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An open-label Phase 2 trial to investigate efficacy and safety of intraamniotic administrations of ER004 to male subjects with X-linked hypohidrotic ectodermal dysplasia (XLHED)
EU CTIS ID: 2024-512632-30-00
What this study is testing
To assess the efficacy of intra-amniotic administrations of ER004 on sweating ability in male XLHED subjects with a null mutation in EDA at 6 months of age, compared to untreated matched control subjects.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- For mother : Adult mother with confirmed pregnancy no later than week 23+6 days and genetically confirmed as carrier of an EDA mutation
- For fetus subject: Male Fetal subject with confirmed diagnosis of XLHED
- Untreated relative : Untreated male relative subject ages 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 risk to the child. Documented maternal HIV infection.
- For Untreated Relative: Previous treatment with the study intervention by any route of administration prior to study start.
- For mother: Any pre-existing maternal medical condition that increases the risk of preterm birth or increases the risk of a serious untoward event occurring to the mother during pregnancy.
- For mother: Any pregnancy disorder associated with an increased risk of preterm birth, and/or maternal, fetal or neonatal morbidity/mortality
- For fetal subject: Second major anatomic anomaly (not related to the underlying XLHED) that contributes to a significant morbidity or mortality risk, or echocardiogram or ultrasonography or other findings that indicate a high risk of fetal demise or risk of preterm birth
- For fetal subject: Any condition other than XLHED (i.e., other forms of ectodermal dysplasia, large orofacial clefts) that is likely to have an impact on the number of tooth germs.
See the full eligibility criteria
- For mother : Adult mother with confirmed pregnancy no later than week 23+6 days and genetically confirmed as carrier of an EDA mutation
- For fetus subject: Male Fetal subject with confirmed diagnosis of XLHED
- Untreated relative : Untreated male relative subject ages between 6 months and 75 years with the same EDA mutation as the treated subject
- For mother: Any evidence of active maternal infection associated with a risk of preterm birth and/or congenital anomalies of prenatal and postnatal risk to the child. Documented maternal HIV infection.
- For Untreated Relative: Previous treatment with the study intervention by any route of administration prior to study start.
- For mother: Any pre-existing maternal medical condition that increases the risk of preterm birth or increases the risk of a serious untoward event occurring to the mother during pregnancy.
- For mother: Any pregnancy disorder associated with an increased risk of preterm birth, and/or maternal, fetal or neonatal morbidity/mortality
- For fetal subject: Second major anatomic anomaly (not related to the underlying XLHED) that contributes to a significant morbidity or mortality risk, or echocardiogram or ultrasonography or other findings that indicate a high risk of fetal demise or risk of preterm birth
- For fetal subject: Any condition other than XLHED (i.e., other forms of ectodermal dysplasia, large orofacial clefts) that is likely to have an impact on the number of tooth germs.
- For fetal subject: Any other medical condition which in the opinion of the investigator would not allow for safe conduct of the study for the subject, or that would interfere with efficacy assessments (e.g., any disorders that lead to reduced fetal swallowing).
- For Untreated Relative: Carrier of an hypomorphic EDA mutation.
- For Untreated Relative: Known hypersensitivity to pilocarpine or pilocarpine-like muscarinic agonists.
- For Untreated Relative: Presence of an implanted device (e.g., defibrillator, neurostimulator, pacemaker).
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- United States
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 male, in utero. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.