A Double-blind, Placebo-controlled Study (72 Patients, Randomized 1:1) Followed by a Repeat-dosing Phase to Assess the Efficacy and Safety of DX-88 (Ecallantide; Recombinant Plasma Kallikrein Inhibitor) 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
- 91
- 试验地点
- 1
- 主要终点
- Treatment Outcome Score at 4 Hours Post-Dose
研究概览
简要总结
The purpose of this study is to determine if a subcutaneous dose of DX-88 (ecallantide; an investigational product) is safe and relieves symptoms of HAE in patients suffering from moderate to severe acute attacks of HAE.
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
- •Age 10 and older
- •Documented diagnosis of HAE, Type I or II
- •Executed informed consent
- •Presentation for treatment within 8 hours of patient recognition of moderate to severe HAE attack
排除标准
- •Receipt of investigational drug or device, other than DX-88, within 30 days of treatment
- •Receipt of non-investigational C1-INH (C1 esterase inhibitor) within 7 days of treatment
- •Diagnostic of acquired angioedema, estrogen-dependent angioedema or drug induced angioedema
- •Pregnancy or breastfeeding
- •Patients who have received DX-88 within 7 days of presentation for dosing in the Double-blind Phase
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
Phosphate Buffer Saline (PBS), pH 7.0 given as 3 subcutaneous injections.
干预措施: Phosphate Buffer Saline (PBS), (Drug)
结局指标
主要结局
Treatment Outcome Score at 4 Hours Post-Dose
时间窗: 4 hours post-dose (DOUBLE-BLIND PART)
Treatment Outcome Score (TOS) is a validated, comprehensive measure of symptom response to treatment. At 4 hours , patient assessment of response characterized by their change from baseline in symptom severity and collected by anatomic site of attack involvement, was recorded on a categorical scale (significant improvement \[100\] to significant worsening \[-100\]). The response at each anatomic site was weighted by baseline severity and then the weighted scores across all involved sites were averaged to calculate the TOS. Clinically meaningful improvement was indicated by a TOS of 30 or higher.
次要结局
- Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose(baseline, 4 hours post-dose (DOUBLE-BLIND PART))
- Time to Significant Improvement in Overall Response(4 hours post-dose (DOUBLE-BLIND PART))