Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia

Study Overview
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Brief Summary
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NewGam is a newly developed human normal immunoglobulin solution for intravenous administration (IGIV). This study will evaluate the safety and efficacy of NewGam 10% in patients with primary immune thrombocytopenia.

Detailed Description
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The primary objective of the study is to assess the efficacy of NewGam in correcting the platelet count. The secondary objective of the study is to evaluate the safety of NewGam. Safety will be assessed by monitoring vital signs, physical examination, evaluation of adverse events (AE) and laboratory parameters, and viral safety testing.

Official Title
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Prospective, Open-label, Non-controlled, Multicenter, Phase III Clinical Study to Evaluate the Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia

Conditions
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Primary Thrombocytopenia

Intervention / Treatment
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* Drug: NewGam


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age of ≥ 18 and ≤ 65 years. Confirmed diagnosis of chronic primary immune thrombocytopenia (ITP) (diagnosed with a threshold platelet count < 100x10^9/L) of at least 12 months duration. Platelet count of no more than 20x10^9/L with or without bleeding manifestations. Freely given written informed consent from patient. Women of childbearing potential must have a negative result on a pregnancy test (human chorionic gonadotropin [HCG]-based assay) and need to practice contraception using a method of proven reliability for the duration of the study. Exclusion Criteria: Thrombocytopenia secondary to other diseases (such as acquired immunodeficiency syndrome [AIDS] or systemic lupus erythematosus [SLE]), or drug-related thrombocytopenia. Administration of intravenous immunoglobulin (IGIV), anti-D immunoglobulin or thrombopoietin receptor agonists, or other platelet enhancing drugs (including immunosuppressive or other immunomodulatory drugs) within 3 weeks before enrollment, except for: Long-term corticosteroid therapy when the dose has been stable during the preceding 3 weeks and no dosage change is planned until study Day 22. Long-term azathioprine, cyclophosphamide, or attenuated androgen therapy when the dose has been stable during the preceding 3 months and no dosage change is planned until study Day 22. Unresponsive to previous treatment with IGIV or anti-D immunoglobulin.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 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: NewGam<br>Each participant received 1 g/kg NewGam intravenously on 2 consecutive days. | Drug: NewGam<br>* NewGam is a solution of human normal immunoglobulin 10% treated with solvent/detergent and nanofiltered for intravenous administration.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Responders | A responder is a study participant with an increase in platelets to ≥ 50x10^9/L within 7 days after the first infusion, ie, by study Day 8. | Day 1 to Day 8 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Alternative Responders | An alternative responder is a study participant with an increase in platelets to ≥ 30x10^9/L and to at least double the baseline platelet count, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding. | Day 1 to Day 22 | 
| Percentage of Complete Responders | A complete responder is a study participant with an increase in platelets to ≥ 100x10^9/L, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding. | Day 1 to Day 22 | 
| Percentage of Alternative Responders Who Lost the Response | An alternative responder who lost the response is a study participant who met the criterion for an alternative response but who then deteriorated, ie, their platelet count decreased to < 30x10^9/L, their platelet count decreased to less than double the baseline count, or bleeding occurred. | Day 1 to Day 22 | 
| Percentage of Complete Responders Who Lost the Response | A complete responder who lost the response is a study participant who met the criterion for a complete response but who then deteriorated, ie, their platelet count decreased to < 100x10^9/L or bleeding occurred. | Day 1 to Day 22 | 
| Time to a Response | A study participant had a response if their platelets increased to ≥ 50x10^9/L within 7 days after the first infusion, ie, by study Day 8. | Day 1 to Day 8 | 
| Time to an Alternative Response | A study participant had a response if their platelets increased to ≥ 30x10^9/L and to at least double the baseline platelet count, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding. | Day 1 to Day 22 | 
| Time to a Complete Response | A study participant had a complete response if their platelets increased to ≥ 100x10^9/L, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding. | Day 1 to Day 22 | 
| Duration of a Response | The duration of a response was defined as the time from when a response was achieved until the platelet count fell below 50x10^9/L. | Day 1 to Day 22 | 
| Duration of an Alternative Response | The duration of an alternative response was defined as the time from when an alternative response was achieved until the platelet count fell below 50x10^9/L. | Day 1 to Day 22 | 
| Duration of a Complete Response | The duration of a complete response was defined as the time from when a complete response was achieved until the platelet count fell below 50x10^9/L. | Day 1 to Day 22 | 
| Platelet Count by Visit | The platelet count at each study visit are presented. | Day 1 to Day 22 | 
| Maximum Platelet Count | The maximum platelet count achieved during the study is presented. | Day 1 to Day 22 | 
| Percentage of Responders Who Achieved a Normal Platelet Count | The percentage of responders who achieved a normal platelet count is presented. | Day 1 to Day 22 | 
| Bleeding Intensity | The percentage of participants with various intensities of overall bleeding, epistaxis (bleeding of the nose), oral bleeding, and skin bleeding graded as none, minor, mild, moderate, or severe at Baseline and Day 22 are reported. | Day 1 to Day 22 | 
| Percentage of Participants Who Achieved a Platelet Count > 30x10^9/L | The percentage of participants who achieved a platelet count > 30x10^9/L within 1 and 2 days after infusion is reported. | Day 1 to Day 2 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ITP, Primary immune thrombocytopenia