Hostname: page-component-78c5997874-s2hrs Total loading time: 0 Render date: 2024-11-19T05:40:04.812Z Has data issue: false hasContentIssue false
  • English
  • Français

Impact of Recent Findings on Study Design of Future Autism Clinical Trials

Published online by Cambridge University Press:  07 November 2014

Eric Hollander ,
Ann Phillips ,
Bryan H. King ,
Donald Guthrie ,
Michael G. Aman ,
Paul Law ,
Thomas Owley  and
Ricki Robinson
Get access Rights & Permissions [Opens in a new window]

Abstract

There are specific challenges to studying the design of pharmacologic trials in child/adolescent and adult autism, such as subject stratification and parallel versus crossover designs. This article describes how optimal study design is influenced by subject selection and outcome measures chosen. Lessons learned in study design from the Research Units on Pediatric Psychopharmacology Autism Network trial with risperidone, Seaver Center trials with fluoxetine and valproate, Dartmouth trials with amantadine, and National Institutes of Health secretin trials are highlighted. The Internet System for Assessing Autistic Children system for managing multicenter clinical trials in autism and statistical issues in autism research are also described.

Type
Review Article
Information
CNS Spectrums , Volume 9 , Issue 1 , January 2004 , pp. 49 - 56
Copyright
Copyright © Cambridge University Press 2004

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

REFERENCES

1.McCraken, JT, McGough, J, Shah, B, et al.Risperidone in children with autism and serious behavioral problems. N Engl J Med. 2002:347:314–321.CrossRefGoogle Scholar
2.Lord, C, Rutter, M, LeCouteur, A. Autism Diagnostic Interview-Revised: a revised version of a diagnostic interview for caregivers of individuals with possible pervasive developmental disorders. J Autism Dev Disord. 1994;24:659–685.CrossRefGoogle ScholarPubMed
3.Lord, C, Rutter, M, DiLavre, PC. Autism Diagnostic Observation Schedule-Generic (ADOS-G). San Antonio, Tex: Psychological Corp.; 1998.Google Scholar
4.Sandler, AD, Sutton, KA, DeWeese, J, Girardi, Ma, Sheppard, V, Bodfish, JW. Lack of benefit of a single dose of synthetic human secretin in the treatment of autism and pervasive developmental disorder. N Engl J Med. 1999;341:1801–1806.CrossRefGoogle ScholarPubMed
5.Dunn-Geier, J, Ho, HH, Auersperg, E, et al.Effect of secretin on children with autism: a randomized controlled trial. Dev Med Child Neurol. 2000;42:796–802.CrossRefGoogle ScholarPubMed
6.Buitelaar, JK, Dekker, ME, van Ree, JM, van Engeland, H. A controlled trial with ORG 2766, an ACTH-(4-9) analog, in 50 relatively able children with autism. Eur Neuropsychopharmacol 1996;6:13–19.CrossRefGoogle ScholarPubMed
7.King, BH, Wright, DM, Handen, BL, et al.Double-blind, placebocontrolled study of amantadine hydrochloride in the treatment of children with autistic disorder. J Am Acad Child Adolesc Psychiatry. 2001;40:6658–665.CrossRefGoogle ScholarPubMed
8.Buxbaum, J, Silverman, J, Smith, C, et al.Evidence for a susceptibility gene for autism on chromosome 2 and for genetic heterogeneity. Am J Human Genet. 2001;68:1514–1520.CrossRefGoogle ScholarPubMed
9.Bradford, Y, Haines, J, Hutcheson, H, et al.Incorporating language phenotypes strengthens evidenc of linkage to autism. Am J Med Genet. 2001;105:539–547.CrossRefGoogle Scholar
10.Chez, MG, Buchanan, CP, Bagan, BT, et al.Secretin and autism: a two-part clinical investigation. J Autism Dev Disord. 2000;30:87–94.CrossRefGoogle ScholarPubMed
11.Coniglio, SJ, Lewis, JD, Lang, C, et al.A randomized, double-blind, placebo-controlled trial of single-dose intravenous secretin as treatment for children with autism. J Pediatr. 2001;138:649–655.CrossRefGoogle ScholarPubMed
12.Ekman, G, Miranda-Linne, F, Gillberg, C, Garle, M, Wetterberg, L. Fenfluramine treatment of twenty children with autism. J Autism Dev Disord. 1989;19:511–532.CrossRefGoogle ScholarPubMed
13.Hintze, J. NCSS and PASS, Number Cruncher Statistical Software [computer program]. Kaysville, UT; 2001.Google Scholar
14.Hollander, E, Dolgoff-Kaspar, R, Cartwright, C, Rawitt, R, Novotny, S. An open trial of divalproex sodium in autism spectrum disorders. J Clin Psychiatry. 2001;62:530–534.CrossRefGoogle ScholarPubMed
15.Coccaro, EF, Harvey, PD, Kupsaw-Lawrence, E, Herbert, JL, Bernstein, DP. Development of neuropharmacologically based behavioral assessments of impulsive aggressive behavior. J Neuropsychiatry Clin Neurosci. 1991;3:S44–S51.Google ScholarPubMed
16.Young, RC, Biggs, JT, Ziegler, Ve, Meyer, DA. A rating scale for mania: reliability, validity, and sensitivity. Br J Psychiatry. 1978;133:429–435.CrossRefGoogle ScholarPubMed
17.Poznanski, EO, Cook, SC, Carroll, BJ. A depression rating scale for children. Pediatrics. 1979;64:442–450.CrossRefGoogle ScholarPubMed
18.Owley, T, McMahon, W, Cook, EH, et al.Multisite, double-blind, placebo-controlled trial of porcine secretin in autism. J Am Acad Child Adolesc Psychiatry. 2001;40:1293–1299.CrossRefGoogle ScholarPubMed
19. Internet System for Assessing Autistic Children Web site. Available at: http://www.autismtools.org. Accessed December 1, 2003.Google Scholar