A Trial of Metformin in Individuals With Fragile X Syndrome

• Status: Recruiting
• Trial end date: 2021-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of California, Davis
• Treatments: Metformin

Criteria

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.