Clinical Outcomes and Selection Criteria for Prodromal Huntington's Disease Trials.

Movement Disorders : Official Journal of the Movement Disorder Society
Douglas R Langbehn, Steven Hersch

Abstract

Huntington's disease (HD) develops in individuals with extended cytosine-adenine-guanine (CAG) repeats within the huntingtin (HTT) gene, causing neurodegeneration and progressive motor and cognitive symptoms. The inclusion of mutant HTT carriers in whom overt symptoms are not yet fully manifest in therapeutic trials would enable the development of treatments that delay or halt the accumulation of significant disability. The present analyses assess whether screening prediagnosis (preHD) individuals based on a normalized prognostic index (PIN) score would enable the selection of prodromal preHD subjects in whom longitudinal changes in established outcome measures might provide robust signals. It also compares the relative statistical effect size of longitudinal change for these measures. Individual participant data from 2 studies were used to develop mixed effect linear models to assess longitudinal changes in clinical metrics for participants with preHD and PIN-stratified subcohorts. Relative effect sizes were calculated in 5 preHD studies and internally normalized to evaluate the strength and consistency of each metric across cohorts. Longitudinal modeling data demonstrate the amplification of effect sizes when preHD subcohorts...Continue Reading

References

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Mar 21, 2020·Neurology·Alistair M GliddenChad R Heatwole

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Clinical Trials Mentioned

NCT01574053
NCT00313495
NCT00051324
NCT00052143

Software Mentioned

HD
ENROLL
TRACK
PREDICT
PHAROS
COHORT

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