1. Orodispersible minitablets of enalapril for use in children with heart failure (LENA): Rationale and protocol for a multicentre pharmacokinetic bridging study and follow-up safety study
- Author
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Bajcetic, Milica, de Wildt, Saskia N., Dalinghaus, Michiel, Breitkreutz, Jörg, Klingmann, Ingrid, Lagler, Florian B., Keatley-Clarke, Anne, Breur, Johannes MPJ., Male, Christoph, Jovanovic, Ida, Szatmári, Andras, Ablonczy, László, Burckhardt, Bjoern B., Cawello, Willi, Kleine, Karl, Obarcanin, Emina, Spatenkova, Lucie, Swoboda, Vanessa, van der Meulen, Marijke, Wagner, Peter, Walsh, Jennifer, and Läer, Stephanie
- Abstract
Treatment of paediatric heart failure is based on paradigms extensively tested in the adult population assuming similar underlying pathophysiological mechanisms. Angiotensin converting enzyme inhibitors (ACEI) like enalapril are one of the cornerstones of treatment and commonly used off-label in children. Dose recommendations have been extrapolated from adult experience, but the relationship between dose and pharmacokinetics (PK) in (young) children is insufficiently studied. Furthermore, appropriate paediatric formulations are lacking. Within the European collaborative project LENA, a novel formulation of enalapril orodispersible minitablets (ODMT), suitable for paediatric administration, will be tested in (young) children with heart failure due to either dilated cardiomyopathy or congenital heart disease in two pharmacokinetic bridging studies. Paediatric PK data of enalapril and its active metabolite enalaprilat will be obtained. In a follow-up study, the safety of enalapril ODMTs will be demonstrated in patients on long-term treatment of up to 10 months. Furthermore, additional information about pharmacodynamics (PD) and ODMT acceptability will be collected in all three studies.
- Published
- 2019
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