A Prospective, Open-label, Genotype-match Controlled, Multicenter Clinical Trial to Investigate the Efficacy and Safety of Intra-amniotic ER004 as a Prenatal Treatment for Male Subjects With XLHED
Who is this study for? Males with x-linked hypohidrotic ectodermal dysplasia
What treatments are being studied? ER004
Status: Recruiting
Location: See all (8) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
This is an open-label, prospective, genotype-match controlled for primary estimand, non randomized, multicenter, international Phase 2 clinical trial designed to investigate the efficacy and safety of ER004 administered intraamniotically as a treatment for unborn XLHED male subjects.
Eligibility
Participation Requirements
Sex: Female
Minimum Age: 18
Healthy Volunteers: f
View:
⁃ For mother: adult mother with confirmed pregnancy no later than week 23+6 and genetically confirmed as carrier of an EDA mutation
• For fetal subject : male fetal subject with confirmed diagnosis of XLHED
• For untreated relative: untreated male relative subject aged between 6 months and 75 years with the same EDA mutation as the treated subject
Locations
United States
California
Cedars-Sinai Medical Center
RECRUITING
Los Angeles
Missouri
Washington University
RECRUITING
Saint Louis
Other Locations
France
Hôpital Necker - Enfants Malades
RECRUITING
Paris Cedex 15
Germany
Universitaetsklinikum Erlangen
RECRUITING
Erlangen
Universitaetsklinikum Leipzig AoeR
RECRUITING
Leipzig
Italy
IRCCS Ca' Granda Ospedale Policlinico
RECRUITING
Milan
Spain
Hospital Universitario Virgen de la Arrixaca
RECRUITING
El Palmar
United Kingdom
University Hospital of Wales Cardiff and Vale University Local Health
RECRUITING
Cardiff
Contact Information
Primary
Florence Porte-Thormé, PharmD
Info.er004@esperare.org
+41 22 794 4004
Backup
Marlène Guiraud
contact.edelife@pierre-fabre.com
+33 5 34 50 60 00
Time Frame
Start Date: 2022-04-26
Estimated Completion Date: 2029-01
Participants
Target number of participants: 20
Treatments
Experimental: ER004
Human immunoglobulin G1 constant region - human ectodysplasin-A1 receptor binding domain fusion protein.
Related Therapeutic Areas
Sponsors
Collaborators: Iqvia Pty Ltd, Pierre Fabre Medicament
Leads: EspeRare Foundation