{"entity":{"id":"acns0333","kind":"trial","name":"ACNS0333","aka":["COG ACNS0333"],"tldr":"ACNS0333 was the first trial built only for atypical teratoid/rhabdoid tumour, a brain cancer of very young children that used to be almost always fatal; its intensive plan of chemotherapy, high-dose chemotherapy with stem cell rescue and focused radiotherapy more than halved the risk of relapse or death compared with earlier treatment and became the template for care.","summary":"ACNS0333 was the Children's Oncology Group's first trial specific to atypical teratoid/rhabdoid tumour. After surgery, 70 patients received two courses of multi-agent induction chemotherapy including high-dose methotrexate, then three courses of high-dose carboplatin and thiotepa with peripheral blood stem cell rescue, with involved-field three-dimensional conformal radiotherapy given before or after consolidation according to age and disease location. SMARCB1 testing of tumour and blood was mandatory. The primary analysis compared event-free survival in patients under 36 months with a historical cooperative group cohort.\n\nThe regimen cut the hazard of an event-free survival event by more than half in children under 36 months (hazard rate 0.43), and four-year event-free and overall survival for the whole cohort were 37 and 43 percent, far above historical figures; 91 percent of relapses occurred within two years and four patients died of treatment. The corpus's atypical teratoid/rhabdoid tumour page describes ACNS0333-style induction, high-dose chemotherapy with autologous rescue and focal radiotherapy on this evidence.","status":"positive","asOf":"2026-09-22","links":[{"label":"ClinicalTrials.gov NCT00653068","url":"https://clinicaltrials.gov/study/NCT00653068"}],"tags":["soc-trials"],"related":[],"cancers":["atrt","brain-tumours","childhood-cancers"],"sections":[],"technologies":["cytotoxic-chemotherapy","autologous-stem-cell-transplant","imrt-igrt"],"targets":[],"drugs":["carboplatin","cisplatin","cyclophosphamide","etoposide","thiotepa","methotrexate","vincristine"],"companies":[],"institutions":["childrens-oncology-group"],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-acns0333-atrt-high-dose-chemotherapy-reddy-jco-2020"],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT00653068","phase":"3","setting":"Newly diagnosed atypical teratoid/rhabdoid tumour of the central nervous system from birth to 22 years: surgery, two courses of induction chemotherapy, three courses of high-dose chemotherapy with peripheral blood stem cell rescue and involved-field conformal radiotherapy timed by age and disease extent","sponsor":"Children's Oncology Group","result":"Hazard of an event 0.43 compared with the historical cohort in children under 36 months (p < 0.0005); four-year event-free survival 37 percent and overall survival 43 percent for all 65 evaluable patients.","yearReported":2020,"enrolled":70,"enrolledBasis":"registry","outcomes":[{"endpoint":"Event-free survival, patients under 36 months against a historical cooperative group cohort","primary":true,"arms":[{"name":"ACNS0333 regimen (induction, high-dose chemotherapy with stem cell rescue, conformal radiotherapy)","n":54},{"name":"Historical cohort"}],"hr":0.43,"ci":[0.28,0.66],"p":"<0.0005","source":"https://doi.org/10.1200/JCO.19.01776"},{"endpoint":"Event-free survival at 4 years, entire cohort","unit":"%","arms":[{"name":"ACNS0333 regimen","n":65,"value":37,"note":"95% CI 25 to 49"}],"source":"https://doi.org/10.1200/JCO.19.01776"},{"endpoint":"Overall survival at 4 years, entire cohort","unit":"%","arms":[{"name":"ACNS0333 regimen","n":65,"value":43,"note":"95% CI 31 to 55"}],"source":"https://doi.org/10.1200/JCO.19.01776"},{"endpoint":"Treatment-related deaths","unit":"participants","arms":[{"name":"ACNS0333 regimen","n":65,"value":4}],"source":"https://doi.org/10.1200/JCO.19.01776"}]},"route":"/trials/acns0333/","neighbours":{"cancer":[{"id":"atrt","kind":"cancer","name":"Atypical teratoid/rhabdoid tumour (ATRT)","route":"/cancers/atrt/"},{"id":"brain-tumours","kind":"cancer","name":"Brain and spinal cord tumours (all types)","route":"/cancers/brain-tumours/"},{"id":"childhood-cancers","kind":"cancer","name":"Childhood cancers (all types)","route":"/cancers/childhood-cancers/"}],"technology":[{"id":"autologous-stem-cell-transplant","kind":"technology","name":"Autologous stem cell transplant (high-dose therapy)","route":"/technologies/autologous-stem-cell-transplant/"},{"id":"cytotoxic-chemotherapy","kind":"technology","name":"Cytotoxic chemotherapy","route":"/technologies/cytotoxic-chemotherapy/"},{"id":"imrt-igrt","kind":"technology","name":"IMRT / IGRT (modern external beam)","route":"/technologies/imrt-igrt/"}],"drug":[{"id":"carboplatin","kind":"drug","name":"Carboplatin","route":"/drugs/carboplatin/"},{"id":"cisplatin","kind":"drug","name":"Cisplatin","route":"/drugs/cisplatin/"},{"id":"cyclophosphamide","kind":"drug","name":"Cyclophosphamide","route":"/drugs/cyclophosphamide/"},{"id":"etoposide","kind":"drug","name":"Etoposide","route":"/drugs/etoposide/"},{"id":"methotrexate","kind":"drug","name":"Methotrexate","route":"/drugs/methotrexate/"},{"id":"thiotepa","kind":"drug","name":"Thiotepa","route":"/drugs/thiotepa/"},{"id":"vincristine","kind":"drug","name":"Vincristine","route":"/drugs/vincristine/"}],"institution":[{"id":"childrens-oncology-group","kind":"institution","name":"Children's Oncology Group (COG)","route":"/institutions/childrens-oncology-group/"}],"paper":[{"id":"paper-acns0333-atrt-high-dose-chemotherapy-reddy-jco-2020","kind":"paper","name":"ACNS0333: high-dose chemotherapy and three-dimensional conformal radiation for atypical teratoid/rhabdoid tumour","route":"/key-papers/paper-acns0333-atrt-high-dose-chemotherapy-reddy-jco-2020/"}]}}