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About the LENA Study - Frequently Asked Questions

 

 

 

 

How does Enalapril work?

Enalapril is currently used in children who suffer from high blood pressure (hypertension) or are at risk of heart failure. Enalapril belongs to a group of drugs which are called ACE inhibitors, which allow the blood vessels to expand, thereby lowering the blood pressure. They also directly help the heart tissue to perform better and work more efficiently.

 


ACE inhibitors work by stopping the production of a hormone called angiotensin II. The kidneys can detect a fall in blood pressure and can regulate the blood pressure by allowing the production of angiotensin II, which causes the narrowing of the blood vessels, leading to high blood pressure. Thereby by giving someone an ACE inhibitor this will stop the production of the angiotensin II hormone and lower the blood pressure. Angiotensin II itself works directly on the heart tissue and helps the heart tissue remain in its biomechanical structure to contract efficiently. This effect on the heart tissue is well investigated in adults but not in children. Since the heart tissue is assumed to mature substantially still after birth and even within the first years of life, the effect of an ACE inhibitor on the maturation of the heart tissue cannot be excluded and needs to be studied.

 

 

 

 

Why study Enalapril in children?

The majority of medicines prescribed in hospitals to children are not licensed for children. It is therefore crucial to perform research to improve the health of children and their quality of life in the future by developing medicines specifically formulated for children.

 

Since Enalapril is currently only licensed for adults, it is necessary to study this drug in children to fully understand how this drug interacts with a child’s body. Children are not small adults, their bodies respond to medication differently than an adult body would. For example, congenital heart defects in children are very different from heart problems an adult may encounter.

 

It is therefore very important to understand how a child’s body reacts to Enalapril and we therefore need to study the pharmacokinetics and also the pharmacodynamics of this drug in children. Pharmacokinetics is the rate at which the drug is absorbed, distributed, metabolised and excreted. Understanding this process will allow the optimisation of treatment to ensure that children will receive just the right amount of the drug they need. Pharmacodynamics concerns how a drug works within an organism. Understanding this process will allow us to recognize the possible benefits of using Enalapril for a range of heart diseases in the paediatric population.

 

Another factor that has to be evaluated in this study is that the adult formulation of Enalapril is swallowed as a tablet compared to the newly developed mini-tablets. The mini-tablets dissolve within seconds in the child´s mouth and can then be swallowed without the danger of sticking anywhere in the mouth, throat or oesophagus. To make sure that Enalapril is working similarly as the adult formulation, it is necessary to study the buccal (mouth) versus gastric (stomach) absorption rates, to better understand the amount of drug reaching the body via the stomach. By studying the amount of the drug reaching the body, we understand the bioavailability of the drug and can  predict how much of the drug needs to be taken. 

 

 

 

 

 

 

Why has my child been invited to take part in the LENA study?

Children at any age with dilated cardiomyopathy and/or left ventricular dysfunction (LVD) who are eligible to receive an ACE inhibitor (in addition to standard therapy) are invited to this study.  

 

 

 

 

Who is funding this study?

This study is funded by the European Commission and was started at the end of 2013 and is expected to be completed by 2018. The European Medicines Agency (EMA) has agreed that there is an urgent need to have a safe formulation of Enalapril available to children.  An institutional review board (IRB) has reviewed this study and additionally an independent ethics committee has approved this study.

 

 

 

Will my child still get the best treatment?

Since your child is required to take an ACE inhibitor, taking part in this study will allow your child to receive an ACE inhibitor that has been especially formulated for children.


Previous studies have shown that the mini-tablets are well tolerated in the adult population and are very similar to the adult version.  The new mini-tablets allow to precisely dose the active ingredient, which is currently not possible with the adult version of Enalapril.


To ensure the safety of the mini-tablets, your child will be very closely monitored to make certain that the new formulation is well tolerated.


Your child will therefore receive essentially the same drug with an improved formulation that allows administering this drug more easily to children of all ages.

  

 

 

 

What will happen if my child takes part in this study?

If your child decides to take part in this study, your child will be provided with the mini-tablets, instead of the adult formulation.


Your child will be monitored closely and blood samples will be taken to ensure your child is reacting well to the new formulation. We will also use blood samples from your child to better understand how children will react to Enalapril and how their organs handle the Enalapril differently to adults.


Blood samples will be kept to a minimum and it will be ensured that only phlebotomists and physicians with sufficient expertise are involved in taking blood samples from your child.


We will ensure that we will only take as much blood, as it is ethically adequate. The assays used in the LENA trials are child-like assays and are specifically developed to get the maximum information out of this minimal blood volume taken from the child. Therefore no more blood than would fit in a teaspoon will be taken at a time. 

 

 

 

 

What are the benefits and risks in participating in the LENA study?

Children can only take part in studies where the benefits outweigh the risks and no serious risk to their health is involved. When participating in a study is important to consider all the risks and consequences.

 

Benefits?

Risks?

  • Your child will receive a drug that is formulated  specifically for children, which may be safer and more accurate to administer than the adult version
  • International experts will work together to develop the trial protocol
  • Patient safety comes first, this will ensure that you or your child are monitored more closely than usual
  • You are helping children to get better treatment in the future

 

 

 

  • You may have more tests carried out during your on-going contact with the LENA project
  • The new treatment may prove not to be better than your current medicine

 

What are my child's rights if s/he takes part in this study?

In order for you to sign the consent form it is essential that you feel that you understand what this study is about and have also been told about the possible outcomes. Remember you can leave this study at any time.

 

For an act of informed consent to be considered valid, three criteria must be met:

 

     (a) the person giving consent should be competent to do so,
     (b) giving consent voluntarily without feeling pressured
     (c) and the person giving consent is adequately informed when doing so.

 

 

 

 

What are the ethical guidelines?

This study has been approved by an ethics committee, which has considered the ethical issues of conducting a study in children.

 

The key ethical issues are:

 

  • Autonomy –  It is your own choice to take part in the study
  • Beneficence – We believe that this study is in the best interests of the child
  • Non-maleficence – We believe that the benefits outweigh any risks
  • Justice – Everyone in this study will be treated with fairness and equality

 

 

 

 

 

Should my child take part in this study?

In order to make this decision your family and your child should make sure they feel that they have all the necessary information to make an informed decision. Here are some questions that can help you to make this decision.

 

All these questions should be answered with a ‘yes’ to indicate that you are well informed and feel comfortable to take part in this study.

 

  • Do you feel comfortable talking to medical staff about this study?
  • Does your child feel comfortable speaking to staff and asking questions?
  • Do you feel all your questions are being answered?
  • Do you understand the benefits/risks of this study?
  • Are you aware that there is no obligation to complete the study?
  • Do you feel that no one is pressuring you to take part in this study?
  • Are you aware that Enalapril has been studied in adults, but not in children?
  • Do you know that if you do not feel comfortable to take part in this study, your choice will be respected.
  • If you or your child refuses to take part in this study, will his/her opinion will be respected?

 

 

 

 

Tips to remember

  • Make sure to write down any questions you may want to ask later on, you may forget them by the time you have a chance to ask.
  • Don’t be afraid to ask any questions.
  • If you don’t understand something, make sure to ask again.
  • Make sure your child understands the benefits/risks of this study.
  • Speak to your family and friends about this study.
  • Bring a family member or a friend along, sometimes it is good to have someone there who can help to remember all the information
  • If you feel you are not treated with respect or are uncomfortable about a situation, please let us know as soon as possible.  Your view is vital to us.

  

 

 

 

The LENA team of experts

The LENA team of experts

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