Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Clinical Trial
. 2015 Nov;30(13):1743-8.
doi: 10.1177/0883073815579707. Epub 2015 Apr 20.

Improving Treatment Trial Outcomes for Rett Syndrome: The Development of Rett-specific Anchors for the Clinical Global Impression Scale

Jeffrey L Neul  1 , Daniel G Glaze  2 , Alan K Percy  3 , Tim Feyma  4 , Arthur Beisang  4 , Thuy Dinh  5 , Bernhard Suter  5 , Evdokia Anagnostou  6 , Mike Snape  7 , Joseph Horrigan  8 , Nancy E Jones  8
Affiliations
Clinical Trial

Improving Treatment Trial Outcomes for Rett Syndrome: The Development of Rett-specific Anchors for the Clinical Global Impression Scale

Jeffrey L Neul et al. J Child Neurol. 2015 Nov.

Abstract

Rett syndrome is a genetically based neurodevelopmental disorder. Although the clinical consequences of Rett syndrome are profound and lifelong, currently no approved drug treatments are available specifically targeted to Rett symptoms. High quality outcome measures, specific to the core symptoms of a disorder are a critical component of well-designed clinical trials for individuals with neurodevelopmental disorders. The Clinical Global Impression Scale is a measure of global clinical change with strong face validity that has been widely used as an outcome measure in clinical trials of central nervous system disorders. Despite its favorable assay sensitivity in clinical trials, as a global measure, the Clinical Global Impression Scale is not specific to the signs and symptoms of the disorder under study. Development of key anchors for the scale, specific to the disorder being assessed, holds promise for enhancing the validity and reliability of the measure for disorders such as Rett syndrome.

Keywords: Clinical Global Impression Scales; MECP2; Rett syndrome; clinical trials; outcome measures.

PubMed Disclaimer

Similar articles

See all similar articles

Cited by

See all "Cited by" articles

References

    1. Laurvick CL, de Klerk KN, Bower C, et al. Rett syndrome in Australia: a review of the epidemiology. J Pediatr. 2006;148:347–352. - PubMed
    1. Neul JL, Kaufmann WE, Glaze DG, et al. for the RettSearch Consortium. Rett Syndrome: Revised Diagnostic Criteria and Nomenclature. Ann Neurol. 2010;68:946–951. - PMC - PubMed
    1. Weese-Mayer DE, Lieske SP, Boothby CM, et al. Autonomic nervous system dysregulation: breathing and heart rate perturbation during wakefulness in young girls with Rett syndrome. Pediatr Res. 2006;60:443–449. - PubMed
    1. Guideri F, Acampa M, Hayek Y, Zappella M. Effects of Acetyl-L-carnitine on Cardiac Dysautonomia in Rett Syndrome: Prevention of Sudden Death? Pediatr Cardiol. 2005;26:574–577. - PubMed
    1. Percy AK, Lee HS, Neul JL, et al. Profiling scoliosis in Rett syndrome. Pediatr. Res. 2010;67:435–439. - PMC - PubMed

Publication types

Substances