Endpoints and patient stratification in clinical trials for alcoholic hepatitis.
ABIC
GAHS
Infection
MELD
Surrogate endpoints
Journal
Journal of hepatology
ISSN: 1600-0641
Titre abrégé: J Hepatol
Pays: Netherlands
ID NLM: 8503886
Informations de publication
Date de publication:
02 2019
02 2019
Historique:
received:
01
11
2018
revised:
07
11
2018
accepted:
08
11
2018
entrez:
20
1
2019
pubmed:
20
1
2019
medline:
20
6
2020
Statut:
ppublish
Résumé
In some areas of medicine the clinical development pathway through phase II and III clinical trials has been well mapped out and refined through extensive experience. In contrast, a number of key questions remain unanswered in the development of novel therapeutics for alcoholic hepatitis. The use of mortality as an endpoint in phase II clinical trials will potentially restrict the appeal of this therapeutic area for pharmaceutical companies, as the number of patients required for adequately powered clinical trials becomes impractical. Herein, we discuss alternative endpoints and conclude that dynamic assessment of liver function is the most pragmatic option in early stage studies. Stratification based on disease severity should be applied to avoid uneven distribution of patients with substantially differing mortality risks. Consensus on early phase trial design would help to facilitate new therapeutic development in this area of high unmet medical need.
Identifiants
pubmed: 30658732
pii: S0168-8278(18)32537-6
doi: 10.1016/j.jhep.2018.11.005
pii:
doi:
Substances chimiques
Adrenal Cortex Hormones
0
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
314-318Subventions
Organisme : Department of Health
ID : 08/14/44
Pays : United Kingdom
Organisme : Medical Research Council
ID : MR/R014019/1
Pays : United Kingdom
Informations de copyright
Copyright © 2018 European Association for the Study of the Liver. All rights reserved.