A Trial of Metformin in Individuals With Fragile X Syndrome

Completed · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Fragile X Syndrome, Fragile X Mental Retardation Syndrome, Mental Retardation, X Linked, Genetic Diseases, X-Linked, Trinucleotide Repeat Expansion, Fra(X) Syndrome, Intellectual Disability, FXS, Neurobehavioral Manifestations, Sex Chromosome Disorders

In brief

This study is a controlled trial of metformin in individuals with fragile X syndrome between the ages of 6 and 25 years. Participants will be randomized in a double-blind design to either drug or placebo and will attend three visits to the study site in a 4-month period for a series of tests. The primary objectives are to assess safety, tolerability, and efficacy of metformin in the treatment of language deficits, behavior problems, and obesity/excessive appetite in individuals with fragile X syndrome.

Key facts

Study ID

NCT03479476

Run by

University of California, Davis

People needed

120

Starts

2018-04-30

Expected to finish

2024-05-22

Last updated by the study team

2025-01-17

Who can join

Age: 6 and older, up to 25. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Molecular genetic confirmation of the full FMR1 mutation or mosaicism.
• Males and non-pregnant, non-lactating females age 6 to 25 years, inclusive.
• Ability of subject and/or caregiver to understand, read, write, and speak English fluently to complete study-related materials.
• IQ ≤ 79 as measured by the Leiter-III at screening.
• Participant is able to speak at least occasional 3-word phrases.
• Participant and parent/caregiver are willing to participate in the protocol and able to attend the clinic regularly and reliably.
• Stable concomitant medication dose and dosing regimen for at least 4 weeks prior to the screening/baseline visit, and the intention to maintain a stable regimen of allowed concomitant medications for the full duration of the study.
• Stable behavioral/educational treatments for at least 4 weeks prior to the screening/baseline visit.
• Sexually active women of childbearing potential must be using a medically acceptable method of birth control for the duration of the study and have a negative urine pregnancy test collected at the initial screening/baseline visit.
• For participants who are not their own legal guardian, the parent/legal authorized representative is able to understand and sign an informed consent to participate in the study.

You may not qualify if…

• Non-cooperation or inability to follow through with the study protocol.
• Life-threatening medical problem or other major systemic illness that compromises health or safety and/or would interfere with the study.
• History of intolerable adverse events with metformin.
• Current or recent metformin treatment (within the past year).
• Body mass index (BMI) less than 2 standard deviations for age.
• Serum creatinine > 1.4 mg/dl (female) or > 1.5 mg/dl (male) at screening.
• History of metabolic acidosis or a condition with lactic acidosis.
• Severe B12 deficiency.
• Pregnancy at screening or unwillingness to use acceptable method of birth control, if applicable.

Where it is running

• UC Davis MIND Institute — Sacramento, California, United States
• University of Alberta — Edmonton, Alberta, Canada
• St Justine Hospital — Montreal, Quebec, Canada

Full record on ClinicalTrials.gov

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