Trametinib for Pediatric Neuro-oncology Patients With Refractory Tumour and Activation of the MAPK/ERK Pathway.

Official Title

A Phase 2 Study of Trametinib for Patients With Pediatric Glioma or Plexiform Neurofibroma With Refractory Tumour and Activation of the MAPK/ERK Pathway.


This is a phase 2, open-label, interventional clinical trial that will study the response rate of pediatric glioma and plexiform neurofibroma (PN) to oral administration of trametinib. Patients meeting all inclusion criteria for a given study group will receive the study medication at a daily dose of 0.025 mg/kg up to a total of 18 cycles, in 28-day cycles. A total of 150 patients will be recruited as part of this clinical study. Patients aged between 1 month (corrected age) and 25 years old will be eligible, in order to include a maximum of patients affected by low-grade glioma (LGG) and PN. This study includes four groups: patients with neurofibromatosis type 1 (NF1) and LGG, NF1 patients with PN, patients with LGG with a B-Raf Serine/Threonine-protein Kinase/Proto-oncogene Encoding B-Raf (BRAF) fusion and patients with glioma of any grade with activation of the Mitogen-activated Protein Kinase/Extracellular Signal-regulated Kinases (MAPK/ERK) pathway. All patients except patients with PN must have failed at least one line of treatment. The study will also explore the molecular mechanisms behind tumour development, progression and resistance to treatment. Furthermore, this study will also explore important aspects for patients with brain tumours by including assessment of quality of life and neuropsychological evaluation.

Trial Description

Primary Outcome:

  • Objective Response Rate
Secondary Outcome:
  • Time to Progression
  • Progression Free Survival
  • Overall Survival
  • Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability).
  • Determination of the Serum Level of Trametinib.
  • Evaluation of the Quality of Life During Treatment.

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Canadian Cancer Society

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