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

Immunologic Deficiency Syndromes Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00811174
• Other study ID #: GAM10-03
• Status: Terminated
• Phase: Phase 3
• First received: December 17, 2008
• Last updated: May 6, 2013
• Start date: January 2009

Study information

• Verified date: May 2013
• Source: Octapharma
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationGermany: Paul-Ehrlich-Institut
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Terminated
• Enrollment: 5
• Est. primary completion date: September 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 2 Years to 75 Years

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

Study Design

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

Related Conditions & MeSH terms

• Immunologic Deficiency Syndromes

Intervention

Drug:
• Octagam 10%
300-600 mg/kg every 21 (+/- 3 days) to 28 days (+/- 3 days)

Locations

Country	Name	City	State
Austria	Contact Barbara Pyringer at Octapharma/ Vienna/ Austria for information	Vienna

Sponsors (1)

Lead Sponsor	Collaborator
Octapharma

Country where clinical trial is conducted

Austria, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse events		During infusion or within 72 hours after end of infusion	Yes
Primary	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	No
Secondary	Vital signs		during each treatment	Yes
Secondary	laboratory parameters (hematology, clinical chemistry, direct Coombs test and urin analysis)		at each treatment date (every three to four weeks)	Yes
Secondary	Assessment of viral safety		Every three months	Yes
Secondary	Pre-next-dose levels of serum total IgG		before each treatment	No
Secondary	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	No
Secondary	Therapeutic efficacy (number of infections, number of missed days at school/ work, number of hospitalisation days, and use of antibiotics)		12 months	No