Methodological issues and controversies in clinical trials with child and adolescent patients with bipolar disorder

Report of a consensus conference

Gabrielle A. Carlson, Martha Hellander, Robert Kowatch, Vivek Kusumakar, Thomas Laughren, Ellen Leibenluft, James McCracken, Editha Nottelmann, Daniel Pine, Gary Sachs, David Shaffer, Peter S. Jensen, Renee Simar, Michael Strober, Elizabeth B. Weller, Janet Wozniak, Eric A. Youngstrom, Robert L. Findling, Roger Meyer, Joseph Calabrese & 4 others Melissa P. DelBello, Graham Emslie, Laurie Flynn, Frederick Goodwin

Research output: Contribution to journalArticle

56 Citations (Scopus)

Abstract

Objective: To achieve consensus among researchers, pharmaceutical industry representatives, federal regulatory agency staff, and family advocates on a template for clinical trials of acute mania/bipolar disorder in children and adolescents. Method: The American Academy of Child and Adolescent Psychiatry, in collaboration with Best Practice, convened a group of experts from the key stakeholder communities (including adult psychiatrists with expertise in bipolar disorder) and assigned them to workgroups to examine core methodological issues surrounding the design of clinical trials and, ultimately, to generate a consensus statement encompassing: (1) inclusion/exclusion criteria, (2) investigator training needs and site selection, (3) assessment and outcome measures, (4) protocol design and ethical issues unique to trials involving children/adolescents, and (5) regulatory agency perspectives on these deliberations. Results: Conference participants reached agreement on 18 broad methodological questions. Key points of consensus were to assign priority to placebo-controlled studies of acute manic episodes in children and adolescents aged 10-17 years, who may or may not be hospitalized, and who may or may not suffer from common comorbid psychiatric disorders; to require that specialist diagnostic "gatekeepers" screen youths' eligibility to participate in trials; to monitor interviewer and rater competency over the course of the trial using agreed upon standards; and to develop new tools for assessment, including scales to measure aggression/rage and cognitive function, while using the best available instruments (e.g., Young Mania Rating Scale) in the interim. Conclusions: Methodologically rigorous, large-scale clinical trials of treatment of acute mania are urgently needed to provide information regarding the safety and efficacy, in youth, of diverse agents with potential mood-stabilizing properties.

Original languageEnglish (US)
Pages (from-to)13-27
Number of pages15
JournalJournal of Child and Adolescent Psychopharmacology
Volume13
Issue number1
DOIs
StatePublished - Jan 1 2003

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Bipolar Disorder
Consensus
Clinical Trials
Psychiatry
Research Personnel
Rage
Adolescent Psychiatry
Child Psychiatry
Drug Industry
Aggression
Practice Guidelines
Ethics
Cognition
Placebos
Outcome Assessment (Health Care)
Interviews
Safety
Therapeutics

All Science Journal Classification (ASJC) codes

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

Cite this

Carlson, Gabrielle A. ; Hellander, Martha ; Kowatch, Robert ; Kusumakar, Vivek ; Laughren, Thomas ; Leibenluft, Ellen ; McCracken, James ; Nottelmann, Editha ; Pine, Daniel ; Sachs, Gary ; Shaffer, David ; Jensen, Peter S. ; Simar, Renee ; Strober, Michael ; Weller, Elizabeth B. ; Wozniak, Janet ; Youngstrom, Eric A. ; Findling, Robert L. ; Meyer, Roger ; Calabrese, Joseph ; DelBello, Melissa P. ; Emslie, Graham ; Flynn, Laurie ; Goodwin, Frederick. / Methodological issues and controversies in clinical trials with child and adolescent patients with bipolar disorder : Report of a consensus conference. In: Journal of Child and Adolescent Psychopharmacology. 2003 ; Vol. 13, No. 1. pp. 13-27.
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Carlson, GA, Hellander, M, Kowatch, R, Kusumakar, V, Laughren, T, Leibenluft, E, McCracken, J, Nottelmann, E, Pine, D, Sachs, G, Shaffer, D, Jensen, PS, Simar, R, Strober, M, Weller, EB, Wozniak, J, Youngstrom, EA, Findling, RL, Meyer, R, Calabrese, J, DelBello, MP, Emslie, G, Flynn, L & Goodwin, F 2003, 'Methodological issues and controversies in clinical trials with child and adolescent patients with bipolar disorder: Report of a consensus conference', Journal of Child and Adolescent Psychopharmacology, vol. 13, no. 1, pp. 13-27. https://doi.org/10.1089/104454603321666162

