Your browser doesn't support javascript.
loading
Clinical endpoints and adaptive clinical trials in precirrhotic nonalcoholic steatohepatitis: Facilitating development approaches for an emerging epidemic.
Filozof, Claudia; Chow, Shein-Chung; Dimick-Santos, Lara; Chen, Yeh-Fong; Williams, Richard N; Goldstein, Barry J; Sanyal, Arun.
Afiliación
  • Filozof C; Covance Clinical Development Services Maidenhead United Kingdom.
  • Chow SC; Duke University School of Medicine Durham NC.
  • Dimick-Santos L; U.S. Food and Drug Administration Silver Spring MD.
  • Chen YF; U.S. Food and Drug Administration Silver Spring MD.
  • Williams RN; Covance, Inc., Clinical Development Services Princeton NJ.
  • Goldstein BJ; Covance, Inc., Clinical Development Services Princeton NJ.
  • Sanyal A; Virginia Commonwealth University School of Medicine Richmond VA.
Hepatol Commun ; 1(7): 577-585, 2017 09.
Article en En | MEDLINE | ID: mdl-29404480
Due to the increasing prevalence of nonalcoholic steatohepatitis (NASH) and its associated health burden, there is a high need to develop therapeutic strategies for patients with this disease. Unfortunately, its long and asymptomatic natural history, the uncertainties about disease progression, the fact that most patients are undiagnosed, and the requirement for sequential liver biopsies create substantial challenges for clinical development. Adaptive design methods are increasingly used in clinical research as they provide the flexibility and efficiency for identifying potential signals of clinical benefit of the test treatment under investigation and make prompt preplanned adaptations without undermining the validity or integrity of the trial. Given the high unmet medical need and the lack of validated surrogate endpoints in NASH, the use of adaptive design methods appears reasonable. Furthermore, due to the limited number of patients willing to have multiple liver biopsies and the need for long-term exposure to assess an impact in outcomes, a continuous seamless adaptive design may reduce the overall sample size while allowing patients to continue after each one of the phases. Here, we review strategic frameworks that include potential surrogate endpoints as well as statistical and logistical approaches that could be considered for applying adaptive designs to clinical trials in NASH with the goal of facilitating drug development for this growing medical need. (Hepatology Communications 2017;1:577-585).

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Prognostic_studies / Risk_factors_studies Idioma: En Revista: Hepatol Commun Año: 2017 Tipo del documento: Article Pais de publicación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Prognostic_studies / Risk_factors_studies Idioma: En Revista: Hepatol Commun Año: 2017 Tipo del documento: Article Pais de publicación: Estados Unidos