Effects of Metformin on children with Fragile X Syndrome: a randomized, double-blind, placebo-controlled trial
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View abstract on PubMed
Summary
This summary is machine-generated.Metformin showed no significant improvement in the overall Aberrant Behavior Checklist (ABC) score for Fragile X Syndrome (FXS). However, it improved hyperactivity and sleep issues in FXS patients, warranting further research.
Area Of Science
- Neuroscience
- Pharmacology
- Genetics
Background
- Fragile X Syndrome (FXS) is a leading inherited intellectual disability with no approved treatments.
- Metformin's potential therapeutic role in FXS is suggested by preclinical and clinical findings.
- FXS pathophysiology involves mechanisms that may be targeted by metformin.
Purpose Of The Study
- To evaluate the efficacy and safety of metformin in treating FXS.
- To assess metformin's impact on behavioral, developmental, and sleep outcomes in FXS patients.
Main Methods
- A 6-month, randomized, double-blind, placebo-controlled trial involving 34 participants (aged 2-16) with confirmed FXS.
- Participants received weight-adjusted metformin or placebo, with outcomes measured by ABC, GDS-C, ADOS-2, CSHQ, and RBS-R.
- Primary outcome was change in Aberrant Behavior Checklist (ABC) total score.
Main Results
- Metformin significantly improved hyperactivity (p=0.016) and sleep disturbances (p=0.013), especially bedtime resistance (p=0.004).
- No significant differences were found in cognitive, social, or repetitive behavior measures between groups.
- Adverse events were comparable, with most side effects in the metformin group resolving spontaneously.
Conclusions
- Metformin did not significantly improve the primary ABC total score outcome in FXS.
- Secondary outcomes showed significant benefits in hyperactivity and sleep, supporting further investigation.
- The study's modest sample size and duration limit definitive conclusions on long-term effects.
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