Design for the sacubitril/valsartan (LCZ696) compared with enalapril study of pediatric patients with heart failure due to systemic left ventricle systolic dysfunction (PANORAMA-HF study).
Am Heart J
; 193: 23-34, 2017 Nov.
Article
em En
| MEDLINE
| ID: mdl-29129252
ABSTRACT
BACKGROUND:
Sacubitril/valsartan (LCZ696) is an angiotensin receptor neprilysin inhibitor approved for the treatment of adult heart failure (HF); however, the benefit of sacubitril/valsartan in pediatric HF patients is unknown. STUDYDESIGN:
This global multi-center study will use an adaptive, seamless two-part design. Part 1 will assess the pharmacokinetics/pharmacodynamics of single ascending doses of sacubitril/valsartan in pediatric (1 month to <18 years) HF patients with systemic left ventricle and reduced left ventricular systolic function stratified into 3 age groups (Group 1 6 to <18 years; Group 2 1 to <6 years; Group 3 1 month to <1 year). Part 2 is a 52-week, efficacy and safety study where 360 eligible patients will be randomized to sacubitril/valsartan or enalapril. A novel global rank primary endpoint derived by ranking patients (worst-to-best outcome) based on clinical events such as death, initiation of mechanical life support, listing for urgent heart transplant, worsening HF, measures of functional capacity (NYHA/Ross scores), and patient-reported HF symptoms will be used to assess efficacy.CONCLUSION:
The PANORAMA-HF study, which will be the largest prospective pediatric HF trial conducted to date and the first to use a global rank primary endpoint, will determine whether sacubitril/valsartan is superior to enalapril for treatment of pediatric HF patients with reduced systemic left ventricular systolic function.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Tetrazóis
/
Enalapril
/
Função Ventricular Esquerda
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Isquemia Miocárdica
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Disfunção Ventricular Esquerda
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Valsartana
/
Aminobutiratos
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Insuficiência Cardíaca
Tipo de estudo:
Clinical_trials
/
Diagnostic_studies
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Etiology_studies
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Observational_studies
/
Prognostic_studies
/
Risk_factors_studies
Idioma:
En
Revista:
Am Heart J
Ano de publicação:
2017
Tipo de documento:
Article