LENA stands for Labeling of Enalapril from Neonates up to Adolescents
The goal of this study is to develop a safe, effective and age appropriate formulation of a drug called Enalapril. Enalapril is an ACE inhibitor (or angiotensin-converting-enzyme inhibitor) that was initially used to treat hypertension (high blood pressure) in adults. It was only later on that it was discovered that Enalapril improves the performance of the heart tissue, thereby allowing the heart to work slower and more efficiently. This discovery suggested that Enalapril may also help children suffering from heart failure by reducing the stress of the heart.
Few medicines currently exist in formulations which are specifically developed for children. Enalapril is currently only licensed for paediatric patients above 20 kg, which is on average equal to the body weight of a six year old child. In contrast to adults, Enalapril is only licensed to treat hypertension in children. Enalapril is currently used as an “off-label” drug in children of all ages suffering from heart disease.
Heart failure in children, however, is a completely different disease compared to high blood pressure found in adults. Using Enalapril as an “off-label” drug in children with heart failure means that Enalapril’s effects have not yet been systematically studied in children and the drug is therefore not yet licensed for use in children.
Why do we use Enalapril if it is not licensed for children suffering from heart failure? Enalapril is currently used in children of all ages with heart disease because there is no other long lasting ACE inhibitor currently on the market that is licensed. Enalapril has been specifically developed for adults; therefore no child-size dosage form currently exists. Without having a standardized dosage available for children, it has been impossible to study the safety and the effects of this drug in children suffering from heart failure.
Children suffering from heart failure have a very different disease compared to adults, it is therefore essential to study Enalapril in children with heart conditions to optimize their treatment and to develop medicines specifically for these children’s needs.
At present, Enalapril tablets first have to be crushed then diluted in water before they can be given to children. This is a very inaccurate and difficult preparation method and can put patients at risk by either under or over dosing the medicine. To overcome this problem, orally disintegrating mini-tablets (ODMTs) have been developed. These mini-tablets are very small tablets (less than 3mm) and allow administering the correct dosage of the active ingredients (the drug). The mini-tablets will quickly dissolve in the mouth, thereby allowing to precisely determine the dosage for children of all ages.
Once the LENA studies have been successfully completed, we hope that Enalapril mini-tablets will be licensed for children from neonates to adolescents, allowing the safe and effective treatment for children with heart problems. This study marks a first step in developing additional medicines in the form of mini-tablets which will ultimately help tailor drug treatments to the specific needs of young patients.