Journal article
Preparing for preventive clinical trials: the Predict-HD study
Archives of neurology (Chicago), Vol.63(6), pp.883-890
06/2006
DOI: 10.1001/archneur.63.6.883
PMID: 16769871
Abstract
The optimal design and outcome measures for preventive clinical trials in neurodegenerative diseases are unknown.
To examine measures that may be associated with disease in the largest cohort ever recruited of prediagnosed individuals carrying the gene expansion for Huntington disease (HD).
The Predict-HD study is a multicenter observational research study in progress at 17 sites in the United States, 4 in Canada, and 3 in Australia.
Genetics and HD outpatient clinics.
Five hundred five at-risk individuals who had previously undergone elective DNA analyses for the CAG expansion in the HD gene (predictive testing) and did not currently have a clinical diagnosis of HD.
Basal ganglia volumes on magnetic resonance images, estimated probability of diagnosis (based on CAG repeat length), performances on 21 standardized cognitive tasks, total scores on 3 scales of psychiatric distress, and motor diagnosis based on the Unified Huntington's Disease Rating Scale.
Several variables showed progressive decline as the diagnostic ratings advanced toward manifest disease. Estimated probability of diagnosis was associated with Unified Huntington's Disease Rating Scale prediagnostic stages and varied from 15% in persons with no motor abnormalities to nearly 40% in those with abnormalities suggestive of probable disease. Striatal volumes, cognitive performances, and even psychiatric ratings declined significantly with motor manifestations of disease.
The documentation of biological and refined clinical markers suggests several clinical end points for preventive clinical trials. Longitudinal study is critical to further validate possible markers for prediagnosed HD.
Details
- Title: Subtitle
- Preparing for preventive clinical trials: the Predict-HD study
- Creators
- Jane S Paulsen - Department of Psychiatry, University of Iowa, Iowa City 52242-1000, USA. jane-paulsen@uiowa.eduMichael HaydenJulie C StoutDouglas R LangbehnElizabeth AylwardChristopher A RossMark GuttmanMartha NanceKarl KieburtzDavid OakesIra ShoulsonElise KaysonShannon JohnsonElizabeth PenzinerPredict-HD Investigators of the Huntington Study Group
- Contributors
- Hans J Johnson (Contributor) - University of Iowa, Electrical and Computer Engineering
- Resource Type
- Journal article
- Publication Details
- Archives of neurology (Chicago), Vol.63(6), pp.883-890
- DOI
- 10.1001/archneur.63.6.883
- PMID
- 16769871
- NLM abbreviation
- Arch Neurol
- ISSN
- 0003-9942
- eISSN
- 1538-3687
- Publisher
- American Medical Association; United States
- Grant note
- 16375 / PHS HHS R01 NS040068 / NINDS NIH HHS 40068 / PHS HHS 01579 / PHS HHS 00059 / PHS HHS
- Language
- English
- Date published
- 06/2006
- Academic Unit
- Roy J. Carver Department of Biomedical Engineering; Electrical and Computer Engineering; Psychiatry; Psychological and Brain Sciences; Iowa Neuroscience Institute; The Iowa Institute for Biomedical Imaging; The Iowa Initiative for Artificial Intelligence; Iowa Informatics Initiative
- Record Identifier
- 9984003984802771
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