Diabetes drug metformin tested for fragile x symptoms
NCT ID NCT03862950
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested whether metformin, a common diabetes medication, can help improve language, behavior, and weight problems in people with fragile X syndrome. 125 participants aged 6 to 35 took either metformin or a placebo for 4 months. Researchers measured changes in language skills and behavior to see if the drug made a difference.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Metformin
- What this could lead to
- If it works, this could point toward a treatment to improve language and behavior and reduce obesity in people with fragile X syndrome.
- What could go wrong
- This is a phase 2 trial with only 125 participants, so results may not be conclusive. Metformin is a diabetes drug, and its benefits for fragile X syndrome are not yet proven.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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125 people
The number who actually took part.
- Started
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May 2019
- Finished
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Dec 2025
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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6 to 35 years
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Subject has Fragile X syndrome with a molecular genetic confirmation of the full FMR1 mutation (\>200 CGG repeats) or the other loss of function mutations of the FMR1 gene (SNVs and deletions of the gene). * Subject is a male or non-pregnant, non-lactating female age 6 through 35 years, inclusive. * Subjects who are capable of becoming pregnant must use an acceptable method of birth control for the duration of the study. Acceptable forms of birth control include abstinence (only for subjects who are not sexually active), intrauterine devices in place for at least 3 months, oral contraceptives, surgical sterilization, or adequate barrier methods. * Subject must have a caregiver (parent, guardian, or other legally authorized representative) who is willing to participate in the whole study. * Subject and caregiver are able to attend the clinic regularly and reliably. * Subject and/or subject's caregiver is able to understand, read, write and speak English or French fluently to complete study-related materials. * For subjects who are not their own legal guardian, subject's caregiver is able to understand and sign an informed consent to participate in the study. * The use of concomitant medication must be stable, in terms of dose and dosing regimen, for at least 4 weeks prior to Screening and must remain stable during the period between first visit (Screening) and the commencement of the study; every effort should be made to maintain stable regimens of allowed concomitant medications from the time of commencement of double-blind study medication until the last study assessment. * Behavioral/educational treatments must be stable for 4 weeks prior to first visit (Screening) and must remain stable during the period between Screening and the commencement of randomized double-blind study medication. * 10\. Overall age equivalent is not higher than 13 and IQ is not higher than 85, as assessed at Screening on the Leiter-III, and subject must speak at least occasional 3-word phrases. Exclusion Criteria: * Families that are not cooperative and will not follow through with the demands of this study. * Subject has a life-threatening medical problem or other major systemic illness that compromises health or safety and/or would interfere with this study. * Age younger than 6 or older than 35 years. * History of intolerable adverse events with metformin. * Current or recent metformin treatment (within the past 4-months). * BMI inferior to 2 standard deviations below the mean for age using the World Health Organization scale. * Serum creatinine \> 1.4 mg/dl (female) or \> 1.5 mg/dl (male). * History of metabolic acidosis or a condition with lactic acidosis. * Severe Vitamin B12 deficiency. * Pregnancy at screening or unwillingness to use acceptable method of birth control, if applicable. * Age equivalent higher than 13 or IQ higher than 85 on the Leiter-III at Screening.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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CHU Sainte-Justine
Montreal, Quebec, H3T 1C5, Canada
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University of Alberta
Edmonton, Alberta, T6G 2E1, Canada
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