LCZ696 vs Enalapril in Paediatric Patients with Heart Failure
Multicenter, open-label study to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of LCZ696 followed by a 52-week randomized, double-blind, parallel group, active-controlled study to evaluate the efficacy and safety of LCZ696 compared with enalapril in pediatric patients from 1 month to <18 years of age with heart failure due to systemic left ventricle systolic dysfunction
Novartis Pharma Services AG
PIP02-11-M02, Paediatric Investigational Plan
Duration of Study in the UK
4 years, 3 months, 20 days
Paediatric Heart Failure is characterized by significant morbidity and mortality, frequent hospitalisation and medical care, and poor quality of life.
Many paediatric patients with severe HF are usually listed for heart transplant if available, however, cardiac transplantation is usually a last resort given the limited availability of donor organs, complicated clinical course management and associated morbidity and mortality.
There is limited research into paediatric HF and treatment of HF in children is based on information provided by adult studies.
This study focuses on a subset of paediatric HF patients with systemic left ventricular systolic dysfunction to allow a more homogenous population with a pathophysiology similar to adult reduced ejection fraction heart failure. Studies have shown LCZ696 to be efficacious in this adult population and is licenced in the UK.
This study is in two parts:
Part 1 is an open-label pharmacokinetic and pharmacodynamics study to confirm the dose for part 2.
Part 2 is a double-blind, randomised, trial comparing the safety and efficacy of 52-weeks of treatment of LCZ696 vs enalapril.
London - Riverside Research Ethics Committee
Date of REC Opinion
22 Dec 2016
Further Information Favourable Opinion