Clinical Study to Evaluate the Efficacy and Safety of Octagam 10% in Idiopathic Thrombocytopenic Purpura in Adults

Study Overview
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Brief Summary
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Octagam is a solvent/detergent-treated human normal immunoglobulin (IGIV) solution for intravenous administration. Octagam 5% is currently registered in about 80 countries. This study evaluated the efficacy and safety of Octagam 10% in Idiopathic Thrombocytopenic Purpura (ITP) in adults. As Octagam 10% is essentially similar to Octagam 5%, it is expected that Octagam 10% is as efficacious and safe (in respect to viral safety) as Octagam 5%.

Detailed Description
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The primary objective of the study was to investigate the efficacy of Octagam® 10% in correcting platelet count. The blood count as well as laboratory chemistry were checked repeatedly up to day 21. The secondary objective of the study was to investigate the safety of Octagam® 10%. Safety was assessed by monitoring vital signs, evaluating adverse events, assessing laboratory parameters, and by viral safety testing.

Official Title
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Clinical Study to Evaluate the Efficacy and Safety of Octagam® 10% in Idiopathic Thrombocytopenic Purpura in Adults

Conditions
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Immune Thrombocytopenic Purpura

Intervention / Treatment
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* Drug: Octagam 10%


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Diagnosis of Idiopathic Thrombocytopenic Purpura (ITP) according to standard criteria. Platelet count ≤ 20 x 10^9/L. Key Exclusion Criteria: Chronic refractory ITP patients. Thrombocytopenia secondary to other diseases, or drug related thrombocytopenia. Administration of IGIV, anti-D, or other platelet enhancing drugs within 30 days before enrollment. Administration of thrombocyte concentrates within 72 hours before baseline. Experimental treatment (eg, rituximab) within 3 months before enrollment. Prophylactic preoperative treatment for elective splenectomy. Severe liver or kidney disease. Pregnant or nursing female. History of hypersensitivity to blood or plasma derived products. Emergency operation. Live viral vaccination within the last month prior to study entry. Known IgA deficiency and antibodies against IgA.

Ages Eligible for Study
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Minimum Age: 18 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: Octagam 10% 1 g/kg/day<br>Participants received Octagam 10% (human normal immunoglobulin) 1 g/kg intravenously once a day for 2 days. | Drug: Octagam 10%<br>* Octagam 10% was supplied as a ready-to-use solution in glass bottles.<br>* Other names: Human normal immunoglobulin;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants With a Clinical Response | A clinical response is defined as an increase in platelet count to ≥ 50*10^9/L on any day from Day 2 to Day 7. | Day 2 to Day 7 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to Achieve a Clinical Response | A clinical response is defined as an increase in platelet count to ≥ 50*10^9/L on any day from Day 2 to Day 7. | Day 2 to Day 7 | 
| Maximum Platelet Count | Platelet count was assessed on Days 2 through 7 and on Days 14, 21, and 63. The maximum measured platelet count is reported. | Day 2 to the end of the study (Day 63) | 
| Duration of the Clinical Response | The duration of the clinical response was the number of days that the platelet count remained ≥ 50*10^9/L. Platelet count was assessed on Days 2 through 7 and on Days 14, 21, and 63. A conservative method was used to calculate the duration of the clinical response. For example, if the platelet count was ≥ 50*10^9/L on Day 7 and dropped below 50*10^9/L at Day 14, Day 7 was used as the last day to calculate the duration of the clinical response. The same procedure was used if the platelet count dropped below 50*10^9/L at Day 21 from Day 14 or Day 63 from Day 21. | Day 2 to the end of the study (Day 63) | 
| Percentage of Participants With None, Minor, Mild, or Moderate Bleeding at Day 7 | The investigator evaluated the severity of bleeding using the following rating scale: None (definitely no haemorrhage of any kind), Minor (few petechiae [≤ 100 total] and/or ≤ 5 small bruises [≤ 3 cm diameter], no mucosal bleeding), Mild (many petechiae [> 100 total] and/or > 5 large bruises [> 3 cm diameter], no mucosal bleeding), Moderate (overt mucosal bleeding [epistaxis, gum bleeding, oropharyngeal blood blisters, menorrhagia, gastrointestinal bleeding, etc] that does not require immediate medical attention or intervention). | Day 7 |