Clinical trial NCT00811174: Efficacy, Safety and Kinetics Study of Octagam 10% 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 type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Immunologic Deficiency Syndromes
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2008-12-18
Enrollment: 5
Sex: ALL
Minimum age (years): 2
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Adverse Events
timeFrame: During infusion or within 72 hours after end of infusion
measure: 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
timeFrame: after 6 months of treatment
Secondary outcomes:
measure: Vital Signs
timeFrame: during each treatment
measure: Laboratory Parameters (Hematology, Clinical Chemistry, Direct Coombs Test and Urin Analysis)
timeFrame: at each treatment date (every three to four weeks)
measure: Assessment of Viral Safety
timeFrame: Every three months
measure: Pre-next-dose Levels of Serum Total IgG
timeFrame: before each treatment
measure: Pre-next-dose Levels of IgG Subclasses (IgG1, IgG2, IgG3, IgG4) and Pre-next-dose Levels of Specific Antibodies Against Defined Infectious Agents
timeFrame: before treatement 10 and 13 (of 13 or 17 treatments) and at the end
measure: Therapeutic Efficacy (Number of Infections, Number of Missed Days at School/ Work, Number of Hospitalisation Days, and Use of Antibiotics)
timeFrame: 12 months
Other outcomes:
Locations:
city: Vienna
country: Austria
facility: Contact Barbara Pyringer at Octapharma/ Vienna/ Austria for information
Study officials:
affiliation: Octapharma
name: Wolfgang Frenzel, Dr.
role: STUDY_DIRECTOR
References: