risk-Directed Therapy for Infants and Young Children With Newly Diagnosed Medulloblastoma
UM Site Principal Investigator: Santhosh Upadhyaya, MD
Currently Enrolling

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

PNOC Trials

PNOC030: A Registry Study for Children with Atypical Teratoid Rhabdoid Tumor (ATRT)
UM Site Principal Investigator: Andrea Franson, MD
Currently Enrolling

PNOC029: Nivolumab and DAY101 for the treatment of newly diagnosed or recurrent craniopharyngioma in children and young adults
UM Site Principal Investigator: Andrea Franson, MD
Pending

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
Currently Enrolling

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
U-M Site Principal Investigator: 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
Closed to Accrual

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
Closed to Accrual

COG Phase I Trials for Brain Tumor Patients 

COG Phase II-III Trials for Brain Tumor Patients

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
Closed to Accrual

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

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