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Clinical trials in children with Down syndrome: issues from a cognitive research perspective.

Publication ,  Journal Article
Heller, JH; Spiridigliozzi, GA; Crissman, BG; Sullivan-Saarela, JA; Li, JS; Kishnani, PS
Published in: Am J Med Genet C Semin Med Genet
August 15, 2006

Clinical and translational research play a key role in the transition of basic research discoveries to effective therapies. In Down syndrome (DS), these research approaches are not well utilized or developed to test new therapies to improve cognitive and/or adaptive function in this population. This article reviews the history of clinical trial research in children with DS from a cognitive research perspective and discusses important issues relevant to the conduct of well designed clinical trials for this population. Specific issues addressed include: funding, study design, study medication, subject recruitment and retention, safety, and efficacy challenges. The Duke Down Syndrome Research Team's program of clinical research of cholinesterase inhibitors for individuals with DS serves as the model application for the identified research principles. It is hoped that this article will raise awareness of the unmet need for clinical research in the cognitive and adaptive function of individuals with DS, especially children with DS.

Duke Scholars

Published In

Am J Med Genet C Semin Med Genet

DOI

ISSN

1552-4868

Publication Date

August 15, 2006

Volume

142C

Issue

3

Start / End Page

187 / 195

Location

United States

Related Subject Headings

  • Research Design
  • Patient Selection
  • Nootropic Agents
  • Humans
  • Genetics & Heredity
  • Down Syndrome
  • Cognition Disorders
  • Cognition
  • Clinical Trials as Topic
  • Clinical Protocols
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Heller, J. H., Spiridigliozzi, G. A., Crissman, B. G., Sullivan-Saarela, J. A., Li, J. S., & Kishnani, P. S. (2006). Clinical trials in children with Down syndrome: issues from a cognitive research perspective. Am J Med Genet C Semin Med Genet, 142C(3), 187–195. https://doi.org/10.1002/ajmg.c.30103
Heller, James H., Gail A. Spiridigliozzi, Blythe G. Crissman, Jennifer A. Sullivan-Saarela, Jennifer S. Li, and Priya S. Kishnani. “Clinical trials in children with Down syndrome: issues from a cognitive research perspective.Am J Med Genet C Semin Med Genet 142C, no. 3 (August 15, 2006): 187–95. https://doi.org/10.1002/ajmg.c.30103.
Heller JH, Spiridigliozzi GA, Crissman BG, Sullivan-Saarela JA, Li JS, Kishnani PS. Clinical trials in children with Down syndrome: issues from a cognitive research perspective. Am J Med Genet C Semin Med Genet. 2006 Aug 15;142C(3):187–95.
Heller, James H., et al. “Clinical trials in children with Down syndrome: issues from a cognitive research perspective.Am J Med Genet C Semin Med Genet, vol. 142C, no. 3, Aug. 2006, pp. 187–95. Pubmed, doi:10.1002/ajmg.c.30103.
Heller JH, Spiridigliozzi GA, Crissman BG, Sullivan-Saarela JA, Li JS, Kishnani PS. Clinical trials in children with Down syndrome: issues from a cognitive research perspective. Am J Med Genet C Semin Med Genet. 2006 Aug 15;142C(3):187–195.

Published In

Am J Med Genet C Semin Med Genet

DOI

ISSN

1552-4868

Publication Date

August 15, 2006

Volume

142C

Issue

3

Start / End Page

187 / 195

Location

United States

Related Subject Headings

  • Research Design
  • Patient Selection
  • Nootropic Agents
  • Humans
  • Genetics & Heredity
  • Down Syndrome
  • Cognition Disorders
  • Cognition
  • Clinical Trials as Topic
  • Clinical Protocols