What is the
experimental treatment ER004?
What is ER004?
It is hoped ER004 will replace an important protein called EDA (Ectodysplasin-A) which is missing in those affected by XLHED. Absence of this protein during the baby’s development in the womb leads to some important structures not forming properly (sweat glands, hair, teeth etc). When given to XLHED-affected baby boys at the right time in the womb, ER004 should act as a replacement for the missing EDA and trigger the process that leads to the normal development of a baby’s skin, teeth, hair, and sweat glands, leading to better formation of these structures.
A few years ago, Edimer Pharmaceuticals, who previously developed the treatment, conducted a research study giving ER004 to affected newborn babies in the first fourteen days of life.
While ER004 did not lead to any major or significant side effects, it did not work on improving XLHED symptoms.
We now understand that replacing the missing EDA protein with ER004 after birth came too late as the development of the structures had already occurred.
Therefore, for the replacement ER004 to have a chance to work, it must be given during the development of the important structures (sweat glands, hair, teeth etc.), before birth.
How is ER004 given to your child in your womb?
We believe ER004 does not cross to the pregnant woman and you should therefore expect no benefits for your own health. During the treatment period, and up until 1 month after your baby’s birth, your health will be closely monitored alongside that of your baby.
Giving ER004 to your baby while still in the womb might sound scary. However, we think this is the only time at which the treatment might work.
Ectodysplasin under normal development
Ectodysplasin (EDA1) is missing in XLHED
ER004 as a replacement for Ectodysplasin (EDA1)
What are the possible side effects of the treatment?
So far, ER004 has not been found to have any major significant undesirable side effects, but it is a product in development and may therefore have unknown side effects.
You will be informed about this later on if you decide to participate in the study.
Safety of the participant is always our first concern in a research study, and your and your unborn son’s safety will be closely monitored for any untoward occurrences throughout your participation in the EDELIFE study.