Metformin in Children With Fragile X Syndrome

NCT05120505 ↗

Phase 4 Recruiting 20 enrolled Children's Hospital of Fudan University

RandomizedParallel-groupDouble-blindTreatment

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.

Timeline

Start: 2021-12-29
Primary completion: 2025-12-30
Completion: 2025-12-30

Outcome

Missed primary endpoint

paper In this controlled trial, metformin did not significantly improve the primary outcome of ABC total score. PMID 41291951 ↗

paper Although the primary endpoint was not met PMID 41291951 ↗

Publications

Zhu Y, Li D, Hu C, Tian Y, Lu P, Hagerman RJ, Xu X, Xu Q. Effects of Metformin on children with Fragile X Syndrome: a randomized, double-blind, placebo-controlled trial. Mol Autism. 2025 Nov 25;16(1):57. doi: 10.1186/s13229-025-00691-z.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Metformin	Small molecule	250 mg	—
Subject	Metformin	Small molecule	1000 mg	—