Bayesian adaptive design for clinical trials in Duchenne muscular dystrophy.
Stat Med
; 40(19): 4167-4184, 2021 08 30.
Article
en 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.
Palabras clave
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Distrofia Muscular de Duchenne
Tipo de estudio:
Clinical_trials
/
Prognostic_studies
Límite:
Humans
Idioma:
En
Revista:
Stat Med
Año:
2021
Tipo del documento:
Article