NCT05120505

Metformin in Children With Fragile X Syndrome

Efficacy and Safety of Metformin in the Treatment of Fragile X Syndrome

• Status: Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Children's Hospital of Fudan University · Academic / Other
• Sex: All
• Age: 2 Years – 16 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Fragile X Syndrome
• Metformin

Interventions

Type	Name	Description
DRUG	Metformin	The patients in this group will be obtain Metformin starting from 50mg everyday at night, and gradually increase to maximum tolerable dose of 1-2g per day according to weights of patients for 6 months.
DRUG	Placebo	The patients in this group will be obtain starch tablets (which had same appearance and size comparing to Metformin) starting from 50mg everyday to 1-2g per day according to weights of patients for 6 months.

Timeline

• Start date: 2021-12-29
• Primary completion: 2025-12-30
• Completion: 2025-12-30
• First posted: 2021-11-15
• Last updated: 2025-02-18

Locations

1 site across 1 country: China