Pierre Fabre

Please help support EDVYCE by answering this SPONSOR SURVEY QUESTION.

PRIZES:

The most thoughtful responses as judged by the sponsor will receive a US-based Amazon gift card of $100 (one prize).

All other respondents will be entered in a raffle to receive one of 5 additional $10 US-based Amazon gift cards.

Prizes will be awarded within 2-4 weeks following the deadline date.

DEADLINE:

July 31, 2023

CLICK ON YOUR CHOICE AND

PLEASE ADD ANY ADDITIONAL CLARIFICATION

OR SUGGESTIONS IN THE COMMENTS BELOW

What actions would you take in order to recruit women pregnant with X-linked hypohidrotic ectodermal dysplasia males into a clinical trial?

A.    Reach out to a particular center or physician (please clarify in Comments)

B.    Partner with a particular association, specialist group, or business excluding the NFED which study is currently partnered  with. (please clarify in Comments)

C.    Other suggestions (please clarify in Comments)

ENTER YOUR CHOICE AND ANY ADDITIONAL SUGGESTIONS IN THE COMMENTS SECTION BELOW

BACKGROUND

The EspeRare Foundation and Pierre Fabre are developing a prenatal treatment for X-linked Hypohidrotic Ectodermal Dysplasia (XLHED), ER004.

We are recruiting pregnant women, no later than week 23+6, EDA mutation / XLHED carrier (clinically or genetically confirmed).

The fetal subject must be male confirmed via ultrasound or genetic testing.  

ER004 is a biotechnology-derived, biologically active, and fully humanized form of functional EDA1. ER004 is a first-in-class signaling protein replacement molecule designed for specific, high-affinity binding to the endogenous EDA1 receptor (EDAR). The proposed mechanism of action of ER004 is the replacement of the missing or nonfunctional EDA1 protein in patients with XLHED.

The aim of ER004 therapy in XLHED patients is to activate the EDA1 signaling pathway within a timeframe that will trigger the normal development of structures derived from the embryonic ectoderm and thereby alleviate the phenotype of XLHED. 

Results from 6 named-patient use case studies in XLHED patients with EDA null mutations, treated by intra-amniotic injections of ER004 during the late second and third trimesters of pregnancy, indicate long-term improvement of several key parameters of XLHED (sweat glands, teeth, Meibomian glands, and salivation) (results from the 3 first cases are described in: Schneider et al., 2018).Please see additional details at:

ClinicalTrials.gov link: https://www.clinicaltrials.gov/ct2/show/NCT04980638?term=edelife&draw=2&rank=1Trial Website: https://edelifeclinicaltrial.com/ and https://edelifeclinicaltrial.com/resources

To reach the study coordinators with any questions: contact.edelife@pierre-fabre.com