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

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

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.

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.

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