Study of Acamprosate in Autism

Double-Blind Placebo-Controlled Study of Acamprosate in Autism

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.

Study Overview

• Status: Completed
• Conditions: Autistic Disorder
• Intervention / Treatment: Drug: Placebo; Drug: Acamprosate

Detailed Description

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
• Enrollment (Actual): 36
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 17 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: BASIC_SCIENCE
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
PLACEBO_COMPARATOR: Placebo/sugar pill; Placebo will be dosed similar to acamprosate, in terms of dosage form, frequency and duration.	Drug: Placebo; Subjects receiving placebo will be dosed similarly to the acamprosate group.
ACTIVE_COMPARATOR: Acamprosate; 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; 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 Names: Campral

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Children's Hospital Medical Center, Cincinnati
• Collaborators: Autism Speaks

Study record dates

Study Major Dates

• Study Start (ACTUAL): July 2, 2013
• Primary Completion (ACTUAL): September 7, 2017
• Study Completion (ACTUAL): September 7, 2017

Study Registration Dates

• First Submitted: January 30, 2013
• First Submitted That Met QC Criteria: March 13, 2013
• First Posted (ESTIMATE): March 18, 2013

Study Record Updates

• Last Update Posted (ACTUAL): February 3, 2021
• Last Update Submitted That Met QC Criteria: February 1, 2021
• Last Verified: March 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Child Development Disorders, Pervasive; Autism Spectrum Disorder; Autistic Disorder; Alcohol Deterrents; Acamprosate
• Other Study ID Numbers: CIN001-Acamprosate in Autism