REGENERATE: Design of a pivotal, randomised, phase 3 study evaluating the safety and efficacy of obeticholic acid in patients with fibrosis due to nonalcoholic steatohepatitis.
Contemp Clin Trials
; 84: 105803, 2019 09.
Article
em En
| MEDLINE
| ID: mdl-31260793
ABSTRACT
BACKGROUND:
Nonalcoholic steatohepatitis (NASH) is a chronic, progressive, and severe form of nonalcoholic fatty liver disease. In FLINT, obeticholic acid (OCA) treatment improved multiple histological NASH features. The design and endpoints of REGENERATE, an ongoing phase 3 study, further evaluate OCA treatment in patients with fibrosis due to NASH.AIMS:
The Month 18 interim analysis assesses the effect of OCA on liver histology, defined as improvement of fibrosis by ≥1 stage with no worsening of NASH or resolution of NASH with no worsening of fibrosis. The end-of-study analyses evaluate the effect of OCA on mortality, liver-related clinical outcomes, and long-term safety.METHODS:
REGENERATE is a pivotal, long-term study of ~2400 patients with NASH, including ~2100 patients with stage 2 or 3 liver fibrosis. Additionally, ~300 patients with stage 1 fibrosis andâ¯≥1 accompanying comorbidity are included to gather information on the safety of OCA and liver disease progression. Patients are randomised 111 to receive placebo or OCA (10 or 25â¯mg). A liver biopsy evaluation occurs at screening, Months 18 and 48, and end of study. The duration of the study is dependent upon accrual of a predetermined number of clinical outcome events.CONCLUSIONS:
REGENERATE is designed in conjunction with regulatory authorities to support regulatory approvals in NASH. This robust phase 3 study assesses the effect of OCA on liver histology as a surrogate for transplant-free survival and liver-related outcomes, including progression to cirrhosis and mortality, and will ultimately assess clinical benefit through specific evaluation of these outcomes. CLINICAL TRIAL REGISTRATION ClinicalTrials.gov with the identifier NCT02548351.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Ácido Quenodesoxicólico
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Hepatopatia Gordurosa não Alcoólica
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Cirrose Hepática
Tipo de estudo:
Clinical_trials
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Observational_studies
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Risk_factors_studies
Limite:
Humans
Idioma:
En
Revista:
Contemp Clin Trials
Assunto da revista:
MEDICINA
/
TERAPEUTICA
Ano de publicação:
2019
Tipo de documento:
Article