Clinical trial

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

NCT05120505

Name

FXS2021_1.0

Description

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.

Trial arms

Trial start

2021-12-29

Estimated PCD

2024-12-30

Trial end

2024-12-30

Status

Recruiting

Phase

Early phase I

Treatment

Metformin

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.

Arms:

Metformin group

Placebo

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.

Arms:

Placebo group

Size

Primary endpoint

Change of scores of Aberrant Behavior Checklist (ABC)

From baseline to the 6th month

Eligibility 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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE4'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}}, 'enrollmentInfo': {'count': 20, 'type': 'ESTIMATED'}}

Updated at

2024-03-07

1 organization

2 drugs

2 indications

Organization

Children's Hospital of Fudan University

Drug

Indication

Indication

Drug