title: Improving Treatment Trial Outcomes for Rett Syndrome: The Development of Rett-specific Anchors for the Clinical Global Impression Scale.
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.
authors.affiliation: Baylor College of Medicine, Jan and Dan Duncan Neurological Research Institute, Texas Children's Hospital, Houston, TX, USA jneul@ucsd.edu.
authors.forename: Jeffrey L
authors.initials: JL
authors.lastname: Neul
authors.affiliation: Baylor College of Medicine, Houston, TX, USA.
authors.forename: Daniel G
authors.initials: DG
authors.lastname: Glaze
authors.affiliation: University of Alabama at Birmingham, Birmingham, AL, USA.
authors.forename: Alan K
authors.initials: AK
authors.lastname: Percy
authors.affiliation: Gillette Children's Specialty Healthcare, St Paul, MN, USA.
authors.forename: Tim
authors.initials: T
authors.lastname: Feyma
authors.affiliation: Gillette Children's Specialty Healthcare, St Paul, MN, USA.
authors.forename: Arthur
authors.initials: A
authors.lastname: Beisang
authors.affiliation: Baylor College of Medicine, Jan and Dan Duncan Neurological Research Institute, Texas Children's Hospital, Houston, TX, USA.
authors.forename: Thuy
authors.initials: T
authors.lastname: Dinh
authors.affiliation: Baylor College of Medicine, Jan and Dan Duncan Neurological Research Institute, Texas Children's Hospital, Houston, TX, USA.
authors.forename: Bernhard
authors.initials: B
authors.lastname: Suter
authors.affiliation: Holland Bloorview Kids Rehab Hospital, Toronto, Canada.
authors.forename: Evdokia
authors.initials: E
authors.lastname: Anagnostou
authors.affiliation: Autism Therapeutics, London, United Kingdom.
authors.forename: Mike
authors.initials: M
authors.lastname: Snape
authors.affiliation: Neuren Pharmaceuticals, Melbourne, Australia.
authors.forename: Joseph
authors.initials: J
authors.lastname: Horrigan
authors.affiliation: Neuren Pharmaceuticals, Melbourne, Australia.
authors.forename: Nancy E
authors.initials: NE
authors.lastname: Jones
mesh_terms: D000293:Adolescent; D000328:Adult; D002138:Calibration; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D051783:Methyl-CpG-Binding Protein 2 / Q000235:genetics; D008875:Middle Aged; D015518:Rett Syndrome / Q000175:diagnosis* / Q000235:genetics / Q000628:therapy*; D012720:Severity of Illness Index; D055815:Young Adult
chemical_list: C497392:MECP2 protein, human; D051783:Methyl-CpG-Binding Protein 2
keywords: Clinical Global Impression Scales; MECP2; Rett syndrome; clinical trials; outcome measures