CI

At a glance

ClinicalIndex Comparison Record
Phase 2Recruiting· 20 target
Drug / intervention
ER004biological
Likely dose
ER004 100 mg/kgfrom record
Key inclusion· 4
  • ✓Pregnant woman with confirmed pregnancy no later than week 23+6
  • ✓Genetically confirmed as carrier of an EDA mutation
  • ✓Male fetal subject with confirmed diagnosis of XLHED
  • ✓Untreated male relative aged 6 months to 75 years with same EDA mutation as treated subject
Key exclusion· 11
  • ✕Active maternal infection with risk of preterm birth or congenital anomalies
  • ✕Documented maternal HIV infection
  • ✕Pre-existing maternal condition increasing risk of preterm birth or serious untoward event
  • ✕Pregnancy disorder with increased risk of preterm birth or maternal/fetal/neonatal morbidity/mortality

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT04980638
NCT04980638Phase 2RecruitingOn TrackUpdated 14mo ago
Long Recruiting
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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

EspeRare Foundation·interventional·Posted Jul 28, 2021·Updated Apr 30, 2025

In Brief

A Phase 2 clinical trial evaluating ER004 for X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED). Currently recruiting, targeting 20 participants across 8 sites in 6 countries.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsX-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
CountriesFrance, Germany, Italy, Spain, United Kingdom, United States
CollaboratorsPierre Fabre Medicament, IQVIA Pty Ltd

Timeline

Phase 2Recruiting
202220232024202520262027202820292030203120322033
First PostedJul 28, 2021
Enrollment StartApr 26, 2022
Primary CompletionFeb 1, 2027
Study CompletionDec 1, 2032
TodayJul 2, 2026
Enrollment to primary: 4.8 yearsPosted 4.9 years agoPrimary completion in 7 months

Interventions

ER004biological

Intra-amniotic route 100 mg/kg of estimated fetal weight per injection. 3 injections, approximately 3 weeks apart starting from gestational week 26