Clinical trial NCT01325220: Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Fragile X Syndrome
Lead sponsor: Seaside Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2011-03-29
Enrollment: 172
Sex: ALL
Minimum age (years): 5
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: Aberrant Behavior Checklist-Lethargy Social Withdrawal subscale
timeFrame: at 8 weeks of treatment
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Southwest Autism Research & Resource Center
state: Arizona
city: Long Beach
country: United States
facility: Miller Children's Hospital
state: California
city: Sacramento
country: United States
facility: University of California-Davis, M.I.N.D. Institute
state: California
city: San Diego
country: United States
facility: Psychiatric Centers at San Diego
state: California
city: Aurora
country: United States
facility: University of Colorado Denver, Children's Hospital
state: Colorado
city: Miami
country: United States
facility: University of Miami, Mailman Center for Child Development
state: Florida
city: Orange City
country: United States
facility: Lake Mary Pediatrics
state: Florida
city: Decatur
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Fairway
country: United States
facility: Kansas University Clinical Research Center
state: Kansas
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Worcester
country: United States
facility: University of Massachusetts
state: Massachusetts
city: Detriot
country: United States
facility: Children's Hospital of Michigan
state: Michigan
city: Columbia
country: United States
facility: University of Missouri, Thompson Research Center for Autism & Neurodevelopmental Disorders
state: Missouri
city: New York
country: United States
facility: Seaver Autism Center, Mount Sinai Medical Center
state: New York
city: Staten Island
country: United States
facility: New York State Institute for Basic Research in Developmental Disabilities
state: New York
city: Durham
country: United States
facility: Duke Clinical Research Unit
state: North Carolina
city: Akron
country: United States
facility: Akron Children's Hospital
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Oklahoma City
country: United States
facility: University of Oklahoma, Physician's Child Study Center
state: Oklahoma
city: Media
country: United States
facility: Suburban Research Associates/Elwyn Genetics
state: Pennsylvania
city: Greenwood
country: United States
facility: Greenwood Genetics Center
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt Kennedy Center
state: Tennessee
city: Dallas
country: United States
facility: Childrens Medical Center Dallas
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Houston
country: United States
facility: Red Oaks Psychiatry Associates, P.A.
state: Texas
city: San Antonio
country: United States
facility: Road Runner Research
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
Study officials:
affiliation: Seaside Therapeutics, Inc.
name: Paul Wang, M.D.
role: STUDY_DIRECTOR
References: