This is an open-label, comprehensive, iterative investigation of evaluating the use of induction chemotherapy, high-dose chemotherapy, and focal radiation therapy in children with newly diagnosed Embryonal Tumor With Multilayered Rosettes (ETMR).
PRIMARY OBJECTIVES I. To determine the six-month progression-free survival (PFS6) of participants with newly diagnosed, gross-totally resected, non-metastatic ETMR, treated using a regimen of induction chemotherapy and early focal radiotherapy (Cohort 1) SECONDARY OBJECTIVES I. To determine the two-year progression-free survival (PFS) and overall survival (OS) of participants with newly diagnosed, gross-totally resected, non-metastatic ETMR (Cohort 1). II. To determine the two-year progression-free survival (PFS) and overall survival (OS) of participants with newly diagnosed, gross-totally resected, non-metastatic ETMR (Cohort 2). III. To determine the two-year progression-free survival (PFS), overall survival (OS) and objective response rate of participants with newly diagnosed, incompletely resected and/or metastatic ETMR (Cohort 3A and 3B) EXPLORATORY OBJECTIVES: I. To validate the utility of a liquid miRNA biomarker in blood and Cerebral spinal fluid (CSF) as a correlative marker of a participant's disease status. II. To better define the genomic landscape of ETMR. OUTLINE: Participants with newly diagnosed ETMR will obtain either gross total, or sub-total resection surgery prior to enrollment. After surgery, participants will be assigned to 1 of 4 possible cohorts: Cohorts 1 and 2: Participants with newly diagnosed, gross-totally resected, non-metastatic ETMR. Cohorts 3A and 3B: Participants with newly diagnosed, incompletely resected and/or metastatic ETMR. Participants will be assessed for survival outcomes for up to 2 years. Follow-up procedures are to be captured under the PNOC COMP protocol. Participants will be followed under the Pediatric Neuro-Oncology Consortium (PNOC) COMP protocol until death or withdrawal from study.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
70
Undergo RT
One or more of the following may be assigned by the physician (physician's choice) per standard of care guidelines upon study enrollment following surgery: Cytarabine, Carboplatin, Cisplatin, Vincristine Sulfate injection (Vincristine PFS), Topotecan Hydrochloride, Dactinomycin, Thiotepa, Filgrastim, Cyclophosphamide, or Doxorubicin Hydrochloride. Not all participants will receive all possible drug regimens.
Undergo surgery directly before study enrollment as part of planned care.
University of Alabama at Birmingham
Birmingham, Alabama, United States
RECRUITINGUniversity of California, San Francisco
San Francisco, California, United States
RECRUITINGJohns Hopkins University
Baltimore, Maryland, United States
RECRUITINGMedian Progression-free survival at 6 months (PFS6) (Cohort 1)
The median PFS6 is defined as the median number of months for participants in Cohort 1 who have remained progression-free from the date of initial surgical resection until 6 months.
Time frame: Up to 6 months
Median Progression-free survival at 2 years progression-free survival (PFS)
The median PFS6 is defined as the median number of months for participants by cohort who have remained progression-free from the date of initial surgical resection until 2 years post-surgery.
Time frame: Up to 2 years
Median Overall Survival at 2 years (OS)
The median OS is defined as the median number of months for participants by cohort who are still alive from the date of initial surgical resection until 2 years post-surgery.
Time frame: Up to 2 years
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Participants assigned to or whom receive optional RT will receive concurrent temozolomide
Tumor tissue will be collected for correlative studies
Blood samples will be collected for correlative studies
CSF samples will be collected for correlative studies
Washington University in St. Louis
St Louis, Missouri, United States
RECRUITING