Study of PlasmaCap IG in Adults and Children With PIDD

Study Overview
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Brief Summary
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The purpose of this study is to investigate the efficacy, safety, tolerability, and pharmacokinetic profile of the investigational medicinal product (IMP) and to determine, on the basis of historical control data, how it compares with other 10% intravenous immunoglobulin (IGIV) products currently licensed in North America for the treatment of subjects with primary immune deficiency diseases (PIDD).

Official Title
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A Prospective, Open-Label, Multicenter Study of the Efficacy, Safety, Tolerability, and Pharmacokinetics of Therapure PlasmaCap IG in Adults and Children With Primary Immune Deficiency Diseases

Conditions
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Primary Immune Deficiency Diseases (PIDD)

Intervention / Treatment
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* Biological: 10% IGIV


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subject has a confirmed clinical diagnosis of a PIDD, which requires treatment with IGIV: Subject/guardian has provided written informed consent (and assent, as applicable). Subject is between the ages of 2 and 70 years. Subject has received regular IGIV therapy at 21- or 28-day (±4 days) intervals for at least three consecutive months at a dose between 300-900 mg/kg/month prior to Screening or; Subject has received commercial SCIG at a dose of 300-900 mg/kg/month on any dosing schedule for at least 12 consecutive weeks prior to Screening. Subjects on SCIG must have received and tolerated IGIV treatment prior to SCIG treatment. Subject has a documented trough of ≥500 mg/dL in the 6 months prior to screening. Females of childbearing potential must be willing to use an effective form of birth control (eg, oral contraceptives) for the duration of the study, per IRB/REB guidelines. Subject agrees to comply with the requirements of the protocol. Exclusion Criteria: Subject has secondary immunodeficiency. Subject has history of thrombotic events, such as deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism, etc within the year prior to screening. Subject has had an immune globulin associated arterial or venous thrombotic/thromboembolic event (TEE) within 7 days of infusion or a TEE that is not associated with an immune globulin within one year of screening. Subject has received blood products (except for IGIV, SCIG, or albumin) within 6 months of screening. Subject has anemia (≤8.5 g/dL). Subject has levels of alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >3.0 times the upper limit of normal (ULN). Subject has severe neutropenia (≤1000 neutrophils per mm3). Subject is receiving other immunosuppressive or immunomodulatory drugs or chemotherapy. Subject is taking or has taken within the four weeks prior to screening prednisone at ≥0.15 mg/kg/day for more than 10 days. Subject has ever had a severe anaphylactic reaction to a blood or IgG product. Subject has lymphoid malignancy, leukemia, or any other history of malignancy within the past five years, except squamous cell or basal cell carcinoma of the skin (not melanoma). Subject has hypoalbuminemia, protein-losing enteropathy, or proteinuria greater than 300 mg/24 hours except for subjects with documented orthostatic proteinuria. Subject has immunoglobulin A (IgA) deficiency with known antibodies to IgA. Female who is pregnant, breastfeeding, or planning a pregnancy during the course of the study (women who become pregnant during the study will be withdrawn from the study). Any condition that is likely to interfere with evaluation of IMP or satisfactory conduct of the trial in the PI's opinion. Subjects who may not be compliant or have a history of non-compliance in the opinion of the PI.

Ages Eligible for Study
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Minimum Age: 2 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: Open-Label 10% IGIV<br>IMP will be administered every 21 or 28 days in accordance with the subject's weekly regimen at screening for a period of 12 months. Subjects on a 21-day regimen will receive approximately 17 infusions, and subjects on a 28-day regimen will receive approximately 13 infusions. The starting dose will be the previous IGIV dose or a dose calculated from the previous SCIG dose up to a maximum of 900 mg/kg/mo. | Biological: 10% IGIV<br>* 300-900 mg/kg<br>* Other names: Human Immunoglobulin;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean acute Serious Bacterial Infection (SBI) rate | The primary efficacy objective of the study is to demonstrate the efficacy of the IMP by determining that the mean annual acute SBI rate (as defined in Appendix 20.1) is statistically significantly lower than one infection per subject per year. | 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Immunoglobulin G (IgG) trough concentration | The average serum total IgG trough concentrations prior to each infusion | up to 12 months per subject | 
| Days unable to perform daily activities | The number of days unable to perform daily activities | up to 12 months per subject | 
| Therapeutic IgG levels | The ability of the IMP to maintain stable, therapeutic IgG levels | up to 12 months per subject |