About the

Edelife Study

There is currently no curative treatment for Ectodermal Dysplasia (XLHED). Edelife is a study of intra-uterine administration of an investigational medicine designed to replace a protein that is missing in those affected by XLHED. If successful, this study could lead to an approved treatment for the condition.
information about Edelife study design


Pregnant women who are considering taking part in the study may consider the following:

  • Pregnant women who are expecting a baby boy will take part in the study and be treated with the investigational medicine.
  • Knowing that you may have XLHED and that you are pregnant with a boy is sufficient information to consider taking part in the study.
  • Pregnant women who live in a country where there is no study center may be able to take part, and are strongly encouraged to reach out to the study doctors in the contact section.
information about Edelife study design

Potential Benefits and Study Objectives

Previously, six XLHED-affected boys received the investigational medicine before birth and the results indicated the potential to prevent or at least significantly reduce the severity of some XLHED symptoms. These were reported in scientific journals in 2018 and 2023.

We now want to confirm these findings in the Edelife study, with two aims:

  • To learn if prenatal treatment with the investigational medicine is well tolerated.
  • To learn if prenatal treatment with the investigational medicine can lead to a long-lasting improvement of some symptoms in XLHED-affected boys.
information about Edelife study design

How the Medicine is Given

If a pregnant woman is eligible, a doctor will infuse the investigational medicine into the water (amniotic fluid) around the baby at three time points between pregnancy weeks 26 and 32. During this stage of pregnancy, babies regularly swallow this fluid, and will therefore swallow the investigational medicine at the same time.

information about Edelife study design

Possible Side Effects

So far, the investigational medicine has not been found to have any major significant side effects, but it is a product in development and may have unknown side effects.  Safety of the participant is always our first concern in a research study, and you and your child’s safety will be closely monitored throughout your participation in the Edelife study. If you would like more information on the potential risks and benefits associated with the investigational medicine, please ask a study doctor.

Study Design

Participating in Edelife will require study-specific visits to a clinical center. Pregnant women must be at least 18 years of age and enrolled before week 23 of their pregnancy.

Screening, Treatment and Follow-up
  • Must take place between pregnancy weeks 19-24
  • Discuss Edelife in person with the study doctor
  • Includes specific ultrasound to confirm if baby is a boy with XLHED
  • Confirms whether you are eligible for Edelife
  • Must occur at 3 specific times from pregnancy weeks 26-32
  • Health checks for you and your baby
  • Receive an infusion of the investigational medicine at each visit
  • There is no treatment after your baby is born
  • Five years of follow-up after birth for your son
  • Evaluate if the investigational medicine is working, check any XLHED symptoms
  • Confirm overall health of child

Next Steps

If you are interested in taking part in the study or you have questions, please contact a Study Doctor.

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