Study of Acamprosate in Autism

• ClinicalTrials.gov Identifier: NCT01813318
• Recruitment Status: Completed
• First Posted: March 18, 2013
• Last Update Posted: February 3, 2021
• Sponsor: Children's Hospital Medical Center, Cincinnati
• Collaborator: Autism Speaks
• Information provided by (Responsible Party): Children's Hospital Medical Center, Cincinnati

Tracking Information

• First Submitted Date: January 30, 2013
• First Posted Date: March 18, 2013
• Last Update Posted Date: February 3, 2021
• Actual Study Start Date: July 2, 2013
• Actual Primary Completion Date: September 7, 2017 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 18, 2017)

• Change from baseline to week 10 on the Social WIthdrawal subscale of ABC [ Time Frame: 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. 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.
• Change Clinical Global Impression- Improvement [ Time Frame: Week 10 ]
  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.

Original Primary Outcome Measures (submitted: March 13, 2013)

• Change from baseline to week 10 on the Social WIthdrawal subscale of ABC [ Time Frame: Week 10 ]
• Change Clinical Global Impression- Improvement [ Time Frame: Week 10 ]

Current Secondary Outcome Measures (submitted: July 18, 2017)

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

• Original Secondary Outcome Measures (submitted: March 13, 2013): Change in ABC subscales: Irritability, Stereotypy, Hyperactivity, and Inappropriate Speech from baseline to week 10 [ Time Frame: Week 10 ]
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Study of Acamprosate in Autism
• Official Title: Double-Blind Placebo-Controlled Study of Acamprosate in Autism
• Brief Summary: 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: 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
• Study Phase: Phase 1
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Basic Science
• Condition: Autistic Disorder

Intervention

• 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 Name: Campral
• Drug: Placebo
  Subjects receiving placebo will be dosed similarly to the acamprosate group.

Study Arms

• Placebo Comparator: Placebo/sugar pill
  Placebo will be dosed similar to acamprosate, in terms of dosage form, frequency and duration.
  Intervention: Drug: Placebo
• 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

  Intervention: Drug: Acamprosate

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 13, 2013): 36
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: September 7, 2017
• Actual Primary Completion Date: September 7, 2017 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 5 Years to 17 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01813318
• Other Study ID Numbers: CIN001-Acamprosate in Autism
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Children's Hospital Medical Center, Cincinnati
• Original Responsible Party: Same as current
• Current Study Sponsor: Children's Hospital Medical Center, Cincinnati
• Original Study Sponsor: Same as current
• Collaborators: Autism Speaks

Investigators

• Principal Investigator: Craig A Erickson, M.D.; Children's Hospital Medical Center, Cincinnati

• PRS Account: Children's Hospital Medical Center, Cincinnati
• Verification Date: March 2020