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The first randomised trial to show that a targeted drug pair beats chemotherapy in children with a brain tumour. Children whose low-grade glioma carries a BRAF V600 mutation had far more tumour shrinkage and a much longer time before progression on dabrafenib plus trametinib than on standard carboplatin and vincristine.
Phase 2 randomised trial (Bouffet et al., NEJM 2023). 110 patients randomised (73 to dabrafenib plus trametinib, 37 to carboplatin plus vincristine). At a median follow-up of 18.9 months the independently assessed overall response rate was 47% versus 11% (risk ratio 4.31; 95% CI 1.7 to 11.2), clinical benefit 86% versus 46%, and median progression-free survival 20.1 versus 7.4 months (HR 0.31; 95% CI 0.17 to 0.55). Grade 3 or higher adverse events were less frequent with the targeted pair (47% versus 94%). Basis of the FDA approval of dabrafenib plus trametinib for paediatric BRAF V600E LGG in March 2023, with new liquid and dispersible formulations for young children. A separate cohort in relapsed BRAF V600 high-grade glioma also responded.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
110 randomised.
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Overall response rate (independent review, RANO)primary | Dabrafenib + trametinib | 73 | 47% | - | <0.001 | link |
| Carboplatin + vincristine | 37 | 11% | ||||
| Progression-free survival | Dabrafenib + trametinib | 73 | 20.1 months | 0.31 (0.17 to 0.55) | <0.001 | link |
| Carboplatin + vincristine | 37 | 7.4 months |
The full inclusion and exclusion criteria are on the registry record. Read 2026-10-07. Recruiting overall does not mean a particular site is open; ask the study team.
ClinicalTrials.gov data courtesy of the US National Library of Medicine.
Shares RACE for Children Act, Paediatric low-grade glioma, Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, BRAF and the tags paediatric, cns.
Shares RACE for Children Act, Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first and the tags paediatric, cns.
Shares Dabrafenib + trametinib, Paediatric low-grade glioma, BRAF, RAS / RAF / MEK / ERK (MAPK) and the tags paediatric, cns.
Shares Paediatric low-grade glioma, Vincristine, Carboplatin and the tags paediatric, cns.
Shares RACE for Children Act, Paediatric low-grade glioma, Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first and the tags paediatric, cns.
Shares Vincristine, Carboplatin and the tags paediatric, cns.
Shares Vincristine, Carboplatin and the tags paediatric, cns.
Shares Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first, Vincristine and the tags paediatric, cns.
Every source this page cites, grouped by who published it. Grouping is read from the web address, not assigned by hand.
A source that has moved or gone is reported by the weekly link check. /audit/