Molecular and Clinical Risk-Directed Therapy for Infants and Young Children With Newly Diagnosed Medulloblastoma

Conditions

• Medulloblastoma

Eligibility Criteria

Sex

ALL

Age

N/A - 59 Months

Healthy Volunteers

Not accepted

Interventions

• Surgical resection — PROCEDURE
  All participants enrolled will undergo surgical resection prior to treatment. The maximal resection that can be achieved without undue risk to the patient will be attempted, with decisions about feasibility and extent of resection left to the discretion of the neurosurgeon. In instances where an STR is the best extent of resection achieved prior to start of therapy, a "second-look" surgery may be performed between cycles of chemotherapy after discussion with the principal investigator.
• Ommaya/VPS — PROCEDURE
  All participants enrolled on S-1 will undergo
• Methotrexate — DRUG
  Route of administration: Intravenously (IV)
• Cisplatin — DRUG
  Route of administration: Intravenously (IV)
• Vincristine — DRUG
  Route of administration: Intravenously (IV)
• Cyclophosphamide — DRUG
  Route of administration: Intravenously (IV)
• Carboplatin — DRUG
  Route of administration: Intravenously (IV)
• Topotecan — DRUG
  Route of administration: Intravenously (IV)
• Etoposide — DRUG
  Route of administration: Intravenously (IV)
• Pegfilgrastim — DRUG
  Route of administration: subcutaneous (SQ)
• Filgrastim — DRUG
  Route of administration: subcutaneous (SQ) or Intravenously (IV)
• Irradiation — RADIATION
  All participants in stratum N will undergo craniospinal irradiation (CSI) with boost to the primary tumor site once they reach 36 months of age. The dose given is based on the molecular risk group and disease response to chemotherapy as noted in the arm descriptions. The type of radiation used includes conformal radiation therapy (photons) or intensity modulated radiation therapy (IMRT) or proton beam therapy.
• Educational and Media Intervention — OTHER
  Participants watch a 75-minute caregiver education video program and receive access to interactive games on a smartphone or tablet throughout the optional cognitive study.
• SOC, Educational and Media Intervention — OTHER
  Standard-of-care (SOC) treatment during main cognitive study. After the one-year serial cognitive evaluation, participants will be offered participation in the cognitive study group I intervention.
• Methotrexate — DRUG
  Route of administration: Intravenously (IV) \& Intra Ventricular (IVT)

Study Details

This is a multi-center, multinational phase 2 trial that aims to explore the use of molecular and clinical risk-directed therapy in treatment of children 0-4.99 years of age with newly diagnosed medulloblastoma.

Key Dates

Start date

Dec 20, 2022

Status verified

Feb 2026

Primary completion

Jul 31, 2035

Completion

Jul 31, 2035

Study Design

Enrollment

130 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Stratum S-2
  Patients with Sonic Hedgehog subgroup 2 (SHH-2), 0-2.99 years, or M0 and 3-4.99 years, will receive systemic high-dose methotrexate (HD-MTX) and conventional chemotherapy. Interventions: Surgery, Methotrexate, Cisplatin, Vincristine, Cyclophosphamide, Carboplatin, Topotecan, Pegfilgrastim, Filgrastim
• Experimental: Stratum S-1
  Patients with SHH-1, SHH-3, SHH-4, or SHH-Not otherwise specified (NOS), 0-2.99 years, will receive intraventricular methotrexate (IVT-MTX) in parallel with systemic HD-MTX and conventional chemotherapy. Interventions: Surgery, Methotrexate, Cisplatin, Vincristine, Cyclophosphamide, Carboplatin, Topotecan, Pegfilgrastim, Filgrastim
• Experimental: Stratum N
  Patients with Medulloblastoma (MB) group 3 or group 4 (G3/G4) or MB \[including Non-WNT non-SHH medulloblastoma (NWNS) NOS or otherwise indeterminate cases\] (0-2.99 years) will receive systemic HD-MTX and conventional chemotherapy only for radiation delaying purposes. At 3 years of age, these patients will receive risk-stratified craniospinal irradiation (CSI). Interventions: Surgery, Methotrexate, Cisplatin, Vincristine, Cyclophosphamide, Carboplatin, Topotecan, Etoposide, Pegfilgrastim, Filgrastim, Radiation
• Experimental: Cognitive Study Group I (educational video and games)
  Educational video and games
• Active Comparator: Cognitive Study Group II (standard-of-care control)
  Standard-of-care (SOC) followed by educational video and games

Primary Outcome Measure

Progression free survival of SHH-2 infant (0-2.99 years) and young child (3-4.99 years) medulloblastoma patients treated with systemic HD-MTX-based chemotherapy only. [ Time Frame: Up to 7 years after enrollment with PFS estimation occurring 2 years after treatment initiation of last patient ]

Central Contacts

• Tabatha E. Doyle, RN
  901-595-2544
  [email protected]

Locations (10)

Find similar trials in Palo Alto, CA

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