Methodological Quality of Clinical Trials in Amyotrophic Lateral Sclerosis: A Systematic Review.
J Neuromuscul Dis
; 11(4): 749-765, 2024.
Article
em En
| MEDLINE
| ID: mdl-38759021
ABSTRACT
Background:
More than 200 clinical trials have been performed worldwide in ALS so far, but no agents with substantial efficacy on disease progression have been found.Objective:
To describe the methodological quality of all clinical trials performed in ALS and published before December 31, 2022.Methods:
We conducted a systematic review following the Preferred Reporting Items for Systematic Reviews and Meta Analyses.Results:
213 trials were included. 47.4% manuscripts described preclinical study evaluation, with a positive effect in all. 67.6% of trials were conducted with a parallel-arm design, while 12.7% were cross-over studies; 77% were randomized, while in 5.6% historical-controls were used for comparison. 70% of trials were double blind. Participant inclusion allowed forced vital capacity (or corresponding slow vital capacity)<50% in 15% cases, between 55-65% in 21.6%, between 70-80% in 14.1% reports, and 49.3% of the evaluated manuscripts did not provide a minimum value for respiratory capacity at inclusion. Disease duration was <â6-months in 6 studies, 7-36 months in 68, 37-60 months in 24, 8 trials requested more than 1-month of disease duration, while in 107 reports a disease duration was not described. Dropout rate was ≥20% in 30.5% trials, while it was not reported for 8.5%.Conclusion:
The methodological quality of the included studies was highly variable. Major issues to be addressed in future ALS clinical trials include the requirement for standard animal toxicology and phase I studies, the resource-intensive nature of phase II-III studies, adequate study methodology and design, a good results reporting.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Projetos de Pesquisa
/
Ensaios Clínicos como Assunto
/
Esclerose Lateral Amiotrófica
Limite:
Humans
Idioma:
En
Revista:
J Neuromuscul Dis
Ano de publicação:
2024
Tipo de documento:
Article
País de afiliação:
Itália