A Trial of Metformin in Individuals With Fragile X Syndrome (Met)

Launched by UNIVERSITY OF ALBERTA · Mar 1, 2019

Keywords

ClinConnect Summary

This clinical trial is studying the effects of a medication called metformin in individuals with Fragile X syndrome, a genetic condition that can cause intellectual and developmental challenges. The trial is open to participants aged 6 to 35 years who have been confirmed to have Fragile X syndrome through genetic testing. To be eligible, participants must be able to attend regular appointments, have a caregiver who can support them throughout the study, and be fluent in English or French.

Participants in this study will be randomly assigned to either receive metformin or a placebo (a non-active treatment) and will visit the study site three times over four months for evaluations. The main goals are to check if metformin is safe, how well it is tolerated, and if it can help with language skills, behavior issues, and problems related to weight and appetite. Importantly, participants who can become pregnant must agree to use birth control during the study to ensure safety. This trial is an opportunity to potentially find new ways to support individuals living with Fragile X syndrome.

Gender

ALL

Eligibility criteria

• 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.