Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome
- Registration Number
- NCT01325220
- Lead Sponsor
- Seaside Therapeutics, Inc.
- Brief Summary
There will be four study periods: Screening (up to 14 days in length), the Treatment Period (8 weeks), the Withdrawal Period (22 days), and Follow-up Period (up to 31 days). Subjects will be randomized to receive either STX209 (5 mg twice daily \[BID\], 10 mg BID or 10 mg three times daily \[TID\]) or placebo. Efficacy,safety and tolerability assessments will be performed periodically
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 172
- Molecular documentation of the full FMR1 mutation
- Current pharmacological treatment regimen has been stable for at least 4 weeks prior to Screening.
- Subjects with a history of seizure disorder must currently be receiving treatment with antiepileptics and must have been seizure free for 6 months, or must be seizure free for 3 years if not currently receiving antiepileptics.
- If the subject is already receiving stable non-pharmacologic educational, behavioral, and/or dietary interventions, participation in these programs must have been continuous during the 2 months prior to Screening
- Subjects with any condition, including alcohol and drug abuse, which might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being.
- Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
- Subjects who have taken another investigational drug within the last 30 days.
- Subjects who are not able to take oral medications
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo - STX209 10 mg BID arbaclofen - STX209 5 mg BID arbaclofen - STX209 10 mg TID arbaclofen -
- Primary Outcome Measures
Name Time Method Aberrant Behavior Checklist-Lethargy Social Withdrawal subscale at 8 weeks of treatment This is a single subscale of the aberrant behavior checklist entitiled Lethargy Social Withdrawal
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (28)
New York State Institute for Basic Research in Developmental Disabilities
🇺🇸Staten Island, New York, United States
Kennedy Krieger Institute
🇺🇸Baltimore, Maryland, United States
University of Massachusetts
🇺🇸Worcester, Massachusetts, United States
University of Missouri, Thompson Research Center for Autism & Neurodevelopmental Disorders
🇺🇸Columbia, Missouri, United States
Akron Children's Hospital
🇺🇸Akron, Ohio, United States
Greenwood Genetics Center
🇺🇸Greenwood, South Carolina, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
University of Miami, Mailman Center for Child Development
🇺🇸Miami, Florida, United States
Duke Clinical Research Unit
🇺🇸Durham, North Carolina, United States
Kansas University Clinical Research Center
🇺🇸Fairway, Kansas, United States
Vanderbilt Kennedy Center
🇺🇸Nashville, Tennessee, United States
University of Oklahoma, Physician's Child Study Center
🇺🇸Oklahoma City, Oklahoma, United States
Southwest Autism Research & Resource Center
🇺🇸Phoenix, Arizona, United States
University of California-Davis, M.I.N.D. Institute
🇺🇸Sacramento, California, United States
Miller Children's Hospital
🇺🇸Long Beach, California, United States
Childrens Medical Center Dallas
🇺🇸Dallas, Texas, United States
Children's Hospital of Michigan
🇺🇸Detriot, Michigan, United States
Rush University Medical Center
🇺🇸Chicago, Illinois, United States
Lake Mary Pediatrics
🇺🇸Orange City, Florida, United States
Road Runner Research
🇺🇸San Antonio, Texas, United States
Psychiatric Centers at San Diego
🇺🇸San Diego, California, United States
Emory University School of Medicine
🇺🇸Decatur, Georgia, United States
Seaver Autism Center, Mount Sinai Medical Center
🇺🇸New York, New York, United States
Cincinnati Children's Hospital
🇺🇸Cincinnati, Ohio, United States
Suburban Research Associates/Elwyn Genetics
🇺🇸Media, Pennsylvania, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Red Oaks Psychiatry Associates, P.A.
🇺🇸Houston, Texas, United States
University of Colorado Denver, Children's Hospital
🇺🇸Aurora, Colorado, United States