EUCTR2012-003561-17-GB进行中（未招募）1 期
A Phase 2 open-label, dose-escalation study to evaluate the safety, pharmacokinetics, immunogenicity and pharmacodynamics/efficacy of EDI200, an EDA-A1 replacement protein, administered to male infants with X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED).
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •Subjects for study drug administration must meet all of the following criteria to be enrolled:
- •1. Male with genetic confirmation of an XLHED diagnosis.
- •2. Subject must be at least 48 hours of age and no older than 14 days.
- •3. Subject will have reached term (defined as 37 weeks gestation or older) prior to receiving first dose study drug.
- •4. Written informed consent of both parents (if reasonably available) must be obtained for treatment of their XLHED-affected male infant.
- •5. Neither mother nor the XLHED-affected male infant known to have received an investigational study drug in the 9 months prior to study subject enrollment in this study.
- •6. No major medical issues that the PI considers a contraindication to participation.
- •Male siblings of subjects receiving study drug must meet all of the following criteria to be enrolled in the natural history sub-study (no age limit involved):
- •1. Provide written informed consent/assent.
- •2. A full or half-sibling of a study subject where the study subject has received at least one dose of study drug in the Phase 2 XLHED Neonate Study and has not yet completed the study.
- •3. No major medical issues that the PI considers a contraindication to participation.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 10
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Subjects for study drug administration who meet any of the following criteria cannot be enrolled in this study:
- •1. Medically significant postnatal complications or congenital anomalies outside of those considered to be associated with the diagnosis of XLHED.
- •Male siblings of subjects receiving study drug who meet any of the following criteria cannot be enrolled in the natural history sub-study:
- •1. Known hypersensitivity to pilocarpine or pilocarpine-like muscarinic agonists.
- •2. Known hypersensitivity to lidocaine or lidocaine-like agents.
- •3. Presence of pacemaker.
- •4. Subjects who are not able or are not willing to comply with the procedures of this protocol.
- •5. Subject has a condition, which in the opinion of the PI would not allow for safe conduct of the study.
研究者
相似试验
进行中（未招募）
1 期
A phase 2 study to evaluate the safety of EDI200 administered to male infants with X-Linked Hypohydrotic Ectodermal Dysplasia (XLHED).X-linked hypohidrotic ectodermal dysplasia (XLHED) is caused by inherited defects in the ectodysplasin (EDA) gene that disrupt synthesis and/or function of the primary translational product EDA-A1. The absence of normal EDA-A1 expression results in sweat and secretory gland hypoplasia predisposing XLHED-affected infants to serious and potentially life-threatening hyperthermia and pneumonia.MedDRA version: 17.1Level: LLTClassification code 10072592Term: Hypohidrotic ectodermal dysplasiaSystem Organ Class: 100000004850EUCTR2012-003561-17-DEEdimer Pharmaceuticals, Inc.15
进行中（未招募）
1 期
A phase 2 study to evaluate the safety of EDI200 administered to male infants with X-Linked Hypohydrotic Ectodermal Dysplasia (XLHED).EUCTR2012-003561-17-FREdimer Pharmaceuticals, Inc.10
进行中（未招募）
1 期
A Phase 2 open-label, dose-escalation study to evaluate the safety, pharmacokinetics, immunogeniticy and pharmacodynamics/efficacy of EDI200, an EDA-A1 replacement protein, administered to infants with X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED).X-linked hypohidrotic ectodermal dysplasia (XLHED) is caused by inherited defects in the ectodysplasin (EDA) gene that disrupt synthesis and/or function of the primary translational product EDA-A1. The absence of normal EDA-A1 expression results in sweat and secretory gland hypoplasia predisposing XLHED-affected infants to serious and potentially life-threatening hyperthermia and pneumonia.MedDRA version: 17.0Level: LLTClassification code 10072592Term: Hypohidrotic ectodermal dysplasiaSystem Organ Class: 100000004850EUCTR2012-003561-17-ITEdimer Pharmaceuticals, Inc.10
进行中（未招募）
不适用
An open-label Phase I/II dose escalation study evaluating the safety and efficacy of patupilone in patients with advanced ovarian, primary fallopian, or primary peritoneal cancer.Approximately 75% of women with ovarian cancer present advanced disease. Survival is highly dependent on the stage of disease at the initiation of treatment. Favorable prognostic factors include young age, cell type other than clear cell or mucinous, lower stage, good performance status, small residual tumor volume after surgery, and absence of ascites. The prognosis of patients with resistant / refractory ovarian cancer is uniformly poor with median overall survival ranging from 35 – 41 weeks.EUCTR2005-004913-14-BEovartis Pharma Services AG110
进行中（未招募）
不适用
An open-label Phase I/II dose escalation study evaluating the safety and efficacy of patupilone in patients with advanced ovarian, primary fallopian, or primary peritoneal cancerApproximately 75% of women with ovarian cancer present advanced disease. Survival is highly dependent on the stage of disease at the initiation of treatment. Favorable prognostic factors include young age, cell type other than clear cell or mucinous, lower stage, good performance status, small residual tumor volume after surgery, and absence of ascites. The prognosis of patients with resistant / refractory ovarian cancer is uniformly poor with median overall survival ranging from 35 – 41 weeks.EUCTR2005-004913-14-ATovartis Pharma Services AG110