Study of Acamprosate in Autism

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Double-Blind Placebo-Controlled Study of Acamprosate in Autism

Conditions
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Autistic Disorder

Intervention / Treatment
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* Drug: Acamprosate
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 5 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Basic Science
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo/sugar pill<br>Placebo will be dosed similar to acamprosate, in terms of dosage form, frequency and duration. | Drug: Placebo<br>* Subjects receiving placebo will be dosed similarly to the acamprosate group.<br>|
| Active Comparator: Acamprosate<br>Acamprosate: 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. Other Name: Campral | Drug: Acamprosate<br>* 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.<br>* Other names: Campral;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | Week 10 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | Week 10 |