Metformin in Children With Fragile X Syndrome

NCT05120505 | Recruiting Phase 4

• 1 other identifier: FXS2021_1.0
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Fragile X Syndrome; Metformin

Outcome Measures

Primary Outcomes (1)

• Change of scores of Aberrant Behavior Checklist (ABC)

  Difference on the scores of the ABC from baseline to the 6th month. ABC is a 58-item behavior scale ranked from 0("not a problem") to 3("severe problem") being the most severe for each item. 5 subscales include irritability, lethargy, stereotypy , hyperactivity and inappropriate speech. The same parent of patient will complete the checklist from baseline to 6th month. The global score changes of ABC will be measured as the primary outcome.

  From baseline to the 6th month

Study Arms (2)

Metformin group

EXPERIMENTAL

The patients will be obtain Metformin starting from 50mg everyday to 1-2g per day for 6 months.

Drug: Metformin

Placebo group

PLACEBO COMPARATOR

The patients will be obtain starch tablets starting from 50mg everyday to 1-2g per day for 6 months.

Drug: Placebo

Interventions

Metformin DRUG

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.

Metformin group

Placebo DRUG

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.

Placebo group

Eligibility Criteria

• Age: 2 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Hospital of Fudan University: lead

Study Sites (1)

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, 201102, China

RECRUITING

Related Publications (1)

• 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.

  PMID: 41291951 DERIVED

MeSH Terms

Conditions

Fragile X Syndrome

Interventions

Metformin

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System

Intervention Hierarchy (Ancestors)

Biguanides; Guanidines; Amidines; Organic Chemicals

Central Study Contacts

Qiong Xu, Phd MD

CONTACT

862164931275; stellaxuqiong@hotmail.com

Chunchun Hu, MD

CONTACT

862164931275; 14211240007@fudan.edu.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 3, 2021
• First Posted: November 15, 2021
• Study Start: December 29, 2021
• Primary Completion: December 30, 2025
• Study Completion: December 30, 2025
• Last Updated: February 18, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)