Methodological issues and controversies in clinical trials with child and adolescent patients with bipolar disorder : Report of a consensus conference. / Carlson, Gabrielle A.; Hellander, Martha; Kowatch, Robert; Kusumakar, Vivek; Laughren, Thomas; Leibenluft, Ellen; McCracken, James; Nottelmann, Editha; Pine, Daniel; Sachs, Gary; Shaffer, David; Jensen, Peter S.; Simar, Renee; Strober, Michael; Weller, Elizabeth B.; Wozniak, Janet; Youngstrom, Eric A.; Findling, Robert L.; Meyer, Roger; Calabrese, Joseph; DelBello, Melissa P.; Emslie, Graham; Flynn, Laurie; Goodwin, Frederick.

In: Journal of Child and Adolescent Psychopharmacology, Vol. 13, No. 1, 01.01.2003, p. 13-27.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Methodological issues and controversies in clinical trials with child and adolescent patients with bipolar disorder

T2 - Report of a consensus conference

AU - Carlson, Gabrielle A.

AU - Hellander, Martha

AU - Kowatch, Robert

AU - Kusumakar, Vivek

AU - Laughren, Thomas

AU - Leibenluft, Ellen

AU - McCracken, James

AU - Nottelmann, Editha

AU - Pine, Daniel

AU - Sachs, Gary

AU - Shaffer, David

AU - Jensen, Peter S.

AU - Simar, Renee

AU - Strober, Michael

AU - Weller, Elizabeth B.

AU - Wozniak, Janet

AU - Youngstrom, Eric A.

AU - Findling, Robert L.

AU - Meyer, Roger

AU - Calabrese, Joseph

AU - DelBello, Melissa P.

AU - Emslie, Graham

AU - Flynn, Laurie

AU - Goodwin, Frederick

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N2 - Objective: To achieve consensus among researchers, pharmaceutical industry representatives, federal regulatory agency staff, and family advocates on a template for clinical trials of acute mania/bipolar disorder in children and adolescents. Method: The American Academy of Child and Adolescent Psychiatry, in collaboration with Best Practice, convened a group of experts from the key stakeholder communities (including adult psychiatrists with expertise in bipolar disorder) and assigned them to workgroups to examine core methodological issues surrounding the design of clinical trials and, ultimately, to generate a consensus statement encompassing: (1) inclusion/exclusion criteria, (2) investigator training needs and site selection, (3) assessment and outcome measures, (4) protocol design and ethical issues unique to trials involving children/adolescents, and (5) regulatory agency perspectives on these deliberations. Results: Conference participants reached agreement on 18 broad methodological questions. Key points of consensus were to assign priority to placebo-controlled studies of acute manic episodes in children and adolescents aged 10-17 years, who may or may not be hospitalized, and who may or may not suffer from common comorbid psychiatric disorders; to require that specialist diagnostic "gatekeepers" screen youths' eligibility to participate in trials; to monitor interviewer and rater competency over the course of the trial using agreed upon standards; and to develop new tools for assessment, including scales to measure aggression/rage and cognitive function, while using the best available instruments (e.g., Young Mania Rating Scale) in the interim. Conclusions: Methodologically rigorous, large-scale clinical trials of treatment of acute mania are urgently needed to provide information regarding the safety and efficacy, in youth, of diverse agents with potential mood-stabilizing properties.

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