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A Double-Blind, Placebo-Controlled Trial of Metformin in Individuals with Fragile X Syndrome (FXS)

Clinical Trial | Intellectual Disability | Fragile X Syndrome | Metformin | FXS

Fragile X Syndrome (FXS) is the most common cause of intellectual disability. Some people with FXS have increased hunger, obesity, or delayed puberty. A diabetes medication, metformin, has been shown to improve obesity and increased hunger with little side effects/discomfort. In this study, participants will randomly receive either metformin or placebo (looks the same as metformin, but doesn't have active drug) to assess safety and benefits of metformin in areas of language, brain function, eating, behavior, etc. We will assess usefulness of measures of brain/body function and markers in the body that can predict if someone will benefit from metformin. There is also an optional 2-year extension where all participants will receive metformin.

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Conditions de participation

  • Sexe:

    Female, Male

  • Âges admissibles:

    6 to 35

Critères de participation

Inclusion Criteria:
1. Participant has Fragile X syndrome with the full FMR1 mutation (>200 CGG repeats) or the other loss of function mutations of the FMR1 gene; 2. Participant is a male or non-pregnant, non-lactating female age 6 through 35 years, inclusive; 3. Participants who are capable of becoming pregnant must use an acceptable method of birth control for the duration of the study; 4. Participant must have a caregiver (parent, guardian, or other legally authorized representative) who is willing to participate in the whole study; 5. Participant and caregiver are able to attend the clinic regularly and reliably; 6. Participant and/or participant’s caregiver is able to understand, read, write and speak English or French fluently to complete study-related materials; 7. For participants who are not their own legal guardian, participant’s caregiver is able to understand and sign an informed consent to participate in the study; 8. The use of other medication must be stable for at least 4 weeks prior to starting the study and must remain stable until the last study assessment; 9. Behavioral/educational treatments must be stable for 4 weeks prior to the first visit and must remain stable throughout the study; 10. Overall age equivalent is not higher than 13 and IQ is not higher than 85, as assessed at the first visit, and the participant must speak at least 3-word sentences.
Exclusion Criteria:
1. Families that are not cooperative and will not follow through with the demands of this study; 2. Participant has a life-threatening medical problem or other major systemic illness that compromises health or safety and/or would interfere with this study; 3. Age younger than 6 or older than 35 years; 4. History of intolerable adverse events with metformin; 5. Current or recent metformin treatment (within the past 4-months); 6. Body Mass Index (BMI) less than 2 standard deviations below the average for age using the World Health Organization scale; 7. Serum creatinine greater than 1.4 mg/dl (female) or 1.5 mg/dl (male); 8. History of metabolic acidosis or a condition with lactic acidosis; 9. Severe Vitamin B12 deficiency; 10. Pregnancy at screening or unwillingness to use acceptable method of birth control, if applicable; 11. Age equivalent higher than 13 or IQ higher than 85 as assessed at the first visit.

Lieu de l'étude

University of Alberta
University of Alberta
Edmonton, Alberta
Canada

Contactez l'équipe d'étude

Primary Contact

Kerri Whitlock

[email protected]
780-492-9461
Étude parrainée par
University of Alberta
Participants recherchés
Plus d'informations
ID de l'étude: Pro00081059