Study of Efficacy and Safety of Dabrafenib in Combination With Trametinib in Pediatric Patients With BRAF V600 Mutation Positive LGG or Relapsed or Refractory HGG Tumors

Summary

The purpose of this study was to investigate the activity of dabrafenib in combination with trametinib in children and adolescent patients with BRAF V600 mutation positive low grade glioma (LGG) or relapsed or refractory high grade glioma (HGG)

Timeline

Start

2017-12-28

Primary completion

2021-08-23

Completion

2023-04-28

Publications

• Gascon B, Gauvreau C, Yang A, Bennett J, Nobre L, Tabori U, Hawkins C, Tsang D, Pechlivanoglou P, Denburg A. Cost-Effectiveness of Dabrafenib Plus Trametinib Versus Standard Chemotherapy in BRAFV600E-Mutant Pediatric Low-Grade Glioma. JCO Oncol Pract. 2026 Feb 25:OP2501263. doi: 10.1200/OP-25-01263. Online ahead of print.
• Bouffet E, Hansford JR, Garre ML, Hara J, Plant-Fox A, Aerts I, Locatelli F, van der Lugt J, Papusha L, Sahm F, Tabori U, Cohen KJ, Packer RJ, Witt O, Sandalic L, Bento Pereira da Silva A, Russo M, Hargrave DR. Dabrafenib plus Trametinib in Pediatric Glioma with BRAF V600 Mutations. N Engl J Med. 2023 Sep 21;389(12):1108-1120. doi: 10.1056/NEJMoa2303815.
• Hargrave DR, Terashima K, Hara J, Kordes UR, Upadhyaya SA, Sahm F, Bouffet E, Packer RJ, Witt O, Sandalic L, Kieloch A, Russo M, Cohen KJ; Investigators involved in the high-grade glioma cohort. Phase II Trial of Dabrafenib Plus Trametinib in Relapsed/Refractory BRAF V600-Mutant Pediatric High-Grade Glioma. J Clin Oncol. 2023 Nov 20;41(33):5174-5183. doi: 10.1200/JCO.23.00558. Epub 2023 Aug 29.
• Barbato MI, Nashed J, Bradford D, Ren Y, Khasar S, Miller CP, Zolnik BS, Zhao H, Li Y, Bi Y, Shord SS, Amatya AK, Mishra-Kalyani PS, Scepura B, Al-Matari RA, Pazdur R, Kluetz PG, Donoghue M, Singh H, Drezner N. FDA Approval Summary: Dabrafenib in Combination with Trametinib for BRAFV600E Mutation-Positive Low-Grade Glioma. Clin Cancer Res. 2024 Jan 17;30(2):263-268. doi: 10.1158/1078-0432.CCR-23-1503.
• Shahid S, Kushner BH, Modak S, Basu EM, Rubin EM, Gundem G, Papaemmanuil E, Roberts SS. Association of BRAF V600E mutations with vasoactive intestinal peptide syndrome in MYCN-amplified neuroblastoma. Pediatr Blood Cancer. 2021 Oct;68(10):e29265. doi: 10.1002/pbc.29265. Epub 2021 Jul 31.

Drugs

Indications