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Clinical trials in children with Down syndrome: Issues from a cognitive research perspective
Author(s) -
Heller James H.,
Spiridigliozzi Gail A.,
Crissman Blythe G.,
SullivanSaarela Jennifer A.,
Li Jennifer S.,
Kishnani Priya S.
Publication year - 2006
Publication title -
american journal of medical genetics part c: seminars in medical genetics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.419
H-Index - 101
eISSN - 1552-4876
pISSN - 1552-4868
DOI - 10.1002/ajmg.c.30103
Subject(s) - clinical trial , clinical research , cognition , perspective (graphical) , translational research , population , research design , clinical study design , medicine , psychology , clinical psychology , psychiatry , computer science , pathology , sociology , social science , environmental health , artificial intelligence
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. © 2006 Wiley‐Liss, Inc.