Clinical Trials Office

Do You or Your Child Have Fragile X Syndrome (FXS)? Investigational Oral Medication Study for 16 to 50 Year Olds with FXS

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 oral medication, called RO4917523, to treat Fragile X Syndrome (FXS).

Investigational means that this drug has not currently been approved by the United States Food and Drug Administration (FDA) to treat FXS.

Who can participate?

Teens and adults 16 to 50 years old who have been diagnosed with FXS may be eligible to participate.

What is involved?

You or your child will take part in this research study for about 18 weeks and complete 7 to 8 study visits.

During the study, you or your child will be “randomized” into 1 of 3 groups and receive either a dose of the study medication or a placebo (a substance that looks like study medication but has no active ingredient in it). Randomization means that participants are put into the groups by chance, like flipping a coin. No one will know which group you or your child is in, including the researcher.

You will also complete health-assessments, including:

  • Review of medical and medication history
  • Physical exams (including height, vital signs, blood pressure, heart rate, respiration rate, oral temperature and body weight)
  • Electrocardiogram (called an ECG or EKG) to measure the heart’s electrical activity (heartbeat or heart rhythm)
  • Blood and urine tests
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 Fragile X Syndrome in the future.

Will I get all the facts about the study?

If interested in participating, you, as either a participant or parent of a participant, 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.  

Do participants receive pay, compensation or reimbursement?

You will be reimbursed for any study related food and travel costs.  

Who should I contact for more information?

Stephanie Funke

Stephanie.funke@cchmc.org

513-803-1897

Cincinnati Children’s Hospital Medical Center

3333 Burnet Ave., MLC 7004

Cincinnati, OH  45229-3039

Principal Investigator

Craig Erickson, MD

Division of Child Psychiatry

Cincinnati Children’s Hospital Medical Center