269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy - What have we learned? 9-11 December 2022, Hoofddorp, The Netherlands.
Clinical trial
Duchenne muscular dystrophy
Meaningfulness
Outcome measure
Real world data
Journal
Neuromuscular disorders : NMD
ISSN: 1873-2364
Titre abrégé: Neuromuscul Disord
Pays: England
ID NLM: 9111470
Informations de publication
Date de publication:
Nov 2023
Nov 2023
Historique:
received:
30
09
2023
accepted:
10
10
2023
medline:
4
12
2023
pubmed:
6
11
2023
entrez:
5
11
2023
Statut:
ppublish
Résumé
There are multiple avenues for therapeutic development in Duchenne muscular dystrophy (DMD), which are highlighted in the first section of this report for the "10 years of Clinical trials in DMD - What have we learned?" workshop. This report then provides an overview of the presentations made at the workshop grouped into the following core themes: trial outcomes, disease heterogeneity, meaningfulness of outcomes and the utility of real-world data in trials. Finally, we present the consensus that was achieved at the workshop on the learning points from 10 years of clinical trials in DMD, and possible action points from these. This includes further work in expanding the scope and range of trial outcomes and assessing the efficacy of new trial structures for DMD. We also highlight several points which should be addressed during future interactions with regulators, such as clinical meaningfulness and the use of real-world data.
Identifiants
pubmed: 37926638
pii: S0960-8966(23)00748-4
doi: 10.1016/j.nmd.2023.10.003
pii:
doi:
Types de publication
Clinical Conference
Langues
eng
Sous-ensembles de citation
IM
Pagination
897-910Informations de copyright
Copyright © 2023.
Déclaration de conflit d'intérêts
Declaration of Competing Interest None.