Effects of Metformin on children with Fragile X Syndrome: a randomized, double-blind, placebo-controlled trial.
Yiting Zhu, Dongyun Li, Chunchun Hu...
https://pubmed.ncbi.nlm.nih.gov/41291951Actively Recruiting
Phase 4
Age: 2Years - 16Years
All Genders
ID05120505
Efficacy and Safety of Metformin in the Treatment of Fragile X Syndrome
Led by Children's Hospital of Fudan University · Updated on 2025-02-18
20
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the use of metformin in children aged 2 to 16 years with Fragile X Syndrome (FXS), a genetic condition. This randomized, double-blind, placebo-controlled trial aims to study metformin's effects on behavior problems, cognition, and language in children with FXS. The study is conducted at the Children's Hospital of Fudan University and is motivated by previous findings that metformin may influence pathways involved in FXS symptoms. Participants will be randomly assigned to receive either metformin or a placebo for a treatment period of 6 months. Metformin dosing starts at 50mg daily and is gradually increased to a maximum dose of 1 to 2 grams per day based on patient weight. The placebo group will receive starch tablets matching the metformin in appearance and dosage schedule. After the 6-month intervention, participants will have a follow-up visit at 1 year to monitor ongoing effects and safety. During the study, children will undergo regular assessments including behavioral evaluations using the Aberrant Behavior Checklist (ABC) to measure changes from baseline to 6 months. Researchers will monitor side effects and overall safety throughout the trial. The total participation duration includes the 6-month treatment and the follow-up visit at 1 year to evaluate longer-term outcomes.
CONDITIONS
Brief Title
Metformin in Children With Fragile X Syndrome
Who Can Participate
Age: 2Years - 16Years
All Genders
Eligibility Criteria
You may qualify if you...
- Genetic testing confirms the diagnosis of Fragile X Syndrome
- Guardian provides informed consent for participation
- Body mass index (BMI) above the 3rd percentile
- Taking no more than two therapeutic drugs
- Able to attend regular follow-up visits
You will not qualify if you...
- Malnutrition
- Primary heart disease
- Severe infection or acute clinical illness
- Gastrointestinal, renal, or hepatic disease
- Previous history of lactic acidosis
- Previous intolerance to metformin
- Use of angiotensin converting enzyme inhibitors
- Use of anticoagulants
- Vitamin B12 deficiency
- Alcohol consumption
- Unstable systemic diseases other than Fragile X Syndrome
- Changes in clinical medication
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - 6 months
Participants receive metformin or placebo daily with dose starting at 50mg and gradually increasing to a maximum of 1-2g per day according to weight, for symptom improvement in Fragile X Syndrome.
Regular visits during treatment period to monitor progress and side effects
Follow-up
Duration - 6 months
Participants are monitored for safety and long-term effects of the study medication after completing treatment.
1 follow-up visit at 1 year from treatment start
Trial Site Locations
Total: 1 location
1
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China, 201102
Actively Recruiting
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Research Team
Q
Qiong Xu, Phd MD
C
Chunchun Hu, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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