CTIS2024-512632-30-00招募中1 期
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 X-linked hypohidrotic ectodermal dysplasia (XLHED) - EDELIFE
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 13
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- In Utero 至 In Utero（—）
- 性别
- Male
入选标准
- •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
排除标准
- •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).
研究者
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