Methodological issues in designing a multisite trial of risperidone in children and adolescents with autism

Larry Scahill, James McCracken, Christopher J. McDougle, Michael Aman, L. Eugene Arnold, Elaine Tierney, Pegeen Cronin, Mark Da Vies, Jaswinder Ghuman, Nilda Gonzalez, Kathleen Koenig, Ronald Lindsay, Andres Martin, James McGough, David J. Posey, Naomi Swiezy, Fred Volkmar, Louise Ritz, Benedetto Vitiello

Research output: Contribution to journalArticle

38 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)377-388
Number of pages12
JournalJournal of Child and Adolescent Psychopharmacology
Volume11
Issue number4
DOIs
StatePublished - Dec 1 2001

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Psychiatry and Mental health
  • Pharmacology (medical)

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    Scahill, L., McCracken, J., McDougle, C. J., Aman, M., Arnold, L. E., Tierney, E., Cronin, P., Da Vies, M., Ghuman, J., Gonzalez, N., Koenig, K., Lindsay, R., Martin, A., McGough, J., Posey, D. J., Swiezy, N., Volkmar, F., Ritz, L., & Vitiello, B. (2001). Methodological issues in designing a multisite trial of risperidone in children and adolescents with autism. Journal of Child and Adolescent Psychopharmacology, 11(4), 377-388. https://doi.org/10.1089/104454601317261555