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

Phase 3

Terminated

Brief Summary: 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: 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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
Octagam 10%	Octagam 10%	-

Primary Outcome Measures

Name	Time	Method
Adverse Events	During infusion or within 72 hours after end of infusion	Occurrence of Adverse Events
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	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

Secondary Outcome Measures

Name	Time	Method
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
Assessment of Viral Safety	Every three months
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)