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Activity Number:
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314
- Statistical Advancements in Neurodegeneration Trial Designs and Analyses
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Type:
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Topic Contributed
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Date/Time:
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Wednesday, August 5, 2020 : 10:00 AM to 11:50 PM
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Sponsor:
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Section for Statistical Programmers and Analysts
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Abstract #312650
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Title:
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Multivariate Endpoint, Disease Progress Model and Other New Features of the DIAN-TU Platform Trial
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Author(s):
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Guoqiao Wang* and Scott Berry and Chengjie Xiong and Jason Hassenstab and Melanie Quintana and Eric McDade and Paul Delmar and Matteo Vestrucci and Randall Bateman and Andrew Aschenbrenner
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Companies:
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Washington University in St Louis and Berry Consultants and Washington University in St Louis and Washington University in St Louis and Berry Consultants and Washington University in St Louis and F. Hoffmann-La Roche Ltd and University of Texas at Austin and Washington University in St Louis and Washington University in St Louis
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Keywords:
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Platform Trial;
Alzheimer Disease;
Multivariate endpoint;
Proportional Treatment Effect;
Run-in Design;
Dose escalation
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Abstract:
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The Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU) trial is an adaptive platform trial testing multiple drugs to slow or prevent the progression of Alzheimer’s disease (AD) in autosomal dominant Alzheimer’s disease (ADAD) families. Because ADAD is a rare disease representing less than 1% of all AD cases, several innovative strategies have been implemented to reduce the burden of enrolment and improve efficiency. The DIAN-TU is designed to be a platform trial so that multiple drugs can be tested simultaneously or sequentially using a single, master protocol. Rather than employing a cognitive composite like in many other AD trials, a multivariate cognitive endpoint is used as the primary efficacy endpoint. This endpoint is analysed using a multivariate disease progression model with a proportional treatment effect. Prior to randomization, a run-in period is implemented to enrol trial ready participants and to assess primary endpoints. Dose escalation mechanism is planned to increase doses during the trial to prevent trial failure due to insufficient doses. Futility analysis is conducted at the prearranged interim analysis to allow stopping the trial early.
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Authors who are presenting talks have a * after their name.
