HELP Study: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study to Evaluate DX-2930 For Long-Term Prophylaxis Against Acute Attacks of Hereditary Angioedema (HAE)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 125
- 试验地点
- 41
- 主要终点
- Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Treatment Period
研究概览
简要总结
This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females 12 years of age or older at time of screening
- •Documented diagnosis of HAE, Type I or II
- •Baseline rate of at least 1 Investigator-confirmed HAE attack per 4 weeks
- •Adult subjects and caregivers of subjects under the age of 18 are willing and able to read, understand, and sign an informed consent form. Subjects age 12 to 17, whose caregiver provides informed consent, are willing and able to read, understand an dsign an assent form.
- •Males and femailes who are fertile and sexually active must adhere to contraception requirements.
排除标准
- •Concomitant diagnosis of another form of chronic, recurrent angioedema, such as acquired angioedema, idiopathic angioedema, or recurrent angioedema associated with urticaria.
- •Participation in a prior DX-2930 study
- •Treatment with any other investigational drug or exposure to an investigational device within 4 weeks prior screening
- •Exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications within 4 weeks prior to screening.
- •Exposure to androgens within 2 weeks prior to entering the run-in period.
- •Use of long-term prophylactic therapy for HAE within 2 weeks prior to entering the run-in period.
- •Use of short-term prophylaxis for HAE within 7 days prior to entering the run-in period.
- •Any of the following liver function test abnormalities: alanine aminotransferase (ALT) > 3x upper limit of normal, or aspartate aminotransferase (AST) > 3x upper limit of normal, or total bilirubin > 2x upper limit of normal (unless the bilirubin elevation is a result of Gilbert's syndrome).
- •Pregnancy or breastfeeding.
研究组 & 干预措施
DX-2930 300 mg every 2 weeks
300 mg DX-2930 administered every 2 weeks by subcutaneous injection.
干预措施: DX-2930 - 300mg/2wk (Drug)
DX-2930 300 mg every 4 weeks
300 mg DX-2930 administered every 4 weeks by subcutaneous injection
干预措施: DX-2930 - 300mg/4wk (Drug)
DX-2930 150 mg every 4 weeks
150 mg DX-2930 administered every 4 weeks by subcutaneous injection
干预措施: DX-2930 - 150mg/4wk (Drug)
Placebo
Placebo administered every 2 weeks by subcutaneous injection.
干预措施: Placebo (Drug)
结局指标
主要结局
Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Treatment Period
时间窗: From Day 0 to Day 182
HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Rate of investigator confirmed HAE attacks was analyzed using a generalized linear model (GLM) for count data assuming a poisson distribution with a log link function and Pearson chi-square scaling of standard errors to account for potential overdispersion. The logarithm of time in days each subject was observed during the treatment period was used as an offset variable in the model.
次要结局
- Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attack Requiring Acute Treatment(From Day 0 to Day 182)
- Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During Day 14 Through Day 182(From Day 14 to Day 182)
- Rate of Moderate or Severe Investigator Confirmed Hereditary Angioedema (HAE) Attacks(From Day 0 to Day 182)