Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia
Condition(s) studied
Investigational drug(s) / intervention(s)
ER004: Intra-amniotic route 100 mg/kg of estimated fetal weight per injection. 3 injections, approximately 3 weeks apart starting from gestational week 26
Study 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
Primary outcome measure(s)
- 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)
Trial sites (8)
| Facility | City | Region | Status |
|---|---|---|---|
| Cedars-Sinai Medical Center | Los Angeles | California | Recruiting |
| Washington University | St Louis | Missouri | Recruiting |
| Hôpital Necker - Enfants Malades | Paris | Paris | Recruiting |
| Universitaetsklinikum Erlangen | Erlangen | Bavaria | Recruiting |
| Universitaetsklinikum Leipzig AoeR | Leipzig | Saxony | Recruiting |
| IRCCS Ca' Granda Ospedale Policlinico | Milan | Italy | Recruiting |
| Hospital Universitario Virgen de la Arrixaca | El Palmar | Murcia | Recruiting |
| University Hospital of Wales Cardiff and Vale University Local Health | Cardiff | United Kingdom | Recruiting |
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04980638 on ClinicalTrials.gov ↗ ← All trials in Italy