Your browser doesn't support javascript.
loading
Bayesian adaptive design for clinical trials in Duchenne muscular dystrophy.
Lake, Stephen L; Quintana, Melanie A; Broglio, Kristine; Panagoulias, Jennifer; Berry, Scott M; Panzara, Michael A.
Afiliação
  • Lake SL; Wave Life Sciences Ltd, Cambridge, Massachusetts.
  • Quintana MA; Berry Consultants LLC, Austin, Texas.
  • Broglio K; Berry Consultants LLC, Austin, Texas.
  • Panagoulias J; Wave Life Sciences Ltd, Cambridge, Massachusetts.
  • Berry SM; Berry Consultants LLC, Austin, Texas.
  • Panzara MA; Wave Life Sciences Ltd, Cambridge, Massachusetts.
Stat Med ; 40(19): 4167-4184, 2021 08 30.
Article em En | MEDLINE | ID: mdl-33960507
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.
Assuntos
Palavras-chave

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article