|Age:||6 to 25 years old|
|Keywords:||fragile x syndrome, metformin, genetic diseases, mental retardation|
|Type:||Drug study, Phase 2, 3|
If you decide to take part in this study, you or your child will receive either metformin or placebo for 4 months.
You will complete assessments and questionnaires at three separate visits to the MIND Institute. These will occur at: - screening / baseline visit - checkup at 2 months - final follow-up visit at 4 months
A physician will also conduct a physical and neurological exam at every visit. You will discuss of any side effects of the study drug as well as any medical issues the subject may be having.
At every visit, a fasting blood draw will take place to ensure patient safety and to perform exploratory biomarker studies. The total amount of blood drawn at each blood draw will be about 8 teaspoons or less. Urine collection will also be attempted at every visit. This will be for biomarker studies, urinalysis, and pregnancy testing if applicable. At baseline only, a small skin biopsy will be obtained from the back of the shoulder. The team will utilize a small punch biopsy after numbing the skin with lidocaine to minimize discomfort..
For those subjects able to swallow capsules, study drug will be provided in capsule form. For those subjects unable to swallow capsules, liquid study drug may be available. A 2-month supply of the study drug will be dispensed at the baseline visit. Another 2-month supply will be dispensed at the 2-month visit at the MIND Institute.
Between visits to the MIND Institute, we will call you weekly during the first month. They will monitor side effects and medication changes and ensure proper dosing with the study medication. The Week 4 (1-month) phone call will be conducted by the study physician. We will call you once more at Week 12 (halfway between your 2-month and 4-month visits).
Randomization to metformin or placebo will be carried out by the UC Davis pharmacy. You will have an equal (50%) chance of being given each treatment, which is like flipping a coin. Neither you nor the study doctor will know or choose which treatment you are getting at any point during the study. You will receive the same treatment for the full duration of the study.
Have any questions or want to learn more? Leave your contact details below and the research team will reach out to you.