Who can take
part in the study?
There are two different profiles of participants we are looking for in our EDELIFE study
The treated subject group and the comparator group will help us to better evaluate and quantify the effects of the treatment by comparing data collected from the boys who have received ER004 with the data collected from Ectodermal Dysplasia (XLHED) affected males who have not received ER004. If a male blood relative cannot be included in the study, we will use existing and previously collected data for the comparison.
1
The treated subjects group
2
The comparator group
If you are a pregnant woman expecting an Ectodermal Dysplasia (XLHED) affected boy
We need approximately 20 participants to take part in our study and who will be treated with ER004. This “treated subjects’ group” will be made up of pregnant women with a confirmed genetic diagnosis of XLHED, and who will be expecting a baby boy.
You are a pregnant woman (aged 18 or over), with a confirmed genetic diagnosis of XLHED, expecting a boy and your unborn son might be affected by XLHED.
As the treatment is to be given to your XLHED-affected son before he is born, you, as the pregnant woman, are being asked to participate in this study and agree to the treatment of your son whilst in your womb.
We will ask you for the contact details of an XHLED-affected male blood relative of yours who would be willing to participate in the comparator group. If you do not have this information or if your relative is unable to participate, this should not affect your participation in this study as comparator data may be obtained from other sources.
As the treatment is to be given to your XLHED-affected son before he is born, you, as the pregnant woman, are being asked to participate in this study and agree to the treatment of your son whilst in your womb.
We will ask you for the contact details of an XHLED-affected male blood relative of yours who would be willing to participate in the comparator group. If you do not have this information or if your relative is unable to participate, this should not affect your participation in this study as comparator data may be obtained from other sources.
What will participation in the study mean for you?
- The treatment will be given to your unborn son at pregnancy weeks 26, 28-29 and 31-32.
- No treatment will take place after he is born.
- Your son will remain in the study for follow up checks for 5 years after his birth, to understand the full effects of the treatment.
- This study will come on top of your normal pregnancy care and will require study-specific visits to the clinical centre.
If you are a male relative of the pregnant women
We also need 20 participants who are XLHED-affected male blood relatives of the pregnant women to form a “comparator group”. This group will not be treated with ER004.
You are an XLHED-affected male blood relative of a pregnant woman whose son will be treated in the study.
You will not receive any treatment if you decide to take part in this study.
However, your participation in the comparator group is crucial for the success of this study as we need to gather data from non-treated XLHED males to demonstrate whether or not the treatment has worked.
However, your participation in the comparator group is crucial for the success of this study as we need to gather data from non-treated XLHED males to demonstrate whether or not the treatment has worked.
What will participation in the study mean for you?
Your participation in the study is required for 1 visit only, to gather data on your XLHED symptoms.