NCT02017964

Combination Chemotherapy in Treating Younger Patients With Newly Diagnosed, Non-metastatic Desmoplastic Medulloblastoma

A Phase II Study for the Treatment of Non-metastatic Nodular Desmoplastic Medulloblastoma in Children Less Than 4 Years of Age

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 26 (actual)
• Sponsor: Children's Oncology Group · Network
• Sex: All
• Age: 47 Months
• Healthy volunteers: Not accepted

Summary

This phase II trial studies how well combination chemotherapy works in treating younger patients with newly diagnosed, non-metastatic desmoplastic medulloblastoma. Drugs used in chemotherapy, such as vincristine sulfate, cyclophosphamide, methotrexate, etoposide, and carboplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Detailed description

PRIMARY OBJECTIVES: I. Estimate of the progression free survival (PFS) distribution for patients 0-\<4 years of age with M0 desmoplastic medulloblastoma (nodular desmoplastic or medulloblastoma with extensive nodularity) treated with the modified HIT SKK regimen (excluding the use of intraventricular methotrexate). OTHER PRE-SPECIFIED OBJECTIVES: I. Evaluate the feasibility of a rapid central pathology screening review for treatment allocation according to histology and assess agreement between institutional and central pathology review diagnoses as well as among central pathology review diagnoses. II. Prospectively evaluate the molecular profile of nodular desmoplastic (ND)/medulloblastoma with extensive nodularity (MBEN) medulloblastoma in young children. III. Monitor and describe the neurocognitive and adaptive functioning of young children with ND/MBEN medulloblastoma treated on this protocol using the ALTE07C1 protocol. OUTLINE: INDUCTION THERAPY: Patients receive vincristine sulfate intravenously (IV) over 1 minute or infused via minibag on days 1, 15, and 29; cyclophosphamide IV over 1 hour on days 1-3; methotrexate IV over 24 hours on days 15 and 29; etoposide IV over 60-120 minutes on days 43-45; and carboplatin IV over 1 hour on days 43-45. Treatment repeats every 63 days for 3 courses in the absence of disease progression or unacceptable toxicity. CONTINUATION THERAPY: Patients receive vincristine sulfate IV over 1 minute or infused via minibag on day 1, cyclophosphamide IV over 1 hour on days 1-3, etoposide IV over 60-120 minutes on days 21-23, and carboplatin IV over 1 hour on days 21-23. Treatment repeats every 42 days for 2 courses in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 3, 6, 9, 12, 16, 20, 24, 36, 48, 60, and 72 months.

Conditions

• Desmoplastic/Nodular Medulloblastoma
• Medulloblastoma
• Medulloblastoma With Extensive Nodularity
• Nevoid Basal Cell Carcinoma Syndrome

Interventions

Type	Name	Description
DRUG	Carboplatin	Given IV
OTHER	Cognitive Assessment	Optional ancillary studies
DRUG	Cyclophosphamide	Given IV
DRUG	Etoposide	Given IV
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Methotrexate	Given IV
DRUG	Vincristine Sulfate	Given IV

Timeline

• Start date: 2013-12-24
• Primary completion: 2018-03-31
• Completion: 2022-12-31
• First posted: 2013-12-23
• Last updated: 2023-01-09
• Results posted: 2019-04-04

Locations

173 sites across 4 countries: United States, Australia, Canada, Puerto Rico