Open Label Extension Study of STX209 (Arbaclofen) in Autism Spectrum Disorders

An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of STX209 (Arbaclofen) in Subjects With Autism Spectrum Disorders

This is an open-label extension protocol that will provide necessary data on the safety, tolerability, pharmacokinetics and efficacy of STX209 among subjects with ASD.

Study Overview

• Status: Terminated
• Conditions: Autism Spectrum Disorders
• Intervention / Treatment: Drug: STX209 (arbaclofen)

Detailed Description

This is an open label extension study enrolling by invitation only to those subjects that have completed Seaside protocols 209AS208 and 22007.

Protocol 209AS208, "A Randomized,Double-Blind, Placebo-Controlled, Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Withdrawal in Subjects with Autism Spectrum Disorders."

Protocol 22007, "An Open Label Extension Study to Evaluate the Safety, Tolerability and Pharmacokinetics of STX209 in Subjects with Autism Spectrum Disorder."

This open-label extension will provide data on the following:

1. Evaluate the safety and tolerability of long term use of STX209 (Arbaclofen)
2. Provide supporting pharmacokinetic analyses
3. Assess long term efficacy on social behaviors in subjects with ASD.

• Study Type: Interventional
• Enrollment (Anticipated): 165
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85006
      • Southwest Autism Research & Resource Center
  • California
    • Los Angeles, California, United States, 90024
      • University of California-Los Angeles Neuropsychiatric Institute
    • Sacramento, California, United States, 95817
      • UCDavis M.I.N.D Insitute
  • Florida
    • Miami, Florida, United States, 33126
      • Pharmax Research Clinic
    • Orange City, Florida, United States, 32763
      • Lake Mary Pediatrics
  • Georgia
    • Smyrna, Georgia, United States, 30080
      • Institute For Behavioral Medicine
  • Illinois
    • Chicago, Illinois, United States, 60608
      • Institute for Juvenile Research
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Riley Hospital for Children
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Kennedy Krieger Institute
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
    • Worcester, Massachusetts, United States, 01605
      • University of Massachusetts
  • Missouri
    • Columbia, Missouri, United States, 65211
      • University of Missouri, Thompson Research Center for Autism & Neurodevelopmental Disorders
  • New York
    • New York, New York, United States, 10029
      • Seaver Autism Center, Mount Sinai Medical Center
    • Staten Island, New York, United States, 10314
      • NYS Institute for Basic Research in Developmental Disabilities
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • UNC Chapel Hill Department of Psychiatry
  • Ohio
    • Akron, Ohio, United States, 44308
      • Akron Children's Hospital
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73116
      • Cutting Edge Research
  • Oregon
    • Portland, Oregon, United States, 97210
      • Summit Research Network
  • Pennsylvania
    • Media, Pennsylvania, United States, 19063
      • Suburban Research Associates
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • Tennessee
    • Memphis, Tennessee, United States, 38103
      • University of Tennessee Medical Group, LeBonheur Children's Hospital
    • Nashville, Tennessee, United States, 37203
      • Vanderbilt Kennedy Center
  • Texas
    • Houston, Texas, United States, 77090
      • Red Oaks Psychiatry Associates, P.A.
    • San Antonio, Texas, United States, 78258
      • Road Runner Research
  • Washington
    • Seattle, Washington, United States, 98101
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 21 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Subject completed study 22007 or 209AS208, and showed he can adequately follow the protocol, and with adequate medical justification to enter this study.
• Parent or other legally-authorized representative or caregiver is willing and able to perform all protocol-specified functions.
• Treatment with no more than 2 psychoactive medications
• Subjects with a history of seizure disorder must be adequately well-controlled, as specified in the study protocol
• For female subjects, negative pregnancy test

Exclusion Criteria:

• Comorbid conditions that might interfere with the conduct of the study or confound the interpretation of the study data, or endanger the subject.
• Current use of illicit drugs or alcohol abuse.
• Subjects with a serious adverse event or other adverse event in study 22007 or 209AS208 that was related to STX209, and that endangers the subject, in the opinion of the investigator
• Current use of another investigational drug, or of vigabatrin, tiagabine, or riluzole

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: STX209; Active treatment with STX209	Drug: STX209 (arbaclofen); Long-term, daily, orally-administered STX209

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and tolerability of STX209	Spontaneously-reported adverse events, physical examination, and clinical laboratory assessments	100 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Aberrant Behavior Checklist	Open-label assessment of change from baseline on the ABC	100 weeks

Collaborators and Investigators

• Sponsor: Seaside Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start: November 1, 2011
• Primary Completion (Actual): July 1, 2013
• Study Completion (Anticipated): July 1, 2013

Study Registration Dates

• First Submitted: October 10, 2012
• First Submitted That Met QC Criteria: October 10, 2012
• First Posted (Estimate): October 15, 2012

Study Record Updates

• Last Update Posted (Estimate): July 31, 2013
• Last Update Submitted That Met QC Criteria: July 30, 2013
• Last Verified: July 1, 2013

More Information

Terms related to this study

• Keywords: Autism; Autism Spectrum Disorders; Asperger; Pervasive Developmental Disorder - Not otherwise specified
• Additional Relevant MeSH Terms: Mental Disorders; Pathologic Processes; Neurodevelopmental Disorders; Disease; Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive
• Other Study ID Numbers: 209AS209