Study of Metformin Treatment for Children Aged 2 to 16 With Fragile X Syndrome Evaluating Safety and Effects on Behavior, Cognition, and Language

What this Trial Is About

Researchers are evaluating the use of metformin in children aged 2 to 16 years with Fragile X Syndrome FXS, a genetic condition. This randomized, double-blind, placebo-controlled trial aims to study metformins effects on behavior problems, cognition, and language in children with FXS. The study is conducted at the Childrens Hospital of Fudan University and is motivated by previous findings that metformin may influence pathways involved in FXS symptoms. Participants will be randomly assigned to receive either metformin or a placebo for a treatment period of 6 months. Metformin dosing starts at 50mg daily and is gradually increased to a maximum dose of 1 to 2 grams per day based on patient weight. The placebo group will receive starch tablets matching the metformin in appearance and dosage schedule. After the 6-month intervention, participants will have a follow-up visit at 1 year to monitor ongoing effects and safety. During the study, children will undergo regular assessments including behavioral evaluations using the Aberrant Behavior Checklist ABC to measure changes from baseline to 6 months. Researchers will monitor side effects and overall safety throughout the trial. The total participation duration includes the 6-month treatment and the follow-up visit at 1 year to evaluate longer-term outcomes.

Metformin in Children With Fragile X Syndrome