For Healthcare Professionals Only
About the Edelife Trial
Prenatal Amniotic Infusion
Prenatal therapy is an exciting 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.
Edelife Trial Design1
Participation in Edelife will require visits to a clinical study site. Pregnant women must be at least 18 years of age and enrolled before week 24 of pregnancy.
- 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
- 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*
Pregnant women may be eligible for Edelife if they meet the following criteria:
- 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 of ER004 administered intraamniotically as a treatment for XLHED male subjects prior to birth. Study endpoints are as follows:
- Primary: assess efficacy based on mean sweat volume in male XLHED subjects at 6 months of age
- 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 investiga- tional 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 associ- ated with the investigational medicine please see prior clinical publications and/or contact a study investigator.
NEXT STEPS: EDELIFE STUDY ENROLLMENT
Early referral is essential. Please help ensure pregnant women with confirmed or suspected XLHED are aware of the Edelife trial. Contact an Edelife clinical site to refer a patient or request more information.
*For a full list of Eligibility Criteria and Outcome Measures please see https://clinicaltrials.gov/ct2/- show/NCT04980638
References:
1. Schneider, H.; Hadj-Rabia, S.; Faschingbauer, F.; Bodemer, C.; Grange, D.K.; Norton, M.E.; Cavalli, R.; Tadini, G.; Stepan, H.; Clarke, A.; et al. Protocol for the Phase 2 EDELIFE Trial Investigating the Efficacy and Safety of Intra-Amniotic ER004 Administration to Male Subjects with X-Linked Hypohidrotic Ectodermal Dysplasia. Genes 2023, 14, 153
