The EspeRare Foundation and the Pierre Fabre group announced today that the first patient in the EDELIFE clinical trial has received the three rounds of injection as planned.
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EDELIFE Phase II trial explores potential of pioneering prenatal treatment for XLHED GENEVA and Castres, France, July 1, 2022 /PRNewswire/ --The EspeRare Foundation and the Pierre Fabre group announced today that the first patient in the EDELIFE clinical trial has received the three rounds of injection as planned. The pivotal trial is the first-of-its-kind, as the treatment is given to the XLHED (X-linked Hypohidrotic Ectodermal Dysplasia)-affected baby while inside his mother's womb. The trial intends to confirm the efficacy and safety of ER004 given as a pre-natal treatment to XLHED-affected boys.
XLHED is a very rare and debilitating congenital disease which affects approximately 4/100,000 live male births every year. This genetic disorder is a dermatologic-related condition which leads to abnormal development of the skin, sweat glands, sebaceous glands, hair, oral cavity and respiratory mucosal glands resulting in serious clinical manifestations such as hyperthermia, craniofacial anomalies and recurrent respiratory infections. The EDELIFE clinical trial is a confirmatory study that will investigate the efficacy and safety of intra-amniotic ER004 administrations as a prenatal treatment for male foetuses who have been confirmed to have XLHED. ER004 is a pioneering in-utero therapy designed to replace the function of endogenous Ectodysplasin A1 (EDA1), a protein key to the normal development of ectodermal structures in the foetus. If successful, ER004 has the potential to become the first approved 'single-course treatment', administered during the second and third trimesters of pregnancy. "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," said Caroline Kant, the Co-founder and CEO of the EspeRare Foundation. "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." A target number of 15 to 20 baby boys with XLHED will be treated within the EDELIFE trial across 8 centres in Europe and the USA. Clinical centres for the study are now open in Germany, France, Italy, Spain, the United Kingdom and will be open shortly in the USA. A dedicated web site (www.EDELIFEclinicaltrial.com) is available for interested families who want to learn more about the clinical trial and the conditions for enrolment. Beyond the initial English version, it will progressively be developed in German, French, Italian and Spanish. "We are committed to supporting pregnant women with a confirmed diagnosis of XLHED in their treatment journey for their unborn baby boys," said Dr Deborah Szafir, Executive Vice President, Head of Medical and Patient Consumer Division at Pierre Fabre. "15 to 20 patients in the clinical trial may seem like a very small number, but given how rare the condition is, it is essential we facilitate the process of enrolment into the study as much as possible. This includes facilitating the pregnant women's travel to a nearby country if there is no investigational site open in their own country. We feel very proud at Pierre Fabre to support those with rare dermatologic diseases as we have already successfully done in infantile hemangiomas". In the main study phase of the EDELIFE clinical trial, efficacy and safety of approximately 15 treated children will be assessed up to 6 months of age and safety of the mothers will be assessed up to 1 month after delivery. In the long-term follow-up phase, efficacy and safety of the treated children will be assessed up to 5 years of age. The main phase of the clinical study is expected to last until 2025. The ER004 in XLHED program has received a wide range of regulatory support, including Breakthrough Therapy Designation in 2020 by the US Federal Drug Administration (FDA). Its clinical development also benefits from the European Medical Agency's (EMA) PRIME (PRIority MEdicines) program. Details also available on www.clinicaltrials.gov. Study NCT04980638 About XLHED About ER004 About the EspeRare foundation About Pierre Fabre References
Photo - https://mma.prnewswire.com/media/1851585/edelife.jpg CONTACTS : Pierre Fabre
SOURCE Pierre Fabre, EspeRare Foundation |
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