EDEMA4: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Assess the Efficacy and Safety of DX-88 (Ecallantide) for the Treatment of Acute Attacks of Hereditary Angioedema
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 96
- 试验地点
- 45
- 主要终点
- Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of DX-88 (ecallantide) versus placebo in the treatment of moderate to severe acute attacks of hereditary angioedema.
详细描述
This is a randomized placebo-controlled trial.
The study is designed to assess the efficacy and safety of 30 mg subcutaneous ecallantide versus placebo in the treatment of moderate to severe acute attacks of hereditary angioedema. This study is conducted under Special Protocol Assessment with the FDA and is designed to provide pivotal efficacy data on 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
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Executed informed consent
- •Documented diagnosis of HAE (Type I or II)
- •Presentation at the site within 8 hours of patient recognition of an moderate to severe HAE acute attack
排除标准
- •Receipt of an investigational drug or device, within 30 days prior to study treatment
- •Receipt of non-investigational C1-INH within 7 days of treatment
- •Receipt of DX-88 (ecallantide) within 3 days prior to study treatment
- •Diagnosis of acquired angioedema (AAE), estrogen-dependent angioedema or drug-induced angioedema (including angiotensin-converting enzyme inhibitor induced angioedema)
- •Pregnancy or breastfeeding
研究组 & 干预措施
DX-88 (ecallantide)
DX-88 (ecallantide) 30 mg given as three 10 mg/mL subcutaneous injections.
干预措施: ecallantide (Drug)
Placebo
Placebo, Phosphate Buffer Saline (PBS), pH 7.0 given as 3 subcutaneous injections.
干预措施: Phosphate Buffer Saline (PBS), pH 7.0 (Drug)
结局指标
主要结局
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose
时间窗: baseline, 4 hours post-dose
The Mean Symptom Complex Severity (MSCS) score is a validated, comprehensive point-in-time measure of symptom severity. At baseline and 4 hours, 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 at 4 Hours Post-Dose(4 hours post-dose)
- Patients With Significant Improvement in Overall Response(4 hours post-dose)
- Patients With a Successful Response at 4 Hours Post-dosing, Based on the Change From Baseline in the MSCS Score(baseline, 4 hours post-dosing)
- Proportion of Patients Maintaining a Significant Improvement in Overall Response Through 24 Hours(24 hours post-dosing)