A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation)
Condition: Paroxysmal Nocturnal Hemoglobinuria
MeSH terms: Hemoglobinuria; Hemoglobinuria, Paroxysmal
Study type: Interventional
Phase: Phase 3

Summary: 
      A safety and immunogenicity study in long-term treatment of Eculizumab (JSC "GENERIUM",
      Russian Federation) in patients with paroxysmal nocturnal hemoglobinuria, who have been
      involved earlier in the clinical trials of Eculizumab (JSC "GENERIUM", Russian Federation).
    

Detailed description: 
      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®. At the present time Eculizumab (JSC GENERIUM,
      Russia) have already been authorized as Elizaria® in Russia.

      This study is a multicenter open-label study of the safety, immunogenicity and efficacy of
      Eculizumab (JSC GENERIUM, Russia) in patients with paroxysmal nocturnal hemoglobinuria who
      completed participation in previous clinical trials of Eculizumab (JSC GENERIUM, Russia).

      Visit 1 of this study is carried out on the day of the Visit of completion of a previously
      conducted study, or on another day, observing the terms of administration of the drug
      prescribed by the instructions for medical use of Eculizumab.

      At this visit, all patients who signed the informed consent form and were immunized with
      meningococcal vaccine will begin therapy with Eculizumab (GENERIUM JSC), which will continue
      until the drug is registered or, as decided by the sponsoring company, until its commercial
      availability. When the last patient enrolled in the study has completed 6 months of therapy,
      which corresponds to the completion of Visit 13, an interim analysis of the safety and
      immunogenicity data of the study drug will be performed.
    

Intervention name(s): Eculizumab (JSC GENERIUM, Russia)
Intervention type(s): Biological
Intervention description(s): Eculizumab
Intervention MeSH terms: Eculizumab
Intervention model: Single Group Assignment
Intervention model description: Interventional
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: eculizumab; humanized monoclonal antibodies; safety; paroxysmal nocturnal hemoglobinuria; rare disease; biosimilar; complement system; C5 component of complement; pro-inflammatory cytokines; Membrane attack complex (MAC); immunogenicity