Active — Not RecruitingPhase 2

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

Canada114 participantsStarted 2018-08-16

Plain-language 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.

Who can participate

Age range1 Month – 25 Years

SexALL

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Inclusion criteria

✓. Signed written informed consent Prior to study participation, written informed consent from participants, or in the case of minors, written permission (informed consent) from parents, guardians, or legally acceptable representatives must be obtained according to local laws and regulations.
✓. Assent Assent from minor participants should be obtained per local laws and regulations and should be documented in accordance with local requirements.
✓. Study activities compliance. Participants must be willing and able to comply with scheduled visits, treatment schedule, laboratory testing, and other requirements of the study, including disease assessment by contrast-enhanced MRI.
✓. Age Patient must be aged ≥ 1 month (corrected age) to ≤ 25 years when starting trametinib.
✓. Study group Participants must belong to one of the following groups to be eligible. Group 1: NF1 with progressing/refractory LGG Group 2: NF1 with PN Group 3: Progressing/refractory LGG with KIAA 1549-BRAF fursion Group 4: Progressing/refractory glioma with activation of the MPAK/ERK pathway who do not meet criteria for other study groups
✓. Tumor Tissue Sample Tumor tissue will be required for all patients (minimally paraffin-embedded tissue block and additionally fresh frozen tissue \[if available\]). Patients with NF1 and LGG or PN can still be enrolled without tissue if no surgery or biopsy was conducted.

Exclusion criteria

What they're measuring

Objective Response Rate

Timeframe: From date of treatment start until the date of first documented progression, up to completion of treatment (504 treatment days).

Trial details

NCT IDNCT03363217

SponsorSt. Justine's Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-03-01

View on ClinicalTrials.gov More Low-grade Glioma trials