Pierre Fabre group and the EspeRare Foundation have administered investigational in-utero therapy, ER004, to first participant in the EDELIFE clinical trial for the treatment of X-linked Hypohidrotic Ectodermal Dysplasia (XLHED).
ER004 is a recombinant, soluble, and humanised form of the endogenous Ectodysplasin A1 (EDA1), a key protein given for the normal development of ectodermal structures in the foetus.
It has been designed to replace the function of EDA1 that is given as a single course treatment through intra-amniotic injections during the late stage of pregnancy.
The confirmatory study has been designed to evaluate the safety and efficacy of intra-amniotic ER004 to treat male foetuses with confirmed XLHED inside the mother’s womb.
About 15 to 20 baby boys with XLHED will receive treatment in the EDELIFE clinical trial across eight centres in the US and Europe.
At present, clinical centres for the study are open in France, the UK, Germany, Italy, Spain, with centres in the US planned to be open shortly.
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The EspeRare Foundation co-founder and CEO Caroline Kant said: “It is an exciting time for the Hypohidrotic Ectodermal Dysplasia community and for prenatal medical research. For the first time, we can hope to correct major symptoms of XLHED for those who are diagnosed before birth.
“We need the support of the XLHED community to ensure that those who may benefit from this unique treatment are aware of the EDELIFE trial.”
About 15 children treated with ER004 will be evaluated up to six months of age, and safety of the mothers will be assessed up to one month after delivery in the main study phase of the EDELIFE clinical trial.
The treated children’s efficacy and safety will be assessed up to five years of age in the long-term follow-up phase.
The clinical study’s main phase is expected to last until 2025.