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Updated: Aug 10, 2026
Eye Tracking Young Children with Autism
Published on: March 27, 2012
Methodological issues in designing a multisite trial of risperidone in children and adolescents with autism
L Scahill1, J McCracken, C J McDougle
1Research Unit on Pediatric Psychopharmacology Yale University, New Haven, Connecticut 06520, USA. lawrence.scahill@yale.edu
Objective:
To describe the methodological challenges and decisions made in developing a multisite, controlled study of risperidone in children and adolescents with autism.
Methods:
Review the design considerations for clinical trials in children with autistic disorder accompanied by severe tantrums, aggressive and/or self-injurious behaviors. These design considerations include the definition of inclusion criteria that are relevant to clinical practice and matching study design to the goal of evaluating short- and long-term effects. Additional ethical and scientific issues concern the length of trial and sample size.
Results:
We undertook a short-term, placebo-controlled study to evaluate the efficacy and safety of risperidone in children and adolescents with autistic disorder. This trial design was followed by an extended open-label maintenance on risperidone to confirm durability of treatment effects and to monitor safety. Finally, a placebo-controlled discontinuation study tested the need for continuous treatment.
Conclusions:
In the absence of standard pharmacological treatment for children with autistic disorder, a placebo-controlled study remains the most appropriate method of testing efficacy and safety. The clinical relevance of this study is enhanced by the addition of an extended maintenance phase followed by a placebo discontinuation.
