Enhancing Brain Repair with Metformin (Metformin in CP)

Looking back:
our work in Phase 1

Our team continues to study whether metformin, a drug commonly used to treat diabetes, can be used to enhance motor and cognitive skills in children with cerebral palsy (CP).

Over the course of our study, we recruited three participants and formed a Stakeholder Engagement Committee (SEC) consisting of five parent-partners and individuals with lived experience with CP. The SEC has been instrumental in ensuring our project is patient oriented by helping us communicate research terms and procedures in lay language, communicating with families, and helping us modify our study methods and procedures to make them less burdensome and more meaningful to families.

However, our progress in determining the effectiveness of using metformin as an intervention for children with CP, and our progress in determining if it is feasible to do the study has been hampered by slow recruitment, despite many families’ interest in participating. We were proactive in finding potential solutions to this ongoing challenge: we changed the criteria and made some of our research activities virtual to help minimize burden on families. Despite these changes, recruitment continued to be unsuccessful. In October 2021, we presented an update to CHILD-BRIGHT’s Data Safety Monitoring Board (DSMB), a group of researchers and clinicians that reviews the safety, data integrity, progress, and efficacy of our trial. The DSMB concluded that we would not be able to recruit enough participants before the end of Phase 1, and recommended the trial be stopped or significantly redesigned.

We then began working with our SEC to redesign our study. Our goal was to design a study with broader inclusion criteria and other changes that would facilitate recruitment, but still yield preliminary clues about whether metformin can enhance motor and cognitive skills in children with CP. 

In February 2022, our redesigned study was approved by our SEC, CHILD-BRIGHT leadership, and the DSMB. Our modified protocol has also been approved by Health Canada and the REB at Holland Bloorview and is almost approved by the REB at SickKids. We will soon begin recruiting using our modified protocol.

An eye to the future: what Phase 2 has in store for us

Should our study redesign be approved by the research ethics boards, we hope to have 10 participants between the ages of 5 and 18 complete the trial. Once data collection is complete, we will conduct data analysis to answer our study questions about the feasibility, safety, and effectiveness of metformin in improving motor functioning in children and adolescents with CP. We hope to continue to work with our SEC to ensure that our trial is patient oriented in nature, to also help us conduct knowledge translation in a way that is meaningful, relatable and understandable by the CP community and the community at large.