recombinant ectodysplasin A1 (APO200) / Valerio Therap, Pierre Fabre - LARVOL DELTA

EDELIFE: Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia (clinicaltrials.gov) - P2 | N=20 | Recruiting | Sponsor: EspeRare Foundation | Trial completion date: Jan 2029 ➔ Dec 2032 | Trial primary completion date: Apr 2025 ➔ Feb 2027

Trial completion date • Trial primary completion date

EDELIFE: Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia (clinicaltrials.gov) - P2 | N=20 | Recruiting | Sponsor: EspeRare Foundation | Trial primary completion date: Jul 2024 ➔ Apr 2025

Trial primary completion date

Janssen’s ’JNJ-42756493’ remains the most administered to cancer patients without a cure [Google translation] (News The Voice Healthcare) - "Although no medicines have been approved in the past three quarters, how many medicines have been administered to treat patients under the judgment of medical staff? According to the Ministry of Food and Drug Safety's approval status for therapeutic purposes in the third quarter...Next, Big Think's Nerlinx tablet, 'neratinib 40mg', was administered to HER2-positive metastatic breast cancer patients in 9 cases....Eisai's thyroid cancer treatment drug 'Lenvima' was administered to three patients with advanced renal cell carcinoma. Dawonmedax's DMX-101 (DM-BTPS, DM-BNCT) was also administered to three patients with recurrent glioblastoma for treatment."

Commercial • Brain Cancer • Breast Cancer • CNS Tumor • Genito-urinary Cancer • Glioblastoma • HER2 Positive Breast Cancer • Kidney Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor

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. (PubMed, Genes (Basel)) - "In order to confirm these results, we started the EDELIFE trial, a prospective, open-label, genotype-match controlled, multicentre clinical study to investigate the efficacy and safety of intra-amniotic ER004 administration as a prenatal treatment for male subjects with XLHED. This article summarises the rationale, the study protocol, ethical issues of the trial, and potential pitfalls."

Journal • P2 data • Genetic Disorders • Infectious Disease • Respiratory Diseases • NF-κβ

EDELIFE: Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia (clinicaltrials.gov) - P2 | N=20 | Recruiting | Sponsor: EspeRare Foundation | Trial completion date: Apr 2029 ➔ Jan 2029 | Trial primary completion date: Jun 2023 ➔ Jul 2024

Trial completion date • Trial primary completion date

EDELIFE: Intraamniotic Administrations of ER004 in Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia (clinicaltrials.gov) - P2; N=20; Recruiting; Sponsor: EspeRare Foundation; Not yet recruiting ➔ Recruiting; Initiation date: Oct 2021 ➔ Feb 2022

Enrollment open • Trial initiation date

An open-label Phase 2 trial to investigate efficacy and safety of intra-amniotic administrations of ER004 in male subjects with X-linked hypohidrotic ectodermal dysplasia (XLHED) (clinicaltrialsregister.eu) - P2/3; N=20; Ongoing; Sponsor: EspeRare Foundation`Pierre Fabre Médicament

Clinical • New P2/3 trial

EDELIFE: Intraamniotic Administrations of ER004 in Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia (clinicaltrials.gov) - P2; N=20; Not yet recruiting; Sponsor: EspeRare Foundation

New P2 trial

Prenatal Treatment of X-Linked Hypohidrotic Ectodermal Dysplasia using Recombinant Ectodysplasin in a Canine Model. (PubMed, J Pharmacol Exp Ther) - "The results suggested that a single or two temporally spaced injections of EDI200 prenatally might improve the clinical outcome in the dog model...These results exemplify the feasibility of ultrasound-guided intra-amniotic injections for treatment of developmental disorders, with improved formation of specific EDA1-dependent structures in dogs with XLHED. SIGNIFICANCE STATEMENT: N/A."

Journal