About this study
Embryonal tumor with multilayered rosettes (ETMR) is a rare brain tumor that grows quickly. It is most often found in very young children. There is no widely accepted standard treatment for ETMR. In many children, ETMR starts growing again during treatment or soon after it ends. Researchers want to find better treatments and help children live longer.
This study is for people with newly diagnosed ETMR. Each person joins a group based on:
- How much tumor was removed
- Whether the tumor has spread
- Age
- Whether early radiation therapy is planned
Groups 1 and 3A get 12 weeks of chemotherapy and 6 weeks of radiation. Treatment lasts about 18 weeks.
Groups 2 and 3B get 6 weeks of chemotherapy. They then get 3 rounds of high-dose chemotherapy with stem cell rescue. Stem cell rescue gives back blood-making cells after chemotherapy. Radiation may be given later. Treatment lasts about 18 to 24 weeks.
Doctors will watch each person closely during treatment. Tests may include physical exams, blood and urine tests, MRI scans, heart and hearing tests, and tests of spinal fluid. Participants may also have tests that check memory, attention, and thinking.
Researchers will study:
- How long the tumor does not get worse and how long participants live
- Whether signs in blood and spinal fluid match changes in the tumor
- Changes in tumor genes and how those genes work
This is the first clinical trial focused specifically on ETMR. The study will test whether these treatment plans can lower the chance that ETMR comes back early and help people live longer. Researchers will also use blood, spinal fluid, and tumor samples to guide future research.
Eligibility overview
- Newly diagnosed embryonal tumor with multilayered rosettes (ETMR)
- Age 0–21 years
- Has had surgery or a biopsy for the tumor