About the Edelife Trial
Prenatal Amniotic Infusion
Prenatal therapy is an emerging field for rare genetic diseases. XLHED is one of only a small number of rare diseases in the world with a prenatal therapeutic clinical trial currently enrolling.
Edelife is a phase 2, open label, international multicenter clinical trial of prenatal investigational treatment ER004. Edelife eligibility is specific to pregnant woman carriers of XLHED expecting a boy with XLHED. However, pregnant woman with confirmed or suspected hereditary ectodermal dysplasia (HED) unsure of their XLHED status may also be eligible for screening. Early patient referral is essential due to the early screening phase.
Edelife Trial Design1
- Must take place between pregnancy weeks 19-24
- Patient meets investigator at study site, reviews and signs informed consent
- Study screening process including ultrasound performed to confirm if baby in utero is an XLHED-affected boy
- Confirms patient study eligibility
- Must occur at 3 specific times between pregnancy weeks 26-32 (given 3 weeks apart)
- Patient receives in utero infusion of investigational ER004 therapy at each visit
- Additional monitoring and health assessments for the patient and baby
- Five years of follow-up, including several visits to the site
- No treatments administered after the birth of the child
- Evaluation of ER004 safety and efficacy
- Confirmation of overall health of child
Eligibility*
- Must be at least 18 years of age and enrolled before week 24 of their pregnancy
- Requires confirmed XLHED diagnosis with EDA gene mutation for the pregnant woman, this testing may be possible as part of screening visit
- Requires study-specific visits to a clinical trial site
- No cost to participate – this includes any clinical or travel expenses associated with the trial
Endpoints*
Edelife is designed to investigate the efficacy and safety profile of ER004 administered intraamniotically as a potential treatment for XLHED male subjects prior to birth. Study endpoints are as follows:
- Primary: Mean sweat volume in male XLHED subjects at 6 months of age as a measure of efficacy
- Secondary: multiple secondary endpoints including efficacy in male XLHED subjects based on mean sweat pore density, dentition, mean sweat volume, each at 6 months of age and other time points.
Administration
The trial involves infusions of the investigational medicine into the amniotic fluid at three time points approximately three weeks apart, between pregnancy weeks 26 and 32. As babies regularly swallow amniotic fluid during this stage of pregnancy, they will therefore swallow the investigational medicine along with the amniotic fluid.
Safety Profile
To date 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. Participant safety is always our first concern in a research study, and patient safety of both the mother and the child in utero will be closely monitored throughout. For more information on potential risks and benefits associated with the investigational medicine please see prior clinical publications and/or contact a study investigator.
NEXT STEPS: EDELIFE STUDY ENROLLMENT
