Huntington Disease Clinical Trial
Official title:
Natural History Study in Prodromal and Manifest Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD
SHIELD HD is an international, multisite, prospective, longitudinal cohort natural history study to assess the natural history of HD and its biomarkers that are associated with modulation of the number of cytosine-adenine-guanine (CAG) repeats in the mutant Huntingtin (HTT) gene. Approximately 60 patients will be enrolled into the study and followed for up to 24 months at clinical sites in North America and Europe. The results of this study will inform assessments for a future interventional treatment trial.
The rationale for this study is to obtain longitudinal information related to Somatic Instability and DNA damage response genes in HDGECs at various stages of the disease. Established assessments of disease progression will also be recorded. ;
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