Are you interested in a language intervention that may help your child with Fragile X Syndrome communicate better?
Dr. Elizabeth Berry-Kravis at Rush University is the Protocol Principal Investigator and is leading a national study, NN107 FX-LEARN. There are two purposes to the study. First, to find out if the drug AFQ056, made by the pharmaceutical company Novartis, is safe and has beneficial effects on learning in children who have Fragile X syndrome (FXS). Second, to find out if a structured language intervention can help children with Fragile X syndrome communicate better.
The trial will be a multi-center, phase II randomized, double-blind placebo-controlled parallel-group flexible-dose forced-titration study that will use a novel design to address the impact of AFQ056 on language learning in 3- to 6-year-old children with FXS. Subjects will be in the study a total of 21 months (Screening and 12 months placebo-controlled treatment, 8 months open-label extension, 1 month follow up after final assessment visit). The study is conducted by the Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT). The National Institute of Neurological Disorders and Stroke (NINDS), a division of the National Institutes of Health (NIH), is supporting this trial.
Who can participate?
Children ages 3-6:
- With Fragile X Syndrome.
- Who use intentional acts of communication (including words, gestures, and augmentative communication devices).
What does the study involve?
- 15 study visits over 21 months.
- Behavioral and language assessments, medical history, a physical exam, parent questionnaires, and blood and urine sampling.
- Weekly video conferencing sessions in your home to learn strategies to help your child with language.
- Child will be randomized to either AFQ056 or placebo.
What else can I expect?
- Families receive instruction and feedback from expert speech and language pathologists.
- Travel reimbursement is available.
How can I learn more before applying?
- Read the extensive Q&A, visit http://www.neuronext.org or https://clinicaltrials.gov/show/NCT02920892 or watch the NFXF webinar with Dr. Berry-Kravis below.
Where can I participate?
- The following clinical sites are currently enrolling participants. Use the contact form on this page to be connected with the coordinator, Katie Friedmann, RN, who will work to get you enrolled, if eligible, at the most convenient and available site.
|Boston Children’s Hospital||University of Cincinnati Medical Center||Children’s Hospital of Philadelphia|
|University of Colorado – Denver||Children’s National Medical Center (DC)||University of Pittsburgh|
|Columbia University Medical Center (NY)||University of Texas Southwestern Medical Center||Emory University (GA)|
|Vanderbilt University (TN)||Nationwide Children’s Hospital (OH)||Washington University in Saint Louis|
|Rush University (IL)||Yale University (CT)||University of California at Davis|
NFXF FX-LEARN Q&A Webinar with Dr. Berry-Kravis
If you’d like to learn more or apply for this opportunity, please fill out the form to send your contact information to the study coordinators.