Kennedy Krieger Institute is looking for children, ages 5 through 11 years, with Fragile X syndrome to participate in a research study.
The purpose of this study is to determine if an investigational drug, arbaclofen, is an effective treatment for social withdrawal symptoms associated with Fragile X syndrome. This study will also assess whether arbaclofen is safe and tolerated by individuals with Fragile X syndrome.
Social Withdrawal behaviors may include:
- Doing nothing but sitting and watching others
- Isolating himself/herself from other children or adults
- Being difficult to reach, contact, or get through to
- Showing fixed facial expressions or lack of emotions
Eligible children may participate in the treatment phase of the study (involving up to 5 visits and up to 12 phone calls over a 12-week period). During the treatment period, participants will receive the study drug, arbaclofen, or a placebo (no active study drug). Assignment to the treatment group (i.e. arbaclofen or placebo) will be decided by chance, similar to the flip of a coin. Whether your child receives placebo or study drug will be unknown to you, your child, the study team members, and the study doctor.
During the treatment period, participants will meet with the study doctor every 2 to 4 weeks and will be monitored for side effects. At the end of the treatment period, the investigational drug will be gradually withdrawn over a 4-week period. Visits will consist of physical and medical exams (including blood draws and ECG testing) and behavioral assessments. For females, there will be a urine pregnancy testing. The risks of this study include side effects associated with arbaclofen such as drowsiness, dizziness, weakness and fatigue (tiredness). Some participants may also find medical procedures such as blood draws, electrocardiogram (ECG), urine testing and physical examination upsetting or uncomfortable.
Participation in this study will require up to 6 visits (each lasting 2-4 hours) to the Kennedy Krieger Institute in Baltimore, MD and up to 12 between-visit phone calls with the study doctor over an 18-week period. For each completed visit, participants will receive $50 compensation for their time and travel expenses. The investigational drug and all studyrelated evaluations are provided at no cost to the participants.
If you are interested in learning more about this study, please contact:
Study Coordinator 443-923-7619 or email: ResearchTrials@kennedykrieger.org
Michael Johnston MD, Principal Investigator, NA_00050003 www.clinicaltrials.gov (#NCT01325220)
This study is sponsored by Seaside Therapeutics (209FX302) and is being performed at up to 25 other sites in the U.S.