ATRT01
Research type
Research Study
Full title
An international prospective umbrella trial for children with atypical teratoid/rhabdoid tumours (ATRT) including A randomized phase III study evaluating the non-inferiority of three courses of high-dose chemotherapy (HDCT) compared to focal radiotherapy as consolidation therapy
IRAS ID
1006081
Contact name
Patricia O'Hare
Contact email
Sponsor organisation
German Paediatric Oncology Group
Eudract number
2018-003335-29
Clinicaltrials.gov Identifier
German Clinical Trials Register, DRKS00023783
Research summary
Atypical teratoid/rhabdoid tumour (ATRT) is a type of rare brain or spinal cord cancer that occurs in young children, though it can occur in older children, teenagers, and adults. Treatment for ATRT is intensive and includes surgery, chemotherapy, radiotherapy, and sometimes stem cell transplantation. Although the outlook for patients with ATRT has improved over time, further improvements need to be made. One of the major issues faced by patients is that current treatments have long-term side effects of on brain function and development.
The aim of the ATRT01 trial is to compare different treatments to find out which is most effective, and which has fewer effects on patients' brain function and development.
This international study has three parts, which will recruit over 5 years:
• PART A: For children aged between 12 and 35 months old, to find out if treatment with high-dose chemotherapy and stem cell transplantation is as effective as radiotherapy with standard dose chemotherapy. We hope that the long-term effects of high-dose chemotherapy will be less than radiotherapy overall and will not have the negative effects on brain development that radiotherapy may have.
• PART B: In very young patients (aged less than 12 months), to find out how high-dose chemotherapy affects outcomes, including survival and brain function.
• PART C: In older children (aged 3 years and over), to find out how standard dose chemotherapy with radiotherapy affects outcomes, including survival and brain function.
We aim to recruit 53 patients into the study as a whole in the UK, across 17 hospitals. All patients will be carefully monitored during treatment using the same routine tests that would be used if they were not in the trial and will be followed up for up to a maximum of five years. Outcomes of this trial will help doctors give the best treatments to future patients with ATRT and better understand the long term effects of treatment on the developing brain.
REC name
London - Riverside Research Ethics Committee
REC reference
25/LO/0088
Date of REC Opinion
25 Mar 2025
REC opinion
Further Information Favourable Opinion