# ACNS0333

Source: https://onco.cc/trials/acns0333/  
OnCo record `acns0333` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

The 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.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-22
- Also known as: COG ACNS0333
- Tags: soc-trials
- Registry id: 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
- Enrolled: 70
- 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.
- Outcomes: Event-free survival, patients under 36 months against a historical cooperative group cohort: ACNS0333 regimen (induction, high-dose chemotherapy with stem cell rescue, conformal radiotherapy) vs Historical cohort, HR 0.43; Event-free survival at 4 years, entire cohort: ACNS0333 regimen 37%; Overall survival at 4 years, entire cohort: ACNS0333 regimen 43%; Treatment-related deaths: ACNS0333 regimen 4 participants

## Sources

- ClinicalTrials.gov NCT00653068: https://clinicaltrials.gov/study/NCT00653068

## Connected records

- cancers: [Atypical teratoid/rhabdoid tumour (ATRT)](https://onco.cc/cancers/atrt/), [Brain and spinal cord tumours (all types)](https://onco.cc/cancers/brain-tumours/), [Childhood cancers (all types)](https://onco.cc/cancers/childhood-cancers/)
- technologies: [Autologous stem cell transplant (high-dose therapy)](https://onco.cc/technologies/autologous-stem-cell-transplant/), [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/), [IMRT / IGRT (modern external beam)](https://onco.cc/technologies/imrt-igrt/)
- drugs: [Carboplatin](https://onco.cc/drugs/carboplatin/), [Cisplatin](https://onco.cc/drugs/cisplatin/), [Cyclophosphamide](https://onco.cc/drugs/cyclophosphamide/), [Etoposide](https://onco.cc/drugs/etoposide/), [Methotrexate](https://onco.cc/drugs/methotrexate/), [Thiotepa](https://onco.cc/drugs/thiotepa/), [Vincristine](https://onco.cc/drugs/vincristine/)
- institutions: [Children's Oncology Group (COG)](https://onco.cc/institutions/childrens-oncology-group/)
- key papers: [ACNS0333: high-dose chemotherapy and three-dimensional conformal radiation for atypical teratoid/rhabdoid tumour](https://onco.cc/key-papers/paper-acns0333-atrt-high-dose-chemotherapy-reddy-jco-2020/)

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