|Age:||2 to 6 years old|
|Keywords:||Fragile X Syndrome|
|Type:||Drug study, Phase 2|
If you choose to let your child take part in this study, your child will be given either AFQ056 or a placebo throughout the course of the study. A placebo contains no medicine (like a sugar pill). Placebos are used in research studies to see if the results are due to the study drug or due to other reasons.
After 12 months, all participants will take AFQ056 for 8 months during the open-label extension portion of the trial. Doses are increased or decreased depending on how your child tolerates the medicine and if your child has side effects.
Description of Assessments/Questionnaires There will be numerous surveys, tests, and questionnaires related to early learning, language, and behavior. They will take approximately 1 hour depending on the visit. Some of them will be done while your child is alone with a member of the study staff. You will be asked to complete some of the assessments or questionnaires as the parent/caregiver.
￼￼￼￼￼Screening Visit (Visit 1) Study Procedures: There are specific requirements that your child must meet in order to participate in this study. These will be checked by the Study Doctor at the Screening Visit. This initial visit will take approximately 6 to 7 hours.
If your child meets all entry criteria, your child will start taking the medicine (AFQ056 or placebo) twice daily. You will be given bottles with AFQ056 or placebo powder and bottled water that will be used to dissolve the powder. You, the parent/caregiver, will be responsible for mixing the drug and giving it to your child. You will learn how to dissolve the powder in the bottles while you are at the Screening visit. You will have weekly video conference calls with the study team. As a part of monitoring, they will observe you dissolving the study drug each week for the remainder of the study.
Some of the blood collected will also be used to perform genetic testing. We will study components of your child's blood that will help us to understand what happens when genes function abnormally. We want to know how other genes might be related to characteristics of disease in FXS. DNA is the building block of an individual's genetic make-up. We would like to store some of your child‟s blood and DNA samples, if leftover, for future research in fragile X.
Have any questions or want to learn more? Leave your contact details below and the research team will reach out to you.