Study of Acamprosate in Autism

Children's Hospital Medical Center, Cincinnati36 enrolled

Overview

Recent pharmacotherapy research in autism spectrum disorders (ASD) has successfully focused on treatment of co-occurring symptoms, including inattention, hyperactivity, and irritability that commonly occur in persons with ASD. Despite over two decades of significant pharmacotherapy research, to date no medication has been shown in controlled trials to enhance the core social deficits of ASD. Based upon findings describing the neurobiology of ASD combined with our preliminary results, we believe the novel drug acamprosate will show evidence of reducing social skills deficits associated with ASD.

Each subject with ASD will receive 10 weeks of blinded treatment with acamprosate or matching placebo. After completion of the double-blind phase, all subjects will have an opportunity to receive acamprosate as part of the study procedures for 16 weeks.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

BASIC_SCIENCE

Masking

QUADRUPLE

Enrollment

Conditions

Autistic Disorder

Interventions

AcamprosateDRUG

The maximum dose of acamprosate to be used in this study is 1998 mg per day for those subjects weighing greater than 50kg and 1332 mg per day for those less weighing less than 50kg.

PlaceboDRUG

Subjects receiving placebo will be dosed similarly to the acamprosate group.

Eligibility

Sex: ALLMin age: 5 YearsMax age: 17 Years

Medical Language ↔ Plain English

Inclusion Criteria: * 5-17 year-old outpatients * Diagnosis of ASD * General good health (determined by exam, history, and laboratory work up) * Use of up to two concomitant psychotropic drugs (stable dosing for \>60 days) not impacting glutamate or gamma-aminobutyric acid A (GABA) neurotransmission is allowed * Stable seizure disorder (no seizures in 6 months; on same anti-convulsant dose for \>60 days) * Clinical Global Impression Scale Severity score (CGI-S) of 4 (Moderately III) * Score of 13 on the Social Withdrawal subscale of the Aberrant Behavior Checklist (ABC-SW) at screen and baseline Exclusion Criteria: * Potential subjects with a creatinine clearance \< 50 mL/min or evidence of a previous trial of acamprosate will be excluded

Locations (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Outcomes

Primary Outcomes

Change from baseline to week 10 on the Social WIthdrawal subscale of ABC

The subscales of the ABC including the Social Withdrawal subscale have proven to be reliable measure of symptoms and behaviors associated with ASD. Specifically, the ABC-SW has been validated as a measure of social impairment that differentiates the social behavior of persons with ASD from those developmental delay without a comorbid ASD diagnosis.

Time frame: Week 10

Change Clinical Global Impression- Improvement

The CGI-I is a 7-point scale designed to measure symptomatic change at a specific time as compared to baseline. The CGI-I will be focused on the target symptoms of social impairment.

Time frame: Week 10

Secondary Outcomes

Change in ABC subscales: Irritability, Stereotypy, Hyperactivity, and Inappropriate Speech from baseline to week 10

The subscales of the ABC including the Social Withdrawal subscale have proven to be reliable measure of symptoms and behaviors associated with ASD.

Time frame: Week 10

Data from ClinicalTrials.gov

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