A randomized, international, multicenter double-blind study to compare the efficacy,safety, and immunogenicity of PZN-128 powder for solution for subcutaneous injection 250 µg (Pharmasyntez Nord JSC, Russia) versus Ñomparator drug in patients with chronic idiopathic (immune) thrombocytopenic purpura.

ITP is an autoimmune disease characterized by production of antibodies to the membrane structures of platelets and their precursors – megakaryocytes (MKC) causing increased destruction of platelets, inadequate thrombocytopoiesis characterized by isolated thrombocytopenia < 100*109/L and presence or absence of haemorrhagic syndrome of various severity. Ethiopathogenetic mechanisms of ITP development are unknown. ITP morbidity varies from 1.6 to 12.5 cases per 100,000 population per year; the average morbidity value among female patients is higher than in male patients (3.03 per 100,000 persons against 2.77 per 100,000 population, respectively).The main ITP therapy aim – management of haemorrhagic syndrome and increase of platelet count up to safe level – not less than 50.0 × 109/L, which provides good quality of life of a patient with no hematostaxic.Treatment of patients with ITP should be based on individual approach, which is determined by the severity of haemorrhagic syndrome, but not by platelet count. Comorbidity, patient’s life style, complications of previous treatment, planned surgeries, etc. are of value at the therapy selection.No therapy types capable of providing complete recovery are available at present, however an adequate therapy improves the patient’s quality of life with severe chronic – recurrent and refractory forms of the disease. It allows to preserve the ability to work, to perform surgical procedures and surgeries with correct preparation of a patient, allows female patients to become pregnant and to deliver healthy children.One of the most effective and safe therapeutic approaches in the treatment of ITP is based on the use of thrombopoietin receptor agonists – romiplostim. Thrombocytic response while using romiplostim is achieved by 93% of patients, while this drug product may be efficient as second line therapy with contraindications to splenectomy or third line therapy after splenectomy failure. Romiplostim (Nplate) is much more effective in patients with chronic ITP as compared to rituximab and standard therapy.The developer of the drug product and the Sponsor of the study, Pharmasyntez-Nord JSC, Russia, conducted a phase I clinical trial "Double-blind, randomized, cross-over clinical study of comparative pharmacokinetics, pharmacodynamics and safety of the study drug product Romiplostim, (romiplostim, powder for solution for subcutaneous injection, 250 µg, manufacturer is Pharmasyntez-Nord JSC) and the reference drug product Nplate, (romiplostim, powder for solution for subcutaneous injection 250 µg (Amgen Europe B.V., Netherlands) with a single subcutaneous injection to healthy volunteers.44 healthy volunteers were randomized into the study, of which all study participants completed the study protocol.