Abstract
I32 Using the global clinical research platform of enroll-hd to facilitate the enrollment of premanifest Huntington disease (HD) participants in clinical trials
Journal of neurology, neurosurgery and psychiatry, Vol.87(Suppl 1), pp.A70-A70
09/2016
DOI: 10.1136/jnnp-2016-314597.197
Abstract
Background Enroll-HD is a global integrated clinical research platform for Huntington’s disease (HD), which includes structured annual assessments and biosample collection, and allows for in silico participant selection for clinical studies. The HD Prognostic Index Normed (PIN) indexes progression in HD and is a useful tool for identifying appropriate individuals for specific clinical trials. Aims To examine the clinical, genetic, and regional characteristics of 859 premanifest HD (preHD) subjects included in the second periodic dataset of Enroll-HD. Methods/techniques Descriptive statistics, PIN, and willingness of participants to collaborate in future research were evaluated. Results/outcome Most of the preHD participants were from Europe (43%) and North American (46%) sites; the majority was female (67%). Mean (± SD) scores for the preHD cohort were: age 40.6 ± 11.8, TFC 12.7 ± 0.9, MMSE 28.6 ± 1.7, CAG repeat length 42.4 ± 2.8. Almost all subjects agreed to be contacted for additional studies (99%) and contributed biosamples (99%). North American subjects had a higher mean Total Motor Score (5.0 ± 6.1) than their European counterparts (2.7 ± 3.4). Based on PIN, all the regional cohorts had less than 50% probability of motor diagnosis over the next 10 years, but the European cohort was the least progressed (lowest probability of 10 year motor diagnosis), and the Australasian cohort the most progressed (though still less than 50% probability). Conclusions Enroll-HD’s infrastructure is ready to facilitate the enrollment of preHD participants in clinical trials. There are relevant geographic differences in recruitment numbers and clinical characteristics of participants that should be considered.
Details
- Title: Subtitle
- I32 Using the global clinical research platform of enroll-hd to facilitate the enrollment of premanifest Huntington disease (HD) participants in clinical trials
- Creators
- Jody Corey-Bloom - University of California, San DiegoJeffrey D Long - University of UlmCheryl J Fitzer-Attas - CHDI FoundationBernhard Landwehrmeyer - University of UlmCristina Sampaio - CHDI Foundation
- Resource Type
- Abstract
- Publication Details
- Journal of neurology, neurosurgery and psychiatry, Vol.87(Suppl 1), pp.A70-A70
- DOI
- 10.1136/jnnp-2016-314597.197
- ISSN
- 0022-3050
- eISSN
- 1468-330X
- Language
- English
- Date published
- 09/2016
- Academic Unit
- Biostatistics; Psychiatry
- Record Identifier
- 9984281758902771
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