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

Study Overview
=================
Brief Summary
-----------------
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.

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

Conditions
-----------------
Autism Spectrum Disorders

Intervention / Treatment
-----------------
* Drug: Arbaclofen


Participation Criteria
=================
Eligibility Criteria
-----------------
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.

Ages Eligible for Study
-----------------
Minimum Age: 6 Years
Maximum Age: 17 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arbaclofen<br> | Drug: Arbaclofen<br>* variable dose from 1mg bid to 10 mg tid, oral capsule, 8 week treatment period<br>* 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 | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Autism, Autism Spectrum Disorders, irritability, aberrant behavior