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

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.

authors

Scahill L,McCracken J,McDougle CJ,Aman M,Arnold LE,Tierney E,Cronin P,Davies M,Ghuman J,Gonzalez N,Koenig K,Lindsay R,Martin A,McGough J,Posey DJ,Swiezy N,Volkmar F,Ritz L,Vitiello B

doi

10.1089/104454601317261555

keywords:

subject

Has Abstract

pub_date

2001-01-01 00:00:00

pages

377-88

issue

4

eissn

1044-5463

issn

1557-8992

journal_volume

11

pub_type

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