Trametinib for Pediatric Neuro-oncology Patients With Refractory Tumor and Activation of the MAPK/ERK Pathway.
Status:
Recruiting
Trial end date:
2026-12-01
Target enrollment:
Participant gender:
Summary
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 tumor development, progression
and resistance to treatment. Furthermore, this study will also explore important aspects for
patients with brain tumors by including assessment of quality of life and neuropsychological
evaluation.
Phase:
Phase 2
Details
Lead Sponsor:
St. Justine's Hospital
Collaborators:
CHU de Quebec-Universite Laval Montreal Children's Hospital of the MUHC