Bayesian adaptive design for clinical trials in Duchenne muscular dystrophy

被引：10

作者：

Lake, Stephen L. ^{[1
]}

Quintana, Melanie A. ^{[2
]}

Broglio, Kristine ^{[2
]}

Panagoulias, Jennifer ^{[1
]}

Berry, Scott M. ^{[2
]}

Panzara, Michael A. ^{[1
]}

机构：

[1] Wave Life Sci Ltd, Cambridge, MA 02138 USA

[2] Berry Consultants LLC, Austin, TX USA

来源：

STATISTICS IN MEDICINE | 2021年 / 40卷 / 19期

关键词：

adaptive design; Bayesian; clinical trial; clinical trial simulation; interim analysis; METAANALYSIS;

D O I：

10.1002/sim.9021

中图分类号：

Q [生物科学];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

A Bayesian adaptive design is proposed for a clinical trial in Duchenne muscular dystrophy. The trial was designed to demonstrate treatment efficacy on an ambulatory-based clinical endpoint and to identify early success on a biomarker (dystrophin protein levels) that can serve as a basis for accelerated approval in the United States. The trial incorporates placebo augmentation using placebo data from past clinical trials. A thorough simulation study was conducted to understand the operating characteristics of the trial. This trial design was selected for the US FDA Complex Innovative Trial Design Pilot Meeting Program and the experience in that program is summarized.

引用

页码：4167 / 4184

页数：18

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