A Clinical Study to Evaluate the Efficacy and Safety of LIV-GAMMA SN Inj.10% in Primary Immune Thrombocytopenia (ITP)

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the efficacy and safety of LIV-GAMMA SN Inj.10% administered for 2 days in adult subjects with primary immune thrombocytopenia (ITP). The primary objective of this study is to determine the responder rate. A response is defined as a platelet count of ≥30×10^9/L and at least a 2-fold increase of the baseline, confirmed on at least 2 separate occasions at least 7 days apart without bleeding. The secondary objectives are to evaluate the further efficacy assessments including time to response and duration of response, and the safety of LIV-GAMMA SN Inj.10%.

Official Title
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A Multicenter, Open-Label, Phase III Study to Evaluate the Efficacy and Safety of LIV-GAMMA SN Inj.10% in Primary Immune Thrombocytopenia (ITP)

Conditions
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Immune Thrombocytopenia

Intervention / Treatment
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* Biological: LIV-GAMMA SN Inj.10%


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Completed informed consent process Male or female aged ≥19 years Diagnosis of chronic ITP (≥12 months since diagnosis) Mean screening platelet count of <30×10^9/L from 3 qualifying platelet counts performed within 14 days before the start of treatment, with no individual platelet count above 35×10^9/L No other factors inducing ITP If the patient is taking corticosteroid, attenuated androgen, cyclophosphamide, azathioprine or other drugs for ITP, the treatment regimen and dose should be stable at least 1 month prior to screening and should be lasted during this study Females of child-bearing potential with a negative urine pregnancy test and who agree with contraception during this study Exclusion Criteria: Patients who have allergy or hypersensitivity to blood products, blood-derived products, intravenous immunoglobulin (IVIg) or immunoglobulin G (IgG) Patients who have immunoglobulin A (IgA) deficiency Patients who were immunized with live attenuated vaccines within 12 months from the first administration of LIV-GAMMA SN Inj.10% Patients who had received IVIg or blood/blood-derived products within 1 month from the first administration of LIV-GAMMA SN Inj.10% Patients who had received other investigational products within 1 month from the first administration of LIV-GAMMA SN Inj.10% Patients who had received Rituximab within 3 months from the first administration of LIV-GAMMA SN Inj.10% Patients who were taking anticoagulants or other agents related to platelet function (e.g., Aspirin, other NSAID) at the time of screening Patients who are pregnant and nursing Patients who are positive for human immunodeficiency virus (HIV), hepatitis B virus (HBV) or hepatitis C virus (HCV) at the time of screening Patients who have 3-fold higher levels of alanine transaminase (ALT), aspartate transaminase (AST) than the upper limit of normal at the time of screening Patients who suffered from severe renal impairment (eGFR<30 mL/min/1.73 m^2 at the time of screening) Patients who had history of deep vein thrombosis (DVT) or thrombotic complications against IVIg therapy Patients who had history of neurovascular or cardiovascular disorders (e.g., Blood hyperviscosity, transient ischemic attack (TIA), stroke, other thromboembolism, unstable angina) Patients who have an ongoing history of acute or chronic condition that affect to the participation of this study Patients who have an ongoing history of medical condition inducing secondary immune deficiency (e.g., Leukemia, lymphoma, multiple myeloma, HIV infection, chronic or cyclic neutropenia (absolute neutrophil count<500/mm^3) Patients who are suffering from hypertension (systolic blood pressure>160 mmHg or diastolic blood pressure>100 mmHg) Patients who have hemoglobin level≤10 g/dL at the time of screening

Ages Eligible for Study
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Minimum Age: 19 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: LIV-GAMMA SN Inj.10%<br> | Biological: LIV-GAMMA SN Inj.10%<br>* LIV-GAMMA SN Inj. 10% is administered intravenously at a dose of 1 g/kg daily for 2 consecutive days to patients with primary immune thrombocytopenia.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Responder rate (CR or R) | The rate of subjects with complete response (CR) defined as cases with a platelet count ≥100×10^9/L, confirmed on at least 2 separate occasions at least 7 days apart with an absence of bleeding or response (R) defined as cases with a platelet count ≥30×10^9/L and at least a 2-fold increase of the baseline count, confirmed on at least 2 separate occasions at least 7 days apart with an absence of bleeding | 28 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The percentage of subjects with complete response (CR) | The percentage of subjects with complete response (CR) defined as cases with a platelet count ≥100×10^9/L, confirmed on at least 2 separate occasions at least 7 days apart with an absence of bleeding | 28 days | 
| The percentage of subjects with response (R) | The percentage of subjects with response (R) defined as cases with a platelet count ≥30×10^9/L and at least a 2-fold increase of the baseline count, confirmed on at least 2 separate occasions at least 7 days apart with an absence of bleeding | 28 days | 
| Time to response | The time from the treatment initiation to the day when complete response (CR) or response (R) is achieved | 28 days | 
| Duration of response | The time from the achievement of complete response (CR) or response (R) to the day when loss of complete response (CR) or response (R) is achieved | 28 days | 
| Bleeding | Bleeding assessment with ITP-BAT bleeding grading system | 28 days | 
| Adverse events |  | 28 days |