Efficacy, Safety and Kinetics Study of Octagam 10% in Primary Immunodeficiency Diseases

Study Overview
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Brief Summary
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Octagam is a human normal immunoglobulin (IGIV) solution for intravenous administration. Octagam 5% is currently registered in more than 60 countries. This study will evaluate the efficacy, safety and the kinetics of Octagam 10% for replacement therapy in primary immunodeficiency diseases.

Detailed Description
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The primary objective of the study is to investigate the safety of Octagam 10% in replacement therapy in PID and to compare the pharmacokinetic profile of Octagam 10% with that of the previously used Octagam 5%. The secondary objective is to investigate the efficacy of Octagam 10% in replacement therapy in PID by monitoring the rate of occurence of serious bacterial infections, the rate of other infections, the trough (pre-next-dose) levels of total serum IgG and IgG subclasses (IgG1, IgG2, IgG3 and IgG4), the trough (pre-next-dose) levels of selected antigen specific antibodies, the use of antibiotics, the rate of absence from school/ work, and the number of days in hospital.

Official Title
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Clinical Study to Evaluate the Efficacy, Safety and Kinetics of Octagam 10% for Replacement Therapy in Primary Immunodeficiency Diseases

Conditions
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Immunologic Deficiency Syndromes

Intervention / Treatment
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* Drug: Octagam 10%


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Confirmed diagnosis of primary immunodeficiency (acc. WHO) Previous treatment with commercial Octagam 5% every 3-4 weeks for at least 6 infusions intervals Documented IgG trough levels of the two previous infusions before enrollment with a value of at least 5.5 g/L for both Exclusion Criteria: Acute infection requiring intravenous antibiotic treatment within two weeks before screening Exposure to blood or any blood product or derivative other than commercially available Octagam 5%, within the past 3 months History of hypersensitivity to blood or plasma derived products Requirement of any routine premedication for IGIV treatment History of congenital impairment of pulmonary function Severe liver function impairment Severe renal function impairment or predisposition for acute renal failure History of autoimmune haemolytic anaemia History of diabetes mellitus Congestive heart failure NYHA III or IV Non-controlled arterial hypertension History of DVT or thrombotic complications with IGIV treatment Known infection with HIV, HCV or HBV Treatment with steroids, immunosuppressive or immunomodulatory drugs Planned vaccination during study period Pregnant or nursing woman

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 75 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%<br> | Drug: Octagam 10%<br>* 300-600 mg/kg every 21 (+/- 3 days) to 28 days (+/- 3 days)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse Events | Occurrence of Adverse Events | During infusion or within 72 hours after end of infusion | 
| Pharmacokinetics of Serum Total IgG and IgG Subclasses (IgG1, IgG2, IgG3 and IgG4) and Pharmacokinetics of Specific Antibodies Against Defined Infectious Agents Comparing Octagam 5% Treatment With Octagam 10% Treatment | Pharmacokinetics of serum total IgG and IgG subclasses (IgG1, IgG2, IgG3 and IgG4) and pharmacokinetics of specific antibodies against defined infectious agents comparing Octagam 5% treatment with Octagam 10% treatment | after 6 months of treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Vital Signs |  | during each treatment | 
| Laboratory Parameters (Hematology, Clinical Chemistry, Direct Coombs Test and Urin Analysis) |  | at each treatment date (every three to four weeks) | 
| Assessment of Viral Safety |  | Every three months | 
| Pre-next-dose Levels of Serum Total IgG |  | before each treatment | 
| Pre-next-dose Levels of IgG Subclasses (IgG1, IgG2, IgG3, IgG4) and Pre-next-dose Levels of Specific Antibodies Against Defined Infectious Agents |  | before treatement 10 and 13 (of 13 or 17 treatments) and at the end | 
| Therapeutic Efficacy (Number of Infections, Number of Missed Days at School/ Work, Number of Hospitalisation Days, and Use of Antibiotics) |  | 12 months |