A Trial of Metformin in Individuals With Fragile X Syndrome (Met)
Fragile X Syndrome, Fragile X Mental Retardation Syndrome, Mental Retardation, X Linked
About this trial
This is an interventional treatment trial for Fragile X Syndrome focused on measuring Congenital Abnormalities, Metformin, Nervous System Diseases, Chromosome Disorders
Eligibility 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.
Sites / Locations
- UC Davis MIND Institute
- University of AlbertaRecruiting
- St Justine HospitalRecruiting
Arms of the Study
Arm 1
Arm 2
Placebo Comparator
Active Comparator
Placebo Medication
Active Metformin Medication
The placebo will be dosed in a weight-dependent manner. Liquid placebo will be provided to any participants unable to swallow the placebo capsules. For participants under 50kg at baseline, the initial dose will be 250mg once per day, and if this dose is well tolerated, they will increase each week by 250mg until a maximum dose of 1000mg daily is reached. For participants at and above 50kg at baseline, the initial dose will be 500mg once per day, and if this dose is well tolerated, they will increase each week by 500mg until a maximum dose of 2000mg daily is reached. After the 4-week titration period, each participant will continue dosing at his or her maximum tolerated dose daily for the remaining 12 weeks of the study.
The active metformin medication will be dosed in a weight-dependent manner. Liquid metformin (100mg/cc) will be provided to any participants unable to swallow the metformin capsules. For participants under 50kg at baseline, the initial dose will be 250mg once per day, and if this dose is well tolerated, they will increase each week by 250mg until a maximum dose of 1000mg daily is reached. For participants at and above 50kg at baseline, the initial dose will be 500mg once per day, and if this dose is well tolerated, they will increase each week by 500mg until a maximum dose of 2000mg daily is reached. After the 4-week titration period, each participant will continue dosing at his or her maximum tolerated dose daily for the remaining 12 weeks of the study.