CHDI 2024

Innovation in HD clinical trial design

This invited plenary presentation aims to provide potential approached to address the challenge of limited sensitivity that current clinical trial endpoints have to measure change over time in HD, providing two potential approaches that Roche is currently considering.

New fluid biomarker data from GENERATION HD1 and what it means for GENERATION HD2

This invited plenary presentation shows new data on relevant CSF biomarkers in Huntington's disease from GENERATION HD1 and the implication of these findings in GENERATION HD2. An update on the status of the ongoing GENERATION HD2 study is also being presented.

Assay susceptibility to blood contamination limits the utility of total Huntingtin (HTT) protein in cerebrospinal fluid (CSF) as a biomarker for clinical trials

1) Total HTT protein in CSF may have limited value as biomarker in HD clinical trials 2) Future studies are needed to investigate differences in susceptibility to blood contamination between mutant and total HTT assays.

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CHDI 2024