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Clinical Trial Summary

This study is a controlled trial of metformin in children with fragile X syndrome(FXS). The age of FXS children range from 2 to 16 years old. Participants will be randomized in a double-blind design to either drug or placebo for 6-month period. The primary objectives are to assess metformin in treatment of behavior problems, cognitive and language with fragile X syndrome.


Clinical Trial Description

This is a single-center study at the Children's Hospital of Fudan University for FXS patients aged 2 to 16 years inclusive. It is a randomized, double-blind, placebo-controlled trial of metformin. Studies showed that there were pathogenically over activates of mTOR and MAPK/ERK pathways in FXS. Metformin, a guanidine derivative ,has been shown to reduce mTORC1 pathway activity in an AMPK-dependent manner and has also been shown to reduce MAPK pathway activity. Metformin treatments for FXS have been reported in animal experiments, some open label trials of metformin in FXS patients had been reported. Therefore, metformin has potential to rescue symptoms in children with FXS. In this study, researchers hope to investigate the improvement effect of metformin on FXS symptoms such as behavior problems, cognition, language. The intervention period is 6 months, follow-up visit at 1 year. The researchers will also assess the side effects of the study medication throughout the trial. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05120505
Study type Interventional
Source Children's Hospital of Fudan University
Contact Qiong Xu, Phd MD
Phone 862164931275
Email [email protected]
Status Not yet recruiting
Phase Phase 4
Start date December 11, 2021
Completion date December 30, 2024

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