Using metformin to treat behavior and cognitive issues in children with Fragile X Syndrome
Efficacy and Safety of Metformin in the Treatment of Fragile X Syndrome
This study is testing if the diabetes drug metformin can help improve behavior and thinking skills in children with Fragile X Syndrome.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 2 Years to 16 Years |
| Sex | All |
| Sponsor | Children's Hospital of Fudan University Academic / other |
| Locations | 1 site (Shanghai, Shanghai Municipality) |
| Trial ID | NCT05120505 on ClinicalTrials.gov |
What this trial studies
This controlled trial investigates the effects of metformin on children aged 2 to 16 years with Fragile X Syndrome (FXS). Participants will be randomly assigned to receive either metformin or a placebo for a duration of six months in a double-blind design. The study aims to evaluate improvements in behavior, cognition, and language associated with FXS, while also monitoring any side effects of the medication. The research is conducted at the Children's Hospital of Fudan University, focusing on the potential of metformin to alleviate symptoms linked to the overactivation of specific cellular pathways in FXS.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 2 to 16 years with a confirmed genetic diagnosis of Fragile X Syndrome.
Not a fit: Patients with severe systemic diseases, malnutrition, or those who have previously been intolerant to metformin may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option to improve behavioral and cognitive symptoms in children with Fragile X Syndrome.
How similar studies have performed: Previous studies have shown promising results for metformin in treating symptoms of Fragile X Syndrome, although this specific approach is still being explored.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Shanghai, Shanghai Municipality
- Children's Hospital of Fudan University — Shanghai, Shanghai Municipality, China (Recruiting)
Study contacts
- Study coordinator: Qiong Xu, Phd MD
- Email: stellaxuqiong@hotmail.com
- Phone: 862164931275
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.