# TADPOLE (CDRB436G2201)

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

## TL;DR

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.

## Summary

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.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-10
- Tags: nci-coverage; paediatric; cns
- Registry id: NCT02684058
- Phase: 2
- Setting: Newly diagnosed BRAF V600-mutant paediatric low-grade glioma needing systemic therapy: dabrafenib + trametinib vs carboplatin + vincristine (2:1 randomisation)
- Sponsor: Novartis
- Enrolled: 110
- Result: ORR 47% vs 11%; median PFS 20.1 vs 7.4 months, HR 0.31.
- Outcomes: Overall response rate (independent review, RANO): Dabrafenib + trametinib 47% vs Carboplatin + vincristine 11%; Progression-free survival: Dabrafenib + trametinib 20.1 months vs Carboplatin + vincristine 7.4 months, HR 0.31
- Replication: Consistent with earlier single-arm paediatric dabrafenib and trametinib studies; the only randomised comparison so far. FIREFLY-1 addressed the BRAF-fusion population that this trial excluded.

## Sources

- ClinicalTrials.gov NCT02684058: https://clinicaltrials.gov/study/NCT02684058
- Bouffet et al., NEJM 2023: https://doi.org/10.1056/NEJMoa2303815

## Connected records

- cancers: [Paediatric low-grade glioma](https://onco.cc/cancers/paediatric-low-grade-glioma/)
- technologies: [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- targets: [BRAF](https://onco.cc/targets/braf/)
- drugs: [Carboplatin](https://onco.cc/drugs/carboplatin/), [Dabrafenib + trametinib](https://onco.cc/drugs/dabrafenib-trametinib/), [Vincristine](https://onco.cc/drugs/vincristine/)
- companies: [Novartis](https://onco.cc/companies/novartis/)
- pathways: [RAS / RAF / MEK / ERK (MAPK)](https://onco.cc/pathways/ras-mapk/)
- terms: [BRAF V600E mutation](https://onco.cc/terms/braf-v600-mutation/), [RACE for Children Act](https://onco.cc/terms/race-for-children-act/)
- key papers: [Dabrafenib plus Trametinib in Pediatric Glioma with BRAF V600 Mutations](https://onco.cc/key-papers/paper-bouffet-n-engl-j-med/)
- roadmaps: [Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first](https://onco.cc/roadmaps/paediatric-oncology-roadmap/)

---
JSON: https://onco.cc/api/v1/entities/tadpole.json