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

AO GENERIUM18 sites in 1 country160 target enrollmentSeptember 9, 2021

ConditionsIdiopathic Thrombocytopenic Purpura

Overview

Phase: Phase 3
Intervention: Not specified
Conditions: Idiopathic Thrombocytopenic Purpura
Sponsor: AO GENERIUM
Enrollment: 160
Locations: 18
Primary Endpoint: Proportion of patients achieving sustained response to treatment
Status: Completed
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

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

Detailed Description

The drug GNR-069(JSC "GENERIUM", Russia) is biosimilar to the original drug Nplate. This study is aimed to compare the clinical efficacy and safety of the drug GNR-069 and the drug Nplate to register of the drug GNR-069 in the Russian Federation for therapy in patients with idiopathic thrombocytopenic purpura (ITP). The study also provides for the evaluation of pharmacokinetic parameters and immunogenicity.

Registry

clinicaltrials.gov

Start Date

September 9, 2021

End Date

October 19, 2023

Last Updated

2 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

AO GENERIUM

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Written Informed Consent Form to participate in the study;
•Men and women aged 18-75 years inclusive at the time of signing the Informed Consent Form;
•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;
•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;
•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;
•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;

Exclusion Criteria

•Hypersensitivity to the components of investigational or reference drug or E. coli proteins ;
•Unresolved severe hemorrhagic syndrome requiring emergency treatment at the time of initiation of study or reference drug therapy ;
•Fisher-Evans Syndrome;
•Conditions with a high risk of thromboembolic complications ;
•Myelodysplastic syndrome and/or bone marrow transplantation in anamnesis;
•Deviations of clinical and laboratory parameters according to the results of studies of blood samples taken during the screening period;
•Positive test results for hepatitis B, hepatitis C, or human immunodeficiency virus (HIV);
•Pregnancy or breastfeeding;
•Use of drugs:
•romiplostim used less than 3 weeks before treatment with study or reference drug;

Outcomes

Primary Outcomes

Proportion of patients achieving sustained response to treatment

Time Frame: 26 weeks

A sustained response to treatment is defined as the number of platelets ≥ 50.0 x 109/L for at least 9 out of 12 consecutive visits during the treatment period with the study or reference drug.

Secondary Outcomes

Number of cases of emergency therapy for severe hemorrhagic syndrome during the treatment period, starting from the second week of therapy with the investigational or reference drug(25 weeks)
Proportion of patients with no/loss of response to treatment with investigational or reference drug(26 weeks)
Proportion of patients who achieve stable platelet count during treatment with investigational or reference drug(26 weeks)
Time from initiation of therapy with investigational or reference drug to reaching a stable platelet count(26 weeks)
Number of clinically significant bleeding episodes during the treatment period, starting from the second week of therapy with investigational or reference drug(26 weeks)
Change in ITP-specific bleeding assessment tool (ITP-BAT) scores at last visit from baseline at screening(26 weeks)
Proportion of patients receiving approved ITP prophylactic drugs (glucocorticosteroids, azathioprine, danazol) in this study at the time of randomization(26 weeks)