Actively Recruiting
Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia
Led by EspeRare Foundation · Updated on 2025-04-30
20
Participants Needed
8
Research Sites
553 weeks
Total Duration
On this page
Sponsors
E
EspeRare Foundation
Lead Sponsor
P
Pierre Fabre Medicament
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adult pregnant woman with confirmed pregnancy no later than 23 weeks and 6 days and genetically confirmed as carrier of an EDA mutation
- Male fetus with confirmed diagnosis of X-linked hypohidrotic ectodermal dysplasia (XLHED)
- Untreated male relative subject aged between 6 months and 75 years with the same EDA mutation as the treated subject
You will not qualify if you...
- Active maternal infection linked to risk of preterm birth or congenital anomalies
- Documented maternal HIV infection
- Pre-existing maternal medical conditions increasing risk of preterm birth or serious pregnancy complications
- Pregnancy disorders with increased risk of preterm birth or maternal, fetal, or neonatal morbidity/mortality
- Fetal major anatomical anomalies unrelated to XLHED that increase morbidity or mortality risk
- Echocardiogram, ultrasound, or other findings indicating high risk of fetal demise or preterm birth
- Conditions other than XLHED affecting tooth germ number
- Medical conditions that prevent safe study conduct or interfere with efficacy assessments
- Untreated relative carriers of hypomorphic EDA mutation
- Known allergy to pilocarpine or similar drugs
- Presence of implanted devices like defibrillators or pacemakers
- Previous treatment with study drug before trial start
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 8 locations
1
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
Actively Recruiting
2
Washington University
St Louis, Missouri, United States, 63110
Actively Recruiting
3
Hôpital Necker - Enfants Malades
Paris, Paris, France, 75743
Actively Recruiting
4
Universitaetsklinikum Erlangen
Erlangen, Bavaria, Germany, 91054
Actively Recruiting
5
Universitaetsklinikum Leipzig AoeR
Leipzig, Saxony, Germany, 04103
Actively Recruiting
6
IRCCS Ca' Granda Ospedale Policlinico
Milan, Italy, 20122
Actively Recruiting
7
Hospital Universitario Virgen de la Arrixaca
El Palmar, Murcia, Spain, 30120
Actively Recruiting
8
University Hospital of Wales Cardiff and Vale University Local Health
Cardiff, United Kingdom, CF14 4XW
Actively Recruiting
Research Team
A
Agnes Jaulent
CONTACT
M
Marlène Guiraud
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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