RecruitingPhase 4NCT05120505

Metformin in Children With Fragile X Syndrome

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


Sponsor

Children's Hospital of Fudan University

Enrollment

20 participants

Start Date

Dec 29, 2021

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 2 YearsMax Age: 16 Years

Inclusion Criteria5

  • 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 Criteria9

  • 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

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Interventions

DRUGMetformin

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.

DRUGPlacebo

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.


Locations(1)

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, China

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NCT05120505


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