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
Pending
Non-U-M Initiated Clinical Trials Available at U-M for Brain Tumor Patients
Principal Investigator: Andrea Franson, MD
Sponsor: SpringWorks Therapeutics
Closed to Accrual
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
SJ901: SJ901: Study of Mirdametinib as a Treatment for Children, Adolescents and Young Adults with Low-Grade Glioma
UM Site Principal Investigator: Andrea Franson, MD
Pending
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: Chimerix
Closed to Accrual
PNOC Trials
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
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
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
UM Site Principal investigator: Rajen Mody, MD
Currently Enrolling
PEPN2121: Tiragolumab and Atezolizumab for the Treatment of Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
UM Site Principal investigator: Rajen Mody, MD
Currently Enrolling
COG Phase II-III Trials for Brain Tumor Patients
ACNS2031: A Study of Treatment for Medulloblastoma Using Sodium Thiosulfate to Reduce Hearing Loss
UM Site Principal Investigator: Andrea Franson, MDCurrently Enrolling
ACCL2031: A Study to See if Memantine Protects the Brain During Radiation Therapy Treatment for Primary Central Nervous System TumorsUM Site Principal Investigator: Andrea Franson, 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
Closed to Accrual
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
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
