This pilot early phase I trial studies how well surgery and combination chemotherapy work in treating younger patients with a brain tumor called medulloblastoma that has not spread to another place in the body. Drugs used in chemotherapy, such as cisplatin, lomustine, vincristine sulfate, and cyclophosphamide, 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. Giving more than one drug (combination chemotherapy) after surgery may kill any remaining tumor cells.
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT02212574.
PRIMARY OBJECTIVES:
I. To determine the occurrence of relapse, progression, or death due to disease in the first two years after study enrollment of newly diagnosed children with non-metastatic, standard risk, Wnt positive medulloblastoma with a chemotherapy-only approach.
SECONDARY OBJECTIVES:
I. To describe overall survival of children with newly diagnosed non-metastatic, standard risk, Wnt positive medulloblastoma who are treated with a maximal surgical resection and chemotherapy-only approach.
II. To determine the pattern of failure in those children with progressive disease.
III. To correlate the frequency of nuclear beta (b)-catenin with other measures of Wnt-positivity.
OUTLINE:
Patients undergo surgery. Within 30 days, patients also receive Regimen A and Regimen B chemotherapy.
REGIMEN A: Patients receive cisplatin intravenously (IV) over 6 hours on day 1, lomustine orally (PO) on day 1, and vincristine sulfate IV on days 1, 8, and 15 during weeks 1 (course 1), 7 (course 2), 17 (course 4), 23 (course 5), 33 (course 7), and 39 (course 8). Courses repeat every 42 days in the absence of disease progression or unacceptable.
REGIMEN B: Patients receive cyclophosphamide IV over 1 hour on days 1 and 2 and vincristine sulfate IV on days 1 and 8 during weeks 13 (course 3), 29 (course 6), and 45 (course 9). Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at 3, 6, and 9 months; 1, 1.5, 2, 2.5, and 3 years; and then annually for 2 years.
Lead OrganizationJohns Hopkins University/Sidney Kimmel Cancer Center
Principal InvestigatorKenneth J. Cohen