search
Back to results

Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (EU Extension Study)

Primary Purpose

Primary Immunodeficiency (PID)

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
IgPro20
Sponsored by
CSL Behring
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Immunodeficiency (PID) focused on measuring Immune globulin subcutaneous, SCIG, primary immunodeficiency, PID

Eligibility Criteria

2 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 the European Society for Immunodeficiencies (ESID), or X-linked agammaglobulinemia (XLA) as defined by PAGID and ESID, or autosomal recessive agammaglobulinemia who have participated in the study ZLB06_001CR and who have tolerated IgPro well
  • Written informed consent

Exclusion Criteria:

  • Hypoalbuminemia, protein-losing enteropathies, and any proteinuria (defined as total urine protein concentration > 0.2g/L)
  • Other significant medical conditions that could increase the risk to the subject
  • Females who are pregnant, breast feeding or planning a pregnancy during the course of the study
  • Participation in a study with an investigational medicinal product within three months prior to enrollment, except for ZLB06_001CR
  • Evidence of uncooperative attitude
  • Any condition that is likely to interfere with evaluation of the IMP or satisfactory conduct of the study
  • Subjects who are employees at the investigational site, relatives or spouse of the investigator

Sites / Locations

  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site
  • Study Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

IgPro20

Arm Description

Subcutaneous (SC) administration by the subject/parent/guardian with the planned weekly dose of IgPro20 to be the same as the subject's last dose recommended by the investigator in study ZLB06_001CR (NCT00542997).

Outcomes

Primary Outcome Measures

Total Serum IgG Trough Levels
The IgG trough values per subject were aggregated to a median value, and then median values across subjects were summarized using descriptive statistics.

Secondary Outcome Measures

Annualized Rate of Clinically Documented Serious Bacterial Infections (SBIs)
The annualized rate was based on the total number of SBIs and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days. Potential SBIs included bacterial pneumonia, bacteremia and septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess. If an adverse event (AE) was identified as a potential SBI, the AE was adjudicated by the Medical Monitor and Investigator to determine if the event fulfilled the predefined criteria for SBIs.
Annualized Rate of Infection Episodes
The annualized rate was based on the total number of infection episodes occurring during the study divided by the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days.
Number of Infection Episodes
Total number of infections for the specified analysis population
Annualized Rate of Days Out of Work / School / Kindergarten / Day Care or Unable to Perform Normal Activities Due to Infections
The annualized rate was based on the total number of days out of work / school / kindergarten / day care or inability to perform normal activities due to infection, and the total number of subject diary days for all subjects in the specified analysis population and adjusted to 365 days.
Number of Days Out of Work / School / Kindergarten / Day Care or Unable to Perform Normal Activities Due to Infections
Total number of days out of work / school / kindergarten / day care or unable to perform normal activities due to infections, for the specified analysis population
Annualized Rate of Hospitalization Due to Infections
The annualized rate was based on the total number of days of hospitalization due to infections and the total number of subject diary days for all subjects in the specified analysis population and adjusted to 365 days.
Number of Days of Hospitalization Due to Infections
Total number of days of hospitalization due to infections for the specified analysis population
Use of Antibiotics for Infection Prophylaxis and Treatment
Annualized rate of days with antibiotics for infection prophylaxis and treatment. The annualized rate was based on the total number of days of antibiotic use for infection prophylaxis and treatment in the efficacy period, and the total number of subject study days for all subjects in the specified analysis population, and adjusted to 365 days.
Health Related Quality of Life (Short Form 36 Health Survey)
The Short Form 36 Health Survey (SF-36) is a 36-item questionnaire that measures generic health concepts that are relevant across age, disease, and treatment groups. The questions are grouped into eight domains: physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role-emotional, and mental health. Scores range from 0 to 100, with higher scores indicating a better health state.
Clinically Relevant Changes in Vital Signs From Baseline to the Completion Visit.
The total number of subjects with clinically relevant changes in vital signs from baseline to the completion visit. Vital signs included heart rate, systolic blood pressure, diastolic blood pressure, and body temperature.
Clinically Significant Abnormal Changes in Routine Laboratory Parameters Between Baseline and the Completion Visit.
The total number of subjects with clinically significant abnormal changes in routine laboratory parameters between baseline and the completion visit. Routine laboratory parameters included haematology, serum chemistry and urinalysis.
Rate, Severity and Relatedness of Any Adverse Events (AEs) Per Infusion
The rate of AEs was the number of AEs over the number of infusions administered. Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities. At least possibly related AEs included possibly related AEs, probably related AEs, and related AEs.

