Study of Acamprosate in Autism

Cincinnati Children's Hospital Medical Center

Status and phase: Completed; Phase 1

Conditions: Autistic Disorder

Treatments: Drug: Acamprosate; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT01813318

CIN001-Acamprosate in Autism

Details and patient eligibility

About

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.

Full 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.

Enrollment: 36 patients

Sex: All

Ages: 5 to 17 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Basic Science

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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36 participants in 2 patient groups, including a placebo group

Placebo/sugar pill

Placebo Comparator group

Description:

Placebo will be dosed similar to acamprosate, in terms of dosage form, frequency and duration.

Treatment:

Drug: Placebo

Acamprosate

Active Comparator group

Description:

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

Treatment:

Drug: Acamprosate