A Placebo-controlled Randomized Trial to Evaluate the Efficacy and Safety of GNR-038 in Comparison With Berinert® for Acute Attacks Relief in Patients With Hereditary Angioedema
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- AO GENERIUM
- 试验地点
- 5
- 主要终点
- Time to symptoms relief onset of acute HAE attack within 24 hours after the end of the drug administration.
研究概览
简要总结
It is a placebo-controlled randomized trial to evaluate the efficacy and safety of GNR-038 in comparison with Berinert® in patients with hereditary angioedema
详细描述
Hereditary angioedema is a rare, potentially life-threatening genetically determined disease associated with a deficiency or impairment of the functional activity of the C1-esterase inhibitor (C1-inhibitor). The main clinical manifestation of hereditary angioedema is recurrent subcutaneous or submucosal swelling of various localization. Most often, the development of the disease is based on a mutation in the SERPING1 gene. The prevalence of the disease in the world ranges from 1:10 000 to 1:150 000. GNR-038 is a recombinant C1 inhibitor (rhC1INH), which is a complete structural and functional analog of the plasma C1 inhibitor. Phase I study results showed convincing safety and tolerability evidence of GNR-038.
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
- Double (Participant, Investigator)
盲法说明
At the first stage - the study will be blinded, at the second stage - open-label
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Study stage 1: Placebo
Placebo
干预措施: Placebo (Drug)
Study stage 1: GNR-038, 50 МЕ/ kg
Recombinant C1 esterase inhibitor
干预措施: GNR-038, 50 МЕ/ kg (Drug)
Study stage 1: GNR-038, 100 МЕ/ kg
Recombinant C1 esterase inhibitor
干预措施: GNR-038, 100 МЕ/ kg (Drug)
Study stage 1: Berinert®, 20 МЕ/ kg
Human C1 esterase inhibitor
干预措施: Berinert®, 20 МЕ/ kg (Drug)
Study stage 2: GNR-038 in selected dose
Recombinant C1 esterase inhibitor
干预措施: GNR-038. The dose will be selected according to results of stage 1 clinical trial. (Drug)
Study stage 2: Berinert®, 20 МЕ/ kg
Human C1 esterase inhibitor
干预措施: Berinert®, 20 МЕ/ kg (Drug)
结局指标
主要结局
Time to symptoms relief onset of acute HAE attack within 24 hours after the end of the drug administration.
时间窗: 24 hours
A persistent decrease in the intensity of symptoms by 20 mm from the initial level on the visual analogue scale (VAS) will be regarded as a relief of symptoms of HAE. 0 mm is the absence of symptoms, 100 mm is the maximum possible intensity of symptoms
次要结局
- Time to complete resolution of the symptoms of an acute HAE attack within 24 hours after the end of the study drug administration.(24 hours)
- Time to minimum manifestation onset of acute HAE attack symptoms after the completion of study drug administration.(24 hours)
- The proportion of HAE exacerbation episodes that achieved symptom relief after 1 hour and 4 (four) hours after the end of study drug administration.(1 hour; 4 hours.)
- The rate of attacks with HAE current localization relapse or with the occurrence of a new acute attack of a different localization within 24 (twenty-four) hours after the study drug administration.(24 hours)
- The rate of attacks that required additional administration of emergency drugs (human C1-esterase inhibitor or icatibant).(24 hours)
- The rate of attacks that did not respond therapeutically to the study drug administration(4 hours; 24 hours)
- The intensity of acute HAE attack symptoms within 24 (twenty-four) hours after study drug administration.(24 hours)
- The level of anti-drug antibodies neutralizing activity.(7 and 14 days)
- Frequency of adverse events.(14 days)
- Frequency of anti-drug antibody formation.(7 and 14 days)