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New treatment option for X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
Official title Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia
ClinicalTrials.gov ID: NCT04980638
What this study is testing
What is ER004?
ER004 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for x-linked hypohidrotic ectodermal dysplasia (xlhed).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older, women only
You may be able to join if
- 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
You likely can't join if
- For mother: any evidence of active maternal infection associated with a risk of preterm birth and/or congenital anomalies of prenatal and postnatal...
- For fetal subject : second major anatomic anomaly (not related to the underlying XLHED) that contributes to a significant morbidity or mortality...
- For untreated relative: carrier of an hypomorphic EDA mutation. Known hypersensitivity to pilocarpine or pilocarpine-like muscarinic agonists...
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- St Louis, Missouri, United States
- Paris, Paris, France
- Erlangen, Bavaria, Germany
- Leipzig, Saxony, Germany
- Milan, Italy
- El Palmar, Murcia, Spain
- Cardiff, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling female, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; St Louis, Missouri, United States; Paris, Paris, France; Erlangen, Bavaria, Germany; Leipzig, Saxony, Germany; Milan, Italy and 2 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.