Study of Aripiprazole to Treat Children and Adolescents With Autism (PAIRS)

An Open Label Study of Aripiprazole in Children and Adolescents With Autism Spectrum Disorders

The purpose of this open label study in children and adolescents is to examine the acute and long-term effects of aripiprazole on problem behaviors associated with autism spectrum disorders and development in areas which appear to be affected by autism spectrum disorders.

Study Overview

• Status: Completed
• Conditions: Autism Spectrum Disorder; Autistic Disorder; Asperger Syndrome
• Intervention / Treatment: Drug: aripiprazole

Detailed Description

The purpose of this open label study in children and adolescents with autism spectrum disorders (ASD) is to examine the acute (12 week) and long-term (1 year) effects of aripiprazole on problem behaviors associated with ASD and development in three behavioral domains which appear particularly affected by ASD. We are also examining the safety and tolerability of aripiprazole in a range of children with autism. 20 children will be enrolled in the treatment part of the study. An additional 10 children, self selected by guardian desire not to pursue pharmacological treatment, will serve as a nonrandomized control group. This group will be provided with support but not any other treatments, come in for fewer visits and will be asked not to seek pharmacologic treatment outside the study. As of January 2010, we have completed enrollment in the treatment arm. Currently, we are only looking to enroll individuals in the control arm in which participants would not take any psychiatric medication.

• Study Type: Interventional
• Enrollment (Actual): 30
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • University of North Carolina, Department of Psychiatry

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 17 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Subjects must be between 30 months and 17 years 11 months, male or female, of any ethnicity and of any cognitive level
• Meet criteria for diagnosis of autism, Asperger's Disorder or Pervasive Developmental Order NOS (i.e. an autism spectrum disorder) based on clinical evaluation and either the Autism Diagnostic Interview - Revised (ADI-R) or the Autism Diagnostic Observation Scale (ADOS)
• Have a guardian who is able and willing to give written informed consent
• If competent, subject able and willing to give written assent for their own participation
• If on anticonvulsants, subject have been seizure-free and on a stable anticonvulsant regimen for at least 6 months
• Subjects may receive other nonpharmacologic treatment including dietary treatments

Exclusion Criteria:

• Diagnosis of Rett's syndrome or Child Disintegrative Disorder
• Taken psychotropic medications other than clonidine or diphenhydramine within 2 weeks
• Previous trial of aripiprazole
• Pregnant or nursing
• Epilepsy or another significant chronic medical illness

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: open aripipraprazole; Openly provided, flexibly dosed aripiprazole in doses from 1mg to 30mg	Drug: aripiprazole; aripiprazole, open label, flexibly dosed from 1mg to 30mg, provided for up to 12 months; Other Names: Abilify
No Intervention: no medication control; group of children whose parents do not want them to take medications for autism over the year following enrollment in the trial.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Aberrant Behavior Checklist-Irritability at 12 Weeks	The Aberrant Behavior Checklist (ABC) is a caregiver rated questionnaire for assessing problem behaviors of children over the past week relative to typically developing children of the same age. Problem behaviors are rated on a categorical scale between 0 not at all a problem and 3 problem is severe in degree. Raters are instructed to consider both the severity and the frequency of the behavior in determining how severe a problem the behavior is. Thus, if a given behavior occurs more often than in other children of the same age and sex, scores greater than or equal to 1 are warranted. The total score can range from a minimum of 0 (no problem behaviors) to a maximum of 174, higher the number the worse the symptoms.The irritability subscale consists of 15 items with a minimal score of 0 - no irritability problems to 45 - all irritability items rated as severe. A rating of 18 or more on the irritability subscale is considered clinically significant.	Baseline to 12 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical Global Impressions Scale - Severity Score (CGI-S)	One of the most widely used of clinician assessment tools in psychiatry, the CGI is an observer-rated scale that measures illness severity (CGI-S). The CGI is rated on a 7-point scale, with the severity of illness scale using a range of responses from 1 (normal) through to 7 (among the most severely ill patients).	Baseline to 12 weeks
Change From Baseline in the Aberrant Behavior Checklist -Lethargy/Social Withdrawal Subscale at 12 Weeks	The Aberrant Behavior Checklist lethargy/social withdrawal subscale (ABC-SW) is the sum of ratings from 0 - not a problem at all to 3 - problem is severe in degree on 16 items within the Aberrant Behavior checklist (also described in the primary outcome measure section above). Scores can range from 0 to 48, with higher scores indicating more severe problems. The period for the rating is one week and the reference group is typically developing children of the same age and gender as the participant. Both frequency of the behaviors and severity of the problems related to them are considered. High ratings on these items reflect lack of response and interaction with other people in the child's environment.	Baseline to 12 weeks

Collaborators and Investigators

• Sponsor: University of North Carolina, Chapel Hill
• Collaborators: Bristol-Myers Squibb
• Investigators: Principal Investigator: Linmarie Sikich, MD, University of North Carolina, Department of Psychiatry

Study record dates

Study Major Dates

• Study Start: January 1, 2006
• Primary Completion (Actual): June 1, 2012
• Study Completion (Actual): June 1, 2012

Study Registration Dates

• First Submitted: February 7, 2008
• First Submitted That Met QC Criteria: February 7, 2008
• First Posted (Estimate): February 20, 2008

Study Record Updates

• Last Update Posted (Estimate): March 26, 2014
• Last Update Submitted That Met QC Criteria: February 7, 2014
• Last Verified: February 1, 2014

More Information

Terms related to this study

• Keywords: children; adolescents; autism spectrum disorders; open label; aripiprazole
• Additional Relevant MeSH Terms: Mental Disorders; Pathologic Processes; Neurodevelopmental Disorders; Disease; Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive; Asperger Syndrome; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Central Nervous System Depressants; Antipsychotic Agents; Tranquilizing Agents; Psychotropic Drugs; Serotonin Agents; Antidepressive Agents; Dopamine Agonists; Dopamine Agents; Serotonin 5-HT1 Receptor Agonists; Serotonin Receptor Agonists; Serotonin 5-HT2 Receptor Antagonists; Serotonin Antagonists; Dopamine D2 Receptor Antagonists; Dopamine Antagonists; Aripiprazole
• Other Study ID Numbers: 04-1792 GCRC-2491