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ER004

Phase 2

X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) | Monoclonal antibody | Other |IQVIA Holdings, Inc.|Last Updated: Apr 30, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment20
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04980638Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal DysplasiaPHASE2 RECRUITING 20 — — Apr 26, 2022Dec 1, 2032Apr 30, 20258 United States, France +4
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Study Endpoints
Primary Endpoints
Mean sweat volume
at 6 months of age (corrected age for subjects born at < 37 weeks)

For treated subject, mean sweat volume is collected on both forearms after local stimulation with pilocarpine (pilocarpine-induced sweating)

Secondary Endpoints
Mean sweat pore density (number/cm2)
at 6 months of age (key secondary) and other timepoints : 3, 12, 18, 24, 36, 48 and 60 months of age (secondary)
Dental development
at 6 months of age (key secondary) and other timepoints : 12, 18, 24, 36, 48 and 60 months of age (secondary)
Mean sweat volume
At 3, 12, 18, 24, 36, 48, 60 months of age
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ER004EXPERIMENTALHuman immunoglobulin G1 constant region - human ectodysplasin-A1 receptor binding domain fusion protein.
Interventions
NameTypeDescription
ER004BIOLOGICALIntra-amniotic route 100 mg/kg of estimated fetal weight per injection. 3 injections, approximately 3 weeks apart starting from gestational week 26
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Eligibility Criteria
Age Range18 Years — N/A
SexFEMALE
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: 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 ...

Countries:United StatesFranceGermanyItalySpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT04980638studyFirstPostDate: changed