Metformin in Children With Fragile X Syndrome

  • End date
    Dec 30, 2024
  • participants needed
  • sponsor
    Children's Hospital of Fudan University
Updated on 24 March 2022


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.


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.

Condition Fragile X Syndrome, Metformin
Treatment Placebo, Metformin
Clinical Study IdentifierNCT05120505
SponsorChildren's Hospital of Fudan University
Last Modified on24 March 2022


Yes No Not Sure

Inclusion Criteria

Genetic testing confirms the diagnosis of FXS
Participate in the study with the informed consent of the guardian
BMI>the 3rd percentile
Not taking more than 2 therapeutic drugs
Able to receive regular follow-up visits

Exclusion Criteria

Primary heart disease
Severe infection or acute clinical illness
Gastrointestinal, renal, or hepatic disease
Previous history of lactic acidosis
previous use of metformin intolerant
Use of angiotensin converting enzyme inhibitors, use of anticoagulants, vitamin B12 deficiency, alcohol consumption
Unstable systemic diseases other than FXS
Changes in clinical medication
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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