A Study to Find How Safe and Effective GAMMAPLEX® is in Subjects With Chronic Idiopathic Thrombocytopenic Purpura (ITP)

Study Overview
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Brief Summary
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To determine if GAMMAPLEX raises the platelet count of subjects with chronic ITP to a threshold of 50 x 109/L, similar to that of published response >60%. Also to assess the safety of GAMMAPLEX and determine if platelet counts are maintained at 50 x 109/L in subjects with chronic ITP for.

Detailed Description
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The primary objective is to determine if BPL's GAMMAPLEX raises the platelet count of subjects with chronic ITP to a threshold of 50 x 109/L, similar to that of published response >60%. The secondary objectives are: 1) to determine the safety of GAMMAPLEX at the dosage used in this study. 2) to determine if GAMMAPLEX maintains platelet counts of ³ 50 x 109/L in subjects with chronic ITP for a period of time similar to that of a published data.

Official Title
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A Phase III, Multicenter, Open-Label Study To Evaluate the Efficacy and Safety of GAMMAPLEX® in Chronic Idiopathic Thrombocytopenic Purpura

Conditions
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Chronic Idiopathic Thrombocytopenic Purpura

Intervention / Treatment
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* Biological: Gammaplex, intravenous immunoglobulin


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males and females aged between 18 and 70 years. Confirmed diagnosis of chronic ITP of at least 6 months duration. Platelet count of less than or equal to 20 x 10 9/L at enrollment. Absence of other conditions that, in the opinion of the investigator, could cause thrombocytopenia. If subjects were treated with corticosteroids the treatment regimen/dose must have been stable (for a minimum of 2 weeks before screening). The dose of corticosteriod or other immunosuppressant should have remained constant until Day 32. 6) If subjects were being treated with cyclophosphamide, azathioprine or attenuated androgens, the treatment regimen and dose must have been stable for a minimum of 2 months before Day 1. The dose of corticosteriod or other immunosuppressant should have remained constant until Day 32. 7) Splenectomized subjects and both Rh(D)+ and Rh(D)- subjects may be included. 8) The subject has signed an informed consent form (subjects must be at least 18 years old), and/or the subject's legal guardian has signed the informed consent form if indicated 9) If a subject is a female of child-bearing potential, she must have a negative result on a urine-based HCG pregnancy test. 10) If a subject is a female who is or becomes sexually active, she must practice contraception by using a method of proven reliability for the duration of the study. Exclusion Criteria: A history of any severe or anaphylactic reaction to blood or any blood-derived product, or any severe reaction to IGIV or any other IgG preparation. Intolerance to any component of the investigational product. Received any live virus vaccine within the last 3 months prior to Day1. Received an IGIV preparation within 1 month prior to Day 1. Were currently receiving, or has received, any investigational agent within the 1 month prior to Day 1. Received any blood, blood product, or blood derivative within the 1 month prior to Day 1. Received Rituximab within the 3 months before Day 1. Pregnant or nursing. Tested positive for any of the following at screening: HBsAg, NAT for HCV, NAT for HIV, Antibodies to HCV or HIV 1 or 2. Had levels greater than 2.5 times the upper limit of normal at screening, as defined by the central laboratory, of alanine aminotransferase or aspartate aminotransferase. Had severe renal impairment (defined as serum creatinine greater than 2 times the upper limit of normal or BUN greater than 2.5 times the upper limit of normal for the range of the laboratory doing the analysis); on dialysis; a history of acute renal failure. Known to have abused alcohol, opiates, psychotropic agents, or other chemicals or drugs within the past 12 months. History of deep vein thrombosis (DVT) or thrombotic complications of IGIV therapy. Any history or sign of hyperviscosity, transient ischemic attack (TIA), stroke, other thromboembolic event, or unstable angina. Suffered from any acute or chronic medical conditions (e.g., renal disease or predisposing conditions for renal disease, coronary artery disease, or protein losing enteropathy) that, in the opinion of the investigator, may interfere with the conduct of the study. An acquired medical condition, such as chronic lymphocytic leukemia, lymphoma, multiple myeloma, chronic or recurrent neutropenia (defined as an absolute neutrophil count (ANC) < 1 x 109/L). Non-controlled arterial hypertension (systolic blood pressure >160 mmHg and/or diastolic blood pressure >90 mmHg). Anemic (hemoglobin <10 g/dL) at screening. Unlikely to adhere to the protocol requirements of the study or is likely to be uncooperative.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 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: Gammaplex (intravenous immunoglobulin)<br> | Biological: Gammaplex, intravenous immunoglobulin<br>* Dosage form: Gammaplex® is a sterile liquid of 5 % w/v normal immunoglobulin. Gammaplex® contains 5 g/100 mL of human normal immunoglobulin (i.e. 50 g/L, of which virtually 100% is IgG). The first course of GAMMAPLEX will be administered as an intravenous infusion of 1 g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen may be administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX.<br>* Other names: Gammaplex;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The Number of Subjects With Chronic ITP Treated With Gammaplex Whose Platelet Count Reached a Threshold of 50 x 10^9/L. | The number of subjects with chronic ITP treated following treatment with Gammaplex who attained a platelet count of ≥ 50 x 10^9/L by Day 9. | 9 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The Safety of GAMMAPLEX at the Dosage Used in This Study. | The safety variables used to assess safety were the following: Adverse events The number and percent of infusions with at least 1 adverse event(AE) that occurs during an infusion or within 72 hours after the infusion stops Nature, severity, and frequency of AEs Suspected unexpected serious adverse reactions (SUSARs) Vital signs Clinical laboratory tests and Direct Coombs' Test Transmission of viruses Physical examination | AEs were documented from the date the informed consent form was signed until the End of Study visit on Day 90. | 
| Duration of Time That the Platelet Count of Subjects With Chronic ITP Treated With Gammaplex Remained ≥ 50 x 10^9/L. | Blood samples were collected to measure platelet counts and the duration of time for which the platelet count remained ≥50 x 10^9/L was measured. | Days 1, 2, 3, 5, 9, 14, 21, 32. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Idiopathic Thrombocytopenic Purpura, Bleeding disorders, Immune System and Disorders