Full Information

First Posted
September 11, 2008
Last Updated
March 5, 2014
Sponsor
CSL Behring
search

1. Study Identification

Unique Protocol Identification Number
NCT00751621
Brief Title
Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (EU Extension Study)
Official Title
A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency (IgPro20 EU Extension Study)
Study Type
Interventional

2. Study Status

Record Verification Date
December 2012
Overall Recruitment Status
Completed
Study Start Date
August 2008 (undefined)
Primary Completion Date
December 2011 (Actual)
Study Completion Date
December 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CSL Behring

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
This study is a continuation of the study ZLB06_001CR with the objective of assessing efficacy, tolerability, safety of IgPro, as well as long-term health-related quality of life in patients with PID.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Immunodeficiency (PID)
Keywords
Immune globulin subcutaneous, SCIG, primary immunodeficiency, PID

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Actual)

8. Arms, Groups, and Interventions

Arm Title
IgPro20
Arm Type
Experimental
Arm Description
Subcutaneous (SC) administration by the subject/parent/guardian with the planned weekly dose of IgPro20 to be the same as the subject's last dose recommended by the investigator in study ZLB06_001CR (NCT00542997).
Intervention Type
Biological
Intervention Name(s)
IgPro20
Other Intervention Name(s)
IgG with Proline (IgPro), Hizentra
Primary Outcome Measure Information:
Title
Total Serum IgG Trough Levels
Description
The IgG trough values per subject were aggregated to a median value, and then median values across subjects were summarized using descriptive statistics.
Time Frame
Up to 42 months
Secondary Outcome Measure Information:
Title
Annualized Rate of Clinically Documented Serious Bacterial Infections (SBIs)
Description
The annualized rate was based on the total number of SBIs and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days. Potential SBIs included bacterial pneumonia, bacteremia and septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess. If an adverse event (AE) was identified as a potential SBI, the AE was adjudicated by the Medical Monitor and Investigator to determine if the event fulfilled the predefined criteria for SBIs.
Time Frame
Up to 42 months
Title
Annualized Rate of Infection Episodes
Description
The annualized rate was based on the total number of infection episodes occurring during the study divided by the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days.
Time Frame
Up to 42 months
Title
Number of Infection Episodes
Description
Total number of infections for the specified analysis population
Time Frame
Up to 42 months
Title
Annualized Rate of Days Out of Work / School / Kindergarten / Day Care or Unable to Perform Normal Activities Due to Infections
Description
The annualized rate was based on the total number of days out of work / school / kindergarten / day care or inability to perform normal activities due to infection, and the total number of subject diary days for all subjects in the specified analysis population and adjusted to 365 days.
Time Frame
Up to 42 months
Title
Number of Days Out of Work / School / Kindergarten / Day Care or Unable to Perform Normal Activities Due to Infections
Description
Total number of days out of work / school / kindergarten / day care or unable to perform normal activities due to infections, for the specified analysis population
Time Frame
Up to 42 months
Title
Annualized Rate of Hospitalization Due to Infections
Description
The annualized rate was based on the total number of days of hospitalization due to infections and the total number of subject diary days for all subjects in the specified analysis population and adjusted to 365 days.
Time Frame
Up to 42 months
Title
Number of Days of Hospitalization Due to Infections
Description
Total number of days of hospitalization due to infections for the specified analysis population
Time Frame
Up to 42 months
Title
Use of Antibiotics for Infection Prophylaxis and Treatment
Description
Annualized rate of days with antibiotics for infection prophylaxis and treatment. The annualized rate was based on the total number of days of antibiotic use for infection prophylaxis and treatment in the efficacy period, and the total number of subject study days for all subjects in the specified analysis population, and adjusted to 365 days.
Time Frame
Up to 42 months
Title
Health Related Quality of Life (Short Form 36 Health Survey)
Description
