Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy

Phase 3

Completed

• Conditions: Autosomal Recessive Agammaglobulinemia; Common Variable Immunodeficiency; X-linked Agammaglobulinemia
• Interventions: Biological: Human Normal Immunoglobulin for Subcutaneous Administration (IGSC)

• Registration Number: NCT00542997
• Lead Sponsor: CSL Behring

Brief Summary

The objective of this study is to assess the efficacy, tolerability, safety and pharmacokinetics of IgPro20 in patients with primary humoral immunodeficiency (PID).

Detailed Description

This study consisted of a 12-week wash-in/wash-out period followed by a 28-week efficacy period. During the 28-week efficacy period, subjects visited the study site at least every 4 weeks for efficacy and safety evaluations and additionally recorded details regarding IgPro20 dose and certain aspects of efficacy and safety in a diary. Pharmacokinetic (PK) parameters were assessed in a sub-group of subjects during 1 treatment interval at steady-state (Week 28 ± 1).

Study Timeline

• Study Start: 2007-09
• Study First Submitted: 2007-10-11
• Study First Submitted QC: 2007-10-11
• Study First Posted: 2007-10-12
• Primary Completion: 2009-08
• Study Completion: 2009-08
• Results First Submitted: 2011-02-08
• Results First Submitted QC: 2011-02-17
• Results First Posted: 2011-03-15
• Status Verified: 2011-08
• Last Update Submitted: 2011-08-02
• Last Update Posted: 2011-09-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 51

Inclusion Criteria

• Subjects with primary humoral immunodeficiency, namely with a diagnosis of Common Variable Immunodeficiency (CVID) as defined by the Pan-American Group for Immunodeficiency (PAGID) and European Society for Immunodeficiencies (ESID), X-linked agammaglobulinemia (XLA) as defined by PAGID and ESID, or Autosomal Recessive Agammaglobulinemia
• Chest X-ray or CT scan obtained within 1 year prior to enrolment

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Exclusion Criteria

• Newly diagnosed PID, i.e. subjects who have not previously received immunoglobulin replacement therapy
• Ongoing serious bacterial infection at the time of screening
• Malignancies of lymphoid cells such as lymphocytic leukemia, Non-Hodgkin's lymphoma and immunodeficiency with thymoma
• Allergic or other severe reactions to immunoglobulins or other blood products associated with high anti-IgA

Additional criteria may apply and examination by an investigator is required to determine eligibility.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
IgPro20	Human Normal Immunoglobulin for Subcutaneous Administration (IGSC)	-

Primary Outcome Measures

Name	Time	Method
Total Serum IgG Trough Levels	Up to 6 months prior to first IgPro20 treatment (Pre-study treatment) and Week 12 to 17 (IgPro20 treatment)	Total IgG trough levels for IgPro20 treatment at steady state were compared with documented trough level data for IgG treatment received prior to enrolling in the study (either subcutaneous or intravenous IgG). For this purpose, 6 consecutive IgPro20 trough values (obtained prior to infusions 12 to 17) per subject were aggregated to the subject's median value and then median values across subjects were summarised using descriptive statistics. The same procedure was applied to pre-study treatment using the 3 most recent IgG trough values ≥ 5 g/L obtained prior to the first IgPro20 infusion.

Secondary Outcome Measures

Name	Time	Method
Annual Rate of Clinically Documented Serious Bacterial Infections (PPE Population)	Efficacy period: week 12 to week 40 after study start or to the completion visit	Serious bacterial infections (SBIs) included bacterial pneumonia, bacteraemia/septicaemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess. Diagnosis of the SBIs was based on the presence of predefined clinical signs and symptoms as well as on laboratory parameters.; The annual rate was calculated based on the total number of SBIs and the total number of study days during the efficacy period for all subjects in the PPE population and adjusted to 365 days.
Annual Rate of the Number of Days of Hospitalization Due to Infections	Efficacy period: week 12 to week 40 after study start or to the completion visit	The annual rate was calculated based on the total number of days of hospitalization due to infections in the efficacy period divided by the total number of days in the efficacy period for all subjects and adjusted to 365 days.
Annual Rate of Clinically Documented Serious Bacterial Infections (ITT Population)	Efficacy period: week 12 to week 40 after study start or to the completion visit	Serious bacterial infections (SBIs) included bacterial pneumonia, bacteraemia/septicaemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess. Diagnosis of the SBIs was based on the presence of predefined clinical signs and symptoms as well as on laboratory parameters.; The annual rate was calculated based on the total number of SBIs and the total number of study days during the efficacy period for all subjects in the ITT population and adjusted to 365 days.
Annual Rate of Infection Episodes	Efficacy period: week 12 to week 40 after study start or to the completion visit	The annual rate of episodes was calculated based on the total number of any infection type and the total number of study days during the efficacy period for all subjects in the ITT population and adjusted to 365 days.
Annual Rate of Days Out of Work / School / Kindergarten / Day Care or Unable to Perform Normal Activities Due to Infections	Efficacy period: week 12 to week 40 after study start or to the completion visit	The annual rate was calculated based on the total number of days out of work/school/kindergarten/day care or unable to perform normal activities due to infections in the efficacy period divided by the total number of days in the efficacy period for all subjects and adjusted to 365 days.
Annual Rate of Antibiotic Use for Infection Prophylaxis and Treatment	Efficacy period: week 12 to week 40 after study start or to the completion visit	The annual rate was calculated based on the total number of days of antibiotic use in the efficacy period divided by the total number of days in the efficacy period for all subjects and adjusted to 365 days.

Trial Locations

Locations (1)

Study site; 🇬🇧 London, United Kingdom