SJiMB21: Phase 2 Study of Molecular and Clinical Risk-Directed Therapy for Infants and Young Children with Newly Diagnosed Medulloblastoma

Medulloblastoma in children is a fast-growing tumour.  This tumour can spread into the fluid that surrounds the brain and spinal cord (cerebrospinal fluid or CSF). Therapy for medulloblastoma in infants (0 to 2.99 years) and young children (2.99-4.99 years) includes surgery, chemotherapy, and radiation therapy to the brain and spine.

In this study, a new method for diagnosing specific molecular groups and subgroups of Medulloblastoma, based on biological features of the tumour, will be used. The method is called “methylation”, which detects molecular patterns (marks) on DNA of the cells from the patient’s tumour. Different groups and subgroups of medulloblastoma will be identified based on their unique molecular patterns.

Treatment will then be guided based on the tumour group and subgroup.

1. If the tumour belongs to the sonic hedgehog (SHH) group, patient’s will be treated with just chemotherapy.
2. If the tumour belongs to G3/G4 or non-WNT/non-SHH (NWNS) group, patients will be pre-treated with chemotherapy until they are 3 years or 36 months old and then treated with radiation. Chemotherapy will be used to “delay radiation” until the patient is 36 months of age (to allow for brain development). This treatment is based on the information from prior studies, done at St. Jude Children’s Research Hospital (Memphis, Tennessee, USA) and other national and international children’s hospital. The research showed that treatment of G3/G4 or NWNS medulloblastoma required radiation. This molecular group of medulloblastoma cannot be cured with just surgery and chemotherapy.

Overall, the purpose of this study is to determine whether treating infants and young children according to their “molecular group/sub-groups” will improve their survival outcomes for medulloblastoma.  We also want to see how the treatments may affect a patient’s neurocognitive or thinking processes following treatment.

Disease Stage – Newly Diagnosed

Patient Age Range – 0 to 4.99 years

Trial sponsors

• National – ANZCHOG
• International – St Jude Children’s Research Hospital