NCT01420627已完成3 期
A Study of EZN-2279 (Polyethylene Glycol Recombinant Adenosine Deaminase [PEG-rADA]) Administered as a Weekly Intramuscular Injection in Patients With Adenosine Deaminase (ADA)-Deficient Combined Immunodeficiency
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 7
- 试验地点
- 12
- 主要终点
- Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period
研究概览
简要总结
The purpose of this study is to evaluate the safety, efficacy, and pharmacokinetics of EZN-2279 in patients with ADA-deficient combined immunodeficiency currently being treated with Adagen.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of ADA-deficient combined immunodeficiency
- •Stable clinical status while receiving therapy with Adagen®. Patients previously receiving gene therapy or undergoing hematopoietic stem cell transplantation who still require Adagen® treatment are eligible. The dose of Adagen® must be stable for at least 6 months prior to study entry.
- •Have both of the following during the Adagen® Lead-in phase of the study prior to EZN-2279 transition:
- •Trough plasma ADA activity >15 μmol/h/mL while receiving Adagen® and
- •Total erythrocyte dAXP ≤0.02 μmol/mL from a trough blood sample
- •Patients or parent/guardian must be capable of understanding the protocol requirements and risks and providing written informed assent/consent
排除标准
- •Autoimmunity requiring immunosuppressive treatment
- •Patients with detectable neutralizing anti-Adagen® antibodies at screening evaluation
- •Severe thrombocytopenia (platelet count <50 x 10^9/L)
- •Current participation in other therapeutic protocols for ADA-deficient combined immunodeficiency
- •Current or prior participation in another clinical study with an investigational agent and/or use of an investigational drug in the 30 days before study entry
- •Known planned participation in a gene-therapy study for the planned duration of this study
- •Any condition that, in the opinion of the PI, makes the patient unsuitable for the study
- •Inability or unwillingness to administer Adagen® or EZN-2279 on a one-time-per-week regimen
- •Inability to comply with the study protocol
- •Female patients who are pregnant or lactating
- •Female patients who are breast-feeding
- •Female subjects of childbearing potential who are not using an FDA approved birth control method
结局指标
主要结局
Number of Patients Detoxified At Each Visit in EZN-2279 Treatment Period
时间窗: Baseline through Week T-21
Number of patients with total erythrocyte dAXP concentration from a trough blood sample \<0.02 mmol/L
次要结局
- Safety Summary Data(Through end of EZN-2279 study treatment, up to 203 weeks)
- Summary of Trough ADA Activity Levels in EZN-2279 Treatment Period(From Baseline through Week T-21)
- Summary of Trough ADA Activity Levels in EZN-2279 Maintenance Period(Through end of EZN-2279 study treatment, up to 203 weeks)
- Duration of Hospitalization(Through end of EZN-2279 study treatment, up to 203 weeks)
- Summary of Trough dAXP Levels in EZN-2279 Treatment Period(From Baseline through Week T-21)
- Summary of Trough dAXP Levels in EZN-2279 Maintenance Period(Through end of EZN-2279 study treatment, up to 203 weeks)
- Number of Patients With Infections and Hospitalizations(Through end of EZN-2279 study treatment, up to 203 weeks)
- Number of Patients Detoxified At Each Visit in EZN-2279 Maintenance Period(From Week 34 to End of Study/Early Discontinuation, up to 203 weeks)
研究者
研究点 (12)
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