Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® in Patients With Paroxysmal Nocturnal Hemoglobinuria.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- AO GENERIUM
- 入组人数
- 44
- 试验地点
- 8
- 主要终点
- Dynamics of the peripheral blood LDH level during 52 weeks of the maintenance stage of treatment with Elizaria® (between Visits 6 and 13, inclusive).
研究概览
简要总结
This is prospective observational study of long-term pathogenic treatment of Elizaria® in patients with paroxysmal nocturnal hemoglobinuria.
详细描述
Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare, acquired, life-threatening, progressive clonal blood disease that is developed as a result of the expansion of one or more clones of hematopoietic stem cells with a somatic PIG A gene mutation. The main pathogenic mechanism of PNH development is a dysregulation of the complement system, in which there are no complement inhibitors CD55 and CD59 that are bounded by the GPI-anchor on the surface of blood cells, or there is a deficiency of them.
Eculizumab is the first drug for the pathogenetic therapy of PNH, a recombinant humanized monoclonal antibody against the C5 component of complement. Binding with high affinity to C5, eculizumab prevents the cleavage of C5 into C5a and C5b, thereby inhibiting the formation of pro-inflammatory cytokines (via C5a) and Membrane attack complex (MAC) (via C5b).
Using of eculizumab in patients with PNH leads to a significant decrease in hemolysis, the incidence of thrombosis and increasing of the quality of life of patients. It increases life expectancy with long-term using of eculizumab.
The drug Eculizumab, developed by JSC "GENERIUM", Russia, is a biological analogue (biosimilar) of the original drug Soliris®.
The population of the study is the patients with an established diagnosis of PNH who have indications for pathogenic treatment and are receiving Elizaria®.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Male and female patients aged 2 years with an established diagnosis of PNH;
- •Prescribing Elizaria® as a pathogenetic therapy;
- •Signing by the patient of the informed consent form for participation in the study.
排除标准
- •Intolerance to eculizumab or other components of the drug.
结局指标
主要结局
Dynamics of the peripheral blood LDH level during 52 weeks of the maintenance stage of treatment with Elizaria® (between Visits 6 and 13, inclusive).
时间窗: 52-56 weeks
To assess the dynamics of the peripheral blood LDH level during 52 weeks the area under the LDH level - time curve (AUC) will be calculated. Descriptive statistics will be provided for the values of the LDH level at each visit, as well as for the AUC, indicating the geometric means and coefficient of variation.
次要结局
- Number / proportion of patients with a change in the level of hemoglobin in the blood ± 5 g / l and ± 10 g / l. during the period of maintenance therapy with Elizaria® (between Visits 6 and 13 inclusive)(52-56 weeks)
- Changes in hemoglobin levels during maintenance therapy with Elizaria® (between Visits 6 and 13 inclusive)(52-56 weeks)
- The number / proportion of patients requiring transfusion of erythrocyte components of donated blood during treatment with Elizaria®.(52-56 weeks)
- Change in the size of PNH-clone of granulocytes and erythrocytes at Visits 6 and 13 relative to the initial value at screening.(52-56 weeks)
- The number / proportion of patients with various thrombotic complications that developed during treatment with Elizaria®.(52-56 weeks)
- Number/proportion of patients with breakthrough hemolysis while observing an increased LDH, after a previous decrease in the course of treatment.(52-56 weeks)
- Frequency and severity of adverse events (AEs), serious adverse events (SAEs), including those associated with the use of Elizaria®, according to complaints, physical examination, assessment of vital signs, laboratory and instrumental studies, diaries.(52-56 weeks)
- Changes in vital signs, physical examination results, ECG, and laboratory values at Visit 13 from baseline at screening.(52-56 weeks)
- Number and proportion of patients with antidrug antibodies; titer of antidrug antibodies and their neutralizing activity.(52-56 weeks)