Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome
Harbor-C
A Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Function in Children With Fragile X Syndrome
1 other identifier
interventional
172
1 country
28
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
28 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 28, 2011
CompletedFirst Posted
Study publicly available on registry
March 29, 2011
CompletedStudy Start
First participant enrolled
June 1, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2013
CompletedJuly 31, 2013
July 1, 2013
2 years
March 28, 2011
July 30, 2013
Conditions
Outcome Measures
Primary Outcomes (1)
Aberrant Behavior Checklist-Lethargy Social Withdrawal subscale
This is a single subscale of the aberrant behavior checklist entitiled Lethargy Social Withdrawal
at 8 weeks of treatment
Study Arms (4)
STX209 5 mg BID
ACTIVE COMPARATORSTX209 10 mg BID
ACTIVE COMPARATORSTX209 10 mg TID
ACTIVE COMPARATORPlacebo
PLACEBO COMPARATORInterventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (28)
Southwest Autism Research & Resource Center
Phoenix, Arizona, 85006, United States
Miller Children's Hospital
Long Beach, California, 90806, United States
University of California-Davis, M.I.N.D. Institute
Sacramento, California, 95817, United States
Psychiatric Centers at San Diego
San Diego, California, 92108, United States
University of Colorado Denver, Children's Hospital
Aurora, Colorado, 80045, United States
University of Miami, Mailman Center for Child Development
Miami, Florida, 33136, United States
Lake Mary Pediatrics
Orange City, Florida, 32763, United States
Emory University School of Medicine
Decatur, Georgia, 30033, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
Kansas University Clinical Research Center
Fairway, Kansas, 66205, United States
Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
University of Massachusetts
Worcester, Massachusetts, 01605, United States
Children's Hospital of Michigan
Detriot, Michigan, 48201, United States
University of Missouri, Thompson Research Center for Autism & Neurodevelopmental Disorders
Columbia, Missouri, 65211, United States
Seaver Autism Center, Mount Sinai Medical Center
New York, New York, 10029, United States
New York State Institute for Basic Research in Developmental Disabilities
Staten Island, New York, 10314, United States
Duke Clinical Research Unit
Durham, North Carolina, 27710, United States
Akron Children's Hospital
Akron, Ohio, 44308, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
University of Oklahoma, Physician's Child Study Center
Oklahoma City, Oklahoma, 73117, United States
Suburban Research Associates/Elwyn Genetics
Media, Pennsylvania, 19063, United States
Greenwood Genetics Center
Greenwood, South Carolina, 29646, United States
Vanderbilt Kennedy Center
Nashville, Tennessee, 37203, United States
Childrens Medical Center Dallas
Dallas, Texas, 75235, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
Red Oaks Psychiatry Associates, P.A.
Houston, Texas, 77090, United States
Road Runner Research
San Antonio, Texas, 78258, United States
Seattle Children's Hospital
Seattle, Washington, 98101, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Paul Wang, M.D.
Seaside Therapeutics, Inc.
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 28, 2011
First Posted
March 29, 2011
Study Start
June 1, 2011
Primary Completion
June 1, 2013
Last Updated
July 31, 2013
Record last verified: 2013-07