The Short Form 36 Health Survey (SF-36) is a 36-item questionnaire that measures generic health concepts that are relevant across age, disease, and treatment groups. The questions are grouped into eight domains: physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role-emotional, and mental health. Scores range from 0 to 100, with higher scores indicating a better health state.
Time Frame
At baseline and at the last available post-baseline observation for each subject (up to 42 months)
Title
Clinically Relevant Changes in Vital Signs From Baseline to the Completion Visit.
Description
The total number of subjects with clinically relevant changes in vital signs from baseline to the completion visit. Vital signs included heart rate, systolic blood pressure, diastolic blood pressure, and body temperature.
Time Frame
At baseline (data either from Infusion 40 or the completion visit of study ZLB06_001CR), and at completion (up to 42 months).
Title
Clinically Significant Abnormal Changes in Routine Laboratory Parameters Between Baseline and the Completion Visit.
Description
The total number of subjects with clinically significant abnormal changes in routine laboratory parameters between baseline and the completion visit. Routine laboratory parameters included haematology, serum chemistry and urinalysis.
Time Frame
At baseline (data either from Infusion 40 or the completion visit of study ZLB06_001CR), and at completion (up to 42 months).
Title
Rate, Severity and Relatedness of Any Adverse Events (AEs) Per Infusion
Description
The rate of AEs was the number of AEs over the number of infusions administered. Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities. At least possibly related AEs included possibly related AEs, probably related AEs, and related AEs.
Time Frame
Up to 42 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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 the European Society for Immunodeficiencies (ESID), or X-linked agammaglobulinemia (XLA) as defined by PAGID and ESID, or autosomal recessive agammaglobulinemia who have participated in the study ZLB06_001CR and who have tolerated IgPro well Written informed consent Exclusion Criteria: Hypoalbuminemia, protein-losing enteropathies, and any proteinuria (defined as total urine protein concentration > 0.2g/L) Other significant medical conditions that could increase the risk to the subject Females who are pregnant, breast feeding or planning a pregnancy during the course of the study Participation in a study with an investigational medicinal product within three months prior to enrollment, except for ZLB06_001CR Evidence of uncooperative attitude Any condition that is likely to interfere with evaluation of the IMP or satisfactory conduct of the study Subjects who are employees at the investigational site, relatives or spouse of the investigator
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Program Director, Clinical R&D
Organizational Affiliation
CSL Behring
Official's Role
Study Director
Facility Information:
Facility Name
Study Site
City
Paris
ZIP/Postal Code
75743
Country
France
Facility Name
Study Site
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Facility Name
Study Site
City
Freiburg
ZIP/Postal Code
79095
Country
Germany
Facility Name
Study Site
City
Leipzig
ZIP/Postal Code
04129
Country
Germany
Facility Name
Study Site
City
Mainz
ZIP/Postal Code
55131
Country
Germany
Facility Name
Study Site
City
Warsaw
Country
Poland
Facility Name
Study Site
City
Cluj-Napoca
ZIP/Postal Code
400162
Country
Romania
Facility Name
Study Site
City
Timisoara
ZIP/Postal Code
300011
Country
Romania
Facility Name
Study Site
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Study Site
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Facility Name
Study Site
City
Göteborg
ZIP/Postal Code
41685
Country
Sweden
Facility Name
Study Site
City
Bern
ZIP/Postal Code
3010
Country
Switzerland
Facility Name
Study Site
City
London
ZIP/Postal Code
EC1A7BE
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
24412910
Citation
Jolles S, Borte M, Nelson RP Jr, Rojavin M, Bexon M, Lawo JP, Wasserman RL. Long-term efficacy, safety, and tolerability of Hizentra(R) for treatment of primary immunodeficiency disease. Clin Immunol. 2014 Feb;150(2):161-9. doi: 10.1016/j.clim.2013.10.008. Epub 2013 Oct 26.
Results Reference
result
Links:
URL
http://www.cslbehring.com/clinical-trials/contact-us.htm?registryRefNum=NCT00751621&registryName=ctgov
Description
Click here to request more information about this study

Learn more about this trial

Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (EU Extension Study)

We'll reach out to this number within 24 hrs