5 to 22 Year Olds with Fragile X Syndrome Needed for a Research Study
What is the purpose of this study?
The purpose of this research study is to learn more about the safety, tolerability and effectiveness of an investigational drug treatment called acamprosate and see if it reduces the symptoms (inattention/hyperactivity, social impairment) associated with Fragile X Syndrome (FXS).
Investigational means that the United States Food and Drug Administration (FDA) has not currently approved this drug for the treatment of FXS.
Who can participate?
Children, teens and adults, 5 to 22 years old, who have been diagnosed with FXS may be eligible for participation.
What is involved?
You (if you are at least 18) and/or your child will come to Cincinnati Children’s for all study visits. You must attend and/or bring your child to all study visits to be in this study. The first phase of the study will last between 10 to 20 weeks. You or your child may have an opportunity to participate in a second phase of the study, which lasts 16 weeks.
You or your child will be randomly (by chance) assigned to treatment with either acamprosate or placebo (an inactive form of medication). The researchers conducting this study will not know what group you or your child is in. Neither will you or your child.
While in this study, you and/or your child will be asked to complete some tests and procedures, including:
- Review of you or your child’s medical and psychiatric history
- Interviews/questionnaires about you or your child’s symptoms, behaviors and everyday living skills (both over the phone and in the clinic)
- Blood sample (both participants and parents/legal guardians)
- Physical exam and vital signs (height, weight, temperature, blood pressure and pulse)
- Measurement of the circumference of the head
- Completion of an IQ test
- A structured interaction/play session with you or your child will be completed with a member of the research team.
- Eye-gaze assessment
- Expressive language sampling
A detailed list of procedures will be provided to anyone interested in knowing more about this study.
What are the benefits?
You or your child may not receive a direct medical benefit from participating in this study. However, the information learned from this study may benefit other patients with FXS in the future.
Will I get all the facts about the study?
If interested in participating, or having your child participate, you will be given a consent form that explains all of the details of the study. The form covers all of the procedures, risks, benefits, pay, whom to contact with questions or concerns and more. A member of the study staff will review the consent form with you and will be sure that all of your questions are answered.
What are the risks and discomforts of the study?
There are very minimal risks associated with participation in this study. Any discomforts or risks will be discussed with you (as a participant parent or legal guardian) if you are interested in learning more about the study.
Do participants receive pay, compensation or reimbursement?
Participants will be reimbursed $20 per visit for their time and travel.