Metformin in Children With Fragile X Syndrome

Launched by CHILDREN'S HOSPITAL OF FUDAN UNIVERSITY · Nov 3, 2021

Keywords

ClinConnect Summary

This clinical trial is studying the effects of a medication called metformin on children with Fragile X Syndrome (FXS), which is a genetic condition that can affect behavior, learning, and language. The trial is open to children aged 2 to 16 years old who have been diagnosed with FXS through genetic testing. To be eligible, participants should have a body mass index (BMI) above the 3rd percentile and must not be taking more than two other medications. Importantly, they must also be able to attend regular follow-up visits during the study.

During the trial, children will be randomly assigned to receive either metformin or a placebo (which looks like the medicine but has no active ingredients) for a period of six months. The main goal is to see if metformin can help improve behavior, thinking skills, and language abilities in children with FXS. Parents or guardians will need to provide consent for their child to participate. It's essential to note that some children with certain health conditions, like severe infections or specific heart and kidney diseases, will not be eligible for the study. This trial is currently recruiting participants, and it represents an important step in understanding how metformin might help children with Fragile X Syndrome.

Gender

ALL

Eligibility criteria

• 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:
• • Malnutrition
• • 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