Metformin in Children With Fragile X Syndrome

Sponsor
Children's Hospital of Fudan University (Other)
Overall Status
Recruiting
CT.gov ID
NCT05120505
Collaborator
(none)
20
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2
36
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Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
Phase 4

Detailed Description

This is a single-center study at the Children's Hospital of Fudan University for FXS patients aged 2 to 16 years inclusive. It is a randomized, double-blind, placebo-controlled trial of metformin.

Studies showed that there were pathogenically over activates of mTOR and MAPK/ERK pathways in FXS. Metformin, a guanidine derivative ,has been shown to reduce mTORC1 pathway activity in an AMPK-dependent manner and has also been shown to reduce MAPK pathway activity. Metformin treatments for FXS have been reported in animal experiments, some open label trials of metformin in FXS patients had been reported. Therefore, metformin has potential to rescue symptoms in children with FXS.

In this study, researchers hope to investigate the improvement effect of metformin on FXS symptoms such as behavior problems, cognition, language.

The intervention period is 6 months, follow-up visit at 1 year. The researchers will also assess the side effects of the study medication throughout the trial.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
20 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Double (Participant, Investigator)
Primary Purpose:
Treatment
Official Title:
Efficacy and Safety of Metformin in the Treatment of Fragile X Syndrome
Actual Study Start Date :
Dec 29, 2021
Anticipated Primary Completion Date :
Dec 30, 2024
Anticipated Study Completion Date :
Dec 30, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: Metformin group

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

Drug: 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.

Placebo Comparator: Placebo group

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

Drug: 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.

Outcome Measures

Primary Outcome Measures

  1. Change of scores of Aberrant Behavior Checklist (ABC) [From baseline to the 6th month]

    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.

Eligibility Criteria

Criteria

Ages Eligible for Study:
2 Years to 16 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Children's Hospital of Fudan University Shanghai Shanghai China 201102

Sponsors and Collaborators

  • Children's Hospital of Fudan University

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Children's Hospital of Fudan University
ClinicalTrials.gov Identifier:
NCT05120505
Other Study ID Numbers:
  • FXS2021_1.0
First Posted:
Nov 15, 2021
Last Update Posted:
Feb 11, 2022
Last Verified:
Oct 1, 2021
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:

Study Results

No Results Posted as of Feb 11, 2022