Phase III Trial of Metformin for Improving Brain Growth and Cognitive Function in Children and Adolescents After Brain Tumour Treatment

What this Trial Is About

Researchers are evaluating whether metformin can help improve brain growth and cognitive function in children and adolescents who have completed treatment for brain tumors. This Phase III multi-site randomized trial compares 16 weeks of oral metformin treatment to a placebo in participants aged 7 to 21 years and 11 months. The study focuses on survivors of medulloblastoma and other brain tumors treated with cranial radiation, aiming to address cognitive impairments caused by aggressive cancer therapies. Participants will be randomly assigned to receive either metformin or a matching placebo. The dosing starts at approximately 500mgm2day for one week and increases to 1000mgm2day for the following 15 weeks, with doses adjusted in half-tablet increments. The trial includes baseline assessments, a 16-week treatment period, and follow-up evaluations at week 17 and again at 24 weeks post-treatment to monitor cognitive and brain changes. During the study, participants will undergo cognitive testing measuring memory, working memory, processing speed, attention, and executive function. Brain imaging with diffusion MRI will assess white matter growth, especially in the corpus callosum and hippocampus. Parent or guardian reports on global health will also be collected. Safety, adherence, and long-term effects will be monitored throughout the trial, which may last up to 41 weeks from baseline to final follow-up.

Effect of Metformin on Behaviour and the Brain in Children Treated for a Brain Tumour