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.

Study Overview

• Status: Terminated
• Conditions: Immunologic Deficiency Syndromes
• Intervention / Treatment: Drug: Octagam 10%

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.

• Study Type: Interventional
• Enrollment (Actual): 5
• Phase: Phase 3

Contacts and Locations

Study Locations

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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Octagam 10%	Drug: Octagam 10%; 300-600 mg/kg every 21 (+/- 3 days) to 28 days (+/- 3 days)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Events	Occurrence of Adverse Events	During infusion or within 72 hours after end of infusion
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	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

Secondary Outcome Measures

Outcome Measure	Time Frame
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)
Assessment of Viral Safety	Every three months
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

Collaborators and Investigators

• Sponsor: Octapharma
• Investigators: Study Director: Wolfgang Frenzel, Dr., Octapharma

Study record dates

Study Major Dates

• Study Start: January 1, 2009
• Primary Completion (Actual): September 1, 2010
• Study Completion (Actual): September 1, 2010

Study Registration Dates

• First Submitted: December 17, 2008
• First Submitted That Met QC Criteria: December 17, 2008
• First Posted (Estimate): December 18, 2008

Study Record Updates

• Last Update Posted (Actual): July 14, 2017
• Last Update Submitted That Met QC Criteria: June 16, 2017
• Last Verified: June 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Genetic Diseases, Inborn; Immunologic Deficiency Syndromes; Primary Immunodeficiency Diseases; Physiological Effects of Drugs; Immunologic Factors; Immunoglobulins, Intravenous; gamma-Globulins; Rho(D) Immune Globulin
• Other Study ID Numbers: GAM10-03