Multicenter Randomized Double-blind Comparative Study of Clinical Efficacy and Safety of GNR-069 (JSC "GENERIUM", Russia) and Nplate (Amgen Europe BV, The Netherlands) in Patients With Idiopathic Thrombocytopenic Purpura

RMP-ITP-III

It is a phase III multicenter randomized double-blinded comparative study of clinical efficacy and safety of GNR-069 and Nplate in patients with idiopathic thrombocytopenic purpura

2021-09-09

2023-08-22

2023-10-19

Completed

Early phase I

160

Inclusion Criteria: 1. Written Informed Consent Form to participate in the study; 2. Men and women aged 18-75 years inclusive at the time of signing the Informed Consent Form; 3. Documented diagnosis of ITP with a disease duration of more than 12 months from the moment of confirmation of the diagnosis by bone marrow aspirate or biopsy results; 4. A. For patients who have not had splenectomy: * established absence/loss of response to therapy with at least one drug of fist-line treatment for ITP (which include GCs an IVIG); OR * the occurrence of side effects during the course of therapy with the drug of the fist-line, making it impossible to use it further; B. For patients who underwent splenectomy: • loss/lack of response to splenectomy; 5. Thrombocytopenia ≥30.0 x 109/L - \<50.0 x 109/L with severe hemorrhagic syndrome or thrombocytopenia \<30.0 x 109/l, regardless of the presence of hemorrhagic syndrome, according to the results of platelet count conducted in a local laboratory for 7 days before the start of therapy with investigational or reference drug; 6. Patients receiving GCs, azathioprine and danazole should receive these drugs in a maintenance dose for at least 4 weeks before starting therapy with investigational or reference drug; 7. Consent of study participants with preserved childbearing function to use reliable methods of contraception (a combination of at least two methods, including 1 barrier method, for example, the use of a condom and spermicide) from the moment of signing the Informed Consent Form and 3 months after the last administration of investigational or reference drug. Exclusion Criteria: 1. Hypersensitivity to the components of investigational or reference drug or E. coli proteins ; 2. Unresolved severe hemorrhagic syndrome requiring emergency treatment at the time of initiation of study or reference drug therapy ; 3. Fisher-Evans Syndrome; 4. Conditions with a high risk of thromboembolic complications ; 5. Myelodysplastic syndrome and/or bone marrow transplantation in anamnesis; 6. Deviations of clinical and laboratory parameters according to the results of studies of blood samples taken during the screening period; 7. Positive test results for hepatitis B, hepatitis C, or human immunodeficiency virus (HIV); 8. Pregnancy or breastfeeding; 9. Use of drugs: * romiplostim used less than 3 weeks before treatment with study or reference drug; * IVIG - less than 2 weeks prior to initiation of study or reference drug therapy; * eltrombopag - used less than 2 weeks before treatment with study or reference drug, or planned to use eltrombopag while the patient is participating in this study; * rituximab - used less than 14 weeks before treatment with study or reference drug, or planned to use rituximab while the patient is enrolled in this study; * cyclophosphamide, cyclosporine, vincristine, vinblastine and other drugs used to treat ITP not listed above and not included in the list of drugs approved for use during the study - use less than 8 weeks before the start of therapy with the study or reference drug or the use of any of these drugs is planned during the patient's participation in this study; * preparations of any hematopoietic growth factors - use less than 8 weeks before the start of therapy with an investigational or reference drug; * Influenza vaccines - less than 21 days prior to start of treatment with study or reference drug; * vaccines to prevent novel coronavirus disease (COVID-19) - completion of the vaccination program less than 21 days prior to the start of study or reference drug therapy; * other vaccines - less than 8 weeks prior to start of treatment with study or reference drug; 10. Splenectomy within 12 weeks prior to screening; 11. Participation in any clinical trials and/or use of unregistered drugs within 4 weeks prior to screening or 5 drug half-lives (whichever is greater); 12. Any other disease or condition that, in the opinion of the investigator, may preclude the patient from participating in the study.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Parallel assignment', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'DOUBLE', 'maskingDescription': 'Double-blinded', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}}, 'enrollmentInfo': {'count': 160, 'type': 'ACTUAL'}}

2024-03-06