Design and powering of cystic fibrosis clinical trials using rate of FEV(1) decline as an efficacy endpoint.
J Cyst Fibros
; 9(5): 332-8, 2010 Sep.
Article
em En
| MEDLINE
| ID: mdl-20646968
ABSTRACT
BACKGROUND:
Rate of lung function decline (RLFD) (as FEV(1) percent predicted/yr) is a robust measure of CF therapeutic efficacy rarely used as a study endpoint, in part due to uncertainty of sample size requirements.METHODS:
Sample size requirements for 11 randomizations to detect RLFD treatment effects from 20% to 80% were assessed in Epidemiologic Study of CF (ESCF) patients. Effects of measuring FEV(1) 1-4 times per year in studies of 1- to 4-year durations were assessed in 399 patients age ≥ 6 years with FEV(1) ≥ 70%. Impacts of inclusion/exclusion based on risk factors in 2369 ESCF patients were assessed over 1.5 years using semi-annual FEV(1) measures.RESULTS:
Increasing study duration and exclusion of lower risk patients (e.g., no P. aeruginosa infection) both substantially reduced requirements.CONCLUSIONS:
CF RLFD studies of 1.5 years in duration appear feasible provided that investigators account for the beneficial effects of subject inclusion/exclusion based on risk factors in power estimates.
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1
Base de dados:
MEDLINE
Assunto principal:
Projetos de Pesquisa
/
Volume Expiratório Forçado
/
Fibrose Cística
Idioma:
En
Ano de publicação:
2010
Tipo de documento:
Article