Open-Label Study of the Safety and Tolerability of STX209 in Subjects With Autism Spectrum Disorders

An Open-Label, Flexible-Dose Evaluation of the Safety and Tolerability of STX209 for Treatment of Irritability in Subjects With Autism Spectrum Disorders

The study objective is to explore the safety and tolerability of STX209 in subjects with Autism Spectrum Disorders and to obtain preliminary data on several measures of efficacy in treating irritability. We hypothesize that STX209 will be safe and well-tolerated.

Study Overview

• Status: Completed
• Conditions: Autism Spectrum Disorders
• Intervention / Treatment: Drug: Arbaclofen

• Study Type: Interventional
• Enrollment (Actual): 32
• Phase: Phase 2

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
  • Connecticut
    • New Haven, Connecticut, United States, 06520
      • Yale Child Study Center
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Riley Hospital For Children
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • University of North Carolina Neurosciences Hospital
  • Tennessee
    • Nashville, Tennessee, United States, 37203
      • Vanderbilt Kennedy Center
  • Texas
    • Houston, Texas, United States, 77090
      • Red Oaks Psychiatry Associates, PA
  • Washington
    • Seattle, Washington, United States, 98101
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 17 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Male or female subjects 6 to 17 years of age, inclusive.
• Diagnosis of Autistic spectrum disorders
• Clinical Global Impression - Severity (CGI-S) rating for aberrant behavior of moderate or higher at screening and at Visit 1 (Day 1).
• An Aberrant Behavior Checklist (ABC-C) Irritability Subscale score ≥16 at screening and at Visit 1 (Day 1).
• 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 3 months prior to Screening (Visit 1) and subjects and their parent/caregiver/legally authorized representative (LAR) will not electively initiate new or modify ongoing interventions for the duration of the study.
• Exclusion Criteria:
• Subjects with known genetic disorders associated with PDD such as fragile X syndrome.
• Subjects with a history of a seizure disorder who are not currently receiving treatment with antiepileptic medication.
• Subjects with any medical condition, including alcohol and drug abuse that 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 currently treated or have been treated in the last 2 weeks with any psychotropic medication except anti-epileptics or who have been treated with fluoxetine in the last 4 weeks.
• Subjects currently treated with vigabatrin or tiagabine.
• Subjects taking another investigational drug currently or within the last 30 days.
• Subjects who have a history of hypersensitivity to racemic baclofen.

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: Arbaclofen	Drug: Arbaclofen; variable dose from 1mg bid to 10 mg tid, oral capsule, 8 week treatment period; Other Names: STX209

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Irritability Subscale of the Aberrant Behavior Checklist, Community Version	The Aberrant Behavior Checklist-Community Edition (ABC-C) is a 58-item questionnaire composed of five different independent subscales. The questionnaire is completed by the parent/caregiver and lists aberrant behaviors and asks about the severity of the problem. ABC-Irritability is one of the subscales and comprises of 15 items. Minimum score is 0, maximum is 45. A decreased score indicates few aberrant behaviors and clinical improvement. The entire ABC-C assessment is administered at baseline and then at the end of each Intervention Period (4 weeks after Baseline).	At 8 weeks during the treatment period

Collaborators and Investigators

• Sponsor: Seaside Therapeutics, Inc.
• Investigators: Principal Investigator: James McCracken, MD, University of California, Los Angeles; Principal Investigator: Craig Erikson, MD, Riley Hospital For Children; Principal Investigator: Bryan King, MD, PhD, Seattle Children's Hospital; Principal Investigator: Linmarie Sikich, MD, University of North Carolina Neurosciences Hospital; Principal Investigator: Jeremy Veenstra-VanderWeele, MD, Vanderbilt Kennedy Center; Principal Investigator: Lawrence Ginsberg, MD, Red Oaks Psychiatry Associates, PA; Principal Investigator: Raun Melmed, MD, Southwest Autism Research & Resource Center; Principal Investigator: Lawrence Scahill, PhD, Yale University

Study record dates

Study Major Dates

• Study Start: February 1, 2009
• Primary Completion (Actual): June 1, 2010
• Study Completion (Actual): September 1, 2010

Study Registration Dates

• First Submitted: February 17, 2009
• First Submitted That Met QC Criteria: February 17, 2009
• First Posted (Estimate): February 18, 2009

Study Record Updates

• Last Update Posted (Estimate): June 14, 2013
• Last Update Submitted That Met QC Criteria: May 10, 2013
• Last Verified: May 1, 2013

More Information

Terms related to this study

• Keywords: irritability; Autism; aberrant behavior; Autism Spectrum Disorders
• Additional Relevant MeSH Terms: Mental Disorders; Pathologic Processes; Neurodevelopmental Disorders; Disease; Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive
• Other Study ID Numbers: 22003 (Other Identifier: Radiometer)