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

Condition(s) targeted: Immunologic Deficiency Syndromes

Intervention: Octagam 10% (Drug)

Phase: Phase 3

Status: Terminated

Sponsored by: Octapharma

Official(s) and/or principal investigator(s):
Wolfgang Frenzel, Dr., Study Director, Affiliation: Octapharma

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.

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

Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome:

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

Secondary outcome:

Vital signs

laboratory parameters (hematology, clinical chemistry, direct Coombs test and urin analysis)

Assessment of viral safety

Pre-next-dose levels of serum total IgG

Pre-next-dose levels of IgG subclasses (IgG1, IgG2, IgG3, IgG4) and pre-next-dose levels of specific antibodies against defined infectious agents

Therapeutic efficacy (number of infections, number of missed days at school/ work, number of hospitalisation days, and use of antibiotics)

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.

Minimum age: 2 Years. Maximum age: 75 Years. Gender(s): Both.

Criteria:

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

Contact Barbara Pyringer at Octapharma/ Vienna/ Austria for information, Vienna 1100, Austria

Starting date: January 2009
Last updated: May 6, 2013