Open-label Patient Continuation of DX-88 (Ecallantide) for Acute Hereditary Angioedema Attacks
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 147
- 试验地点
- 41
- 主要终点
- Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hrs Post Dosing
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of repeated doses of ecallantide in the treatment of acute attacks of hereditary angioedema and to allow HAE patients continued access to ecallantide. In addition, patients enrolled in DX-88/20 (EDEMA4) trial will be followed up and treated for subsequent attacks in this trial.
详细描述
This is an open label trial.
The study is designed to assess the efficacy and safety of 30 mg subcutaneous ecallantide in the treatment of acute attacks of hereditary angioedema. This study is designed to provide efficacy and safety data on repeated use of ecallantide. These data are intended to support the marketing authorization of ecallantide in the treatment of acute attacks of hereditary angioedema. Efficacy and safety of ecallantide will be evaluated in this study.
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
- 年龄范围
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •10 years of age or older
- •Documented diagnosis of HAE (Type I or II)
- •Willing and able to give informed consent
- •Acute HAE attack at time of presentation
排除标准
- •Receipt of an investigational drug or device, within 30 days prior to study treatment, other than DX-88 (ecallantide)
- •Pregnancy or breastfeeding
- •Receipt of non-investigational C1-INH or DX-88 within 72 hours of treatment
- •Patients eligible for current, ongoing clinical trial in which DX 88 (ecallantide) is offered
研究组 & 干预措施
DX-88 (ecallantide)
DX-88 (ecallantide) Patients were treated with DX-88 (ecallantide) when they experienced an HAE attack. 30 mg dose of ecallantide given via 3 SC injections; a second 30 mg dose can be administered if needed. Patients were to be assessed until 4 hrs post-dose. Patients were asked to return for 3 follow-up visits: 7 days, 28 days and 90 days post-dose.
干预措施: ecallantide (Drug)
结局指标
主要结局
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hrs Post Dosing
时间窗: 4 hrs post dose after every episode
Mean Symptom Complex Severity (MSCS) score is a validated point-in-time measure of symptom severity. At baseline and 4 hrs, patients rated the severity on a categorical scale (0=normal, 1=mild, 2=moderate, 3=severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms; clinically meaningful improvement was indicated by a reduction in the score of 0.30 or more.
次要结局
- Treatment Outcome Score (TOS) at 4 Hrs Post Dosing, Based on the Patient Assessment of Baseline Severity of Symptoms(4 hrs post dose after every episode)
- Time to Significant Improvement(15 min - 4 hrs post dose after every episode)