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Extension Study of Subcutaneous Immunoglobulin Human in Patients With Primary Immunodeficiency (PID)

Primary Purpose

Primary Immune Deficiency

Status
Completed
Phase
Phase 3
Locations
United States
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 Immune Deficiency focused on measuring CVID, XLA

Eligibility Criteria

2 Years - 75 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Subjects with primary humoral immunodeficiency who have participated in the study ZLB04_009CR (NCT00419341), namely with a diagnosis of Common Variable Immunodeficiency (CVID) as defined by PAGID (Pan-American Group for Immunodeficiency) and ESID (European Society for Immunodeficiencies) or X-linked Agammaglobulinemia (XLA) as defined by PAGID and ESID
  • Women of childbearing potential must be using and agree to continue using medically approved contraception and must have a negative pregnancy test at screening
  • Written informed consent

Exclusion Criteria:

  • 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
  • Hypoalbuminemia, protein-losing enteropathies, and any proteinuria (defined by total urine protein concentration > 0.2 g/L)
  • Other significant medical conditions that could increase the risk to the patient
  • Females who are pregnant, breast-feeding or planning a pregnancy during the course of the study
  • A positive result at screening on any of the following viral markers: Human immunodeficiency virus (HIV), Hepatitis C virus (HCV) or Hepatitis B virus (HBV)
  • Aspartate aminotransferase (ASAT) or Alanine aminotransferase (ALAT) concentration > 2.5 times Upper Normal Limit (UNL) at Completion Visit of study ZLB04_009CR (NCT00419341)
  • Creatinine concentration > 1.5 times UNL at Completion Visit of study ZLB04_009CR (NCT00419341)
  • Participation in a study with an investigational product other than IgPro20 within 3 months prior to enrollment
  • Evidence of uncooperative attitude
  • Any condition that is likely to interfere with evaluation of the study drug or satisfactory conduct of the trial
  • Subjects who are employees at the investigational site, relatives or spouse of the investigator

Sites / Locations

  • Contact CSL Behring for facility details
  • Contact CSL Behring for facility details
  • Contact CSL Behring for facility details
  • Contact CSL Behring for facility details

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

IgPro20

Arm Description

The IgPro20 dose will be the same as in the previous pivotal study ZLB04_009CR (NCT00419341) infused subcutaneously weekly or twice a week (in the latter case, half of a weekly dose will be used)

Outcomes

Primary Outcome Measures

Annualized Rate of Serious Bacterial Infection (Intention-to-Treat Population)
The annualized rate was based on the total number of infections and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days. Acute serious bacterial infections included pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess.
Annualized Rate of Serious Bacterial Infection (Per-Protocol Efficacy Population)
The annualized rate was based on the total number of infections and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days. Acute serious bacterial infections included pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess.

Secondary Outcome Measures

Annualized Rate of Any Infection
The annualized rate was based on the total number of infections and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days.
Trough Levels of Total Immunoglobulin G (IgG) Serum Concentrations
Mean of individual median total IgG trough concentration.
Number of Days Out of Work / School / Kindergarten / Day Care or Inability to Perform Normal Activities Due to Infection
Annualized Rate of Days Out of Work / School / Kindergarten / Day Care or Inability to Perform Normal Activities Due to Infection
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 study days for all subjects in the specified analysis population and adjusted to 365 days.
Number of Days of Hospitalization Due to Infection
Annualized Rate of Hospitalization Due to Infection
The annualized rate was based on the total number of days of hospitalization due to infection and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days.
Use of Antibiotics for Infection Prophylaxis and Treatment
Annualized rate of days with antibiotics for infection prophylaxis and treatment.
Rate of All AEs by Relatedness and Severity
The rate of AEs was the number of AEs over the number of infusions administered. At least possibly related AEs included possibly related AEs, probably related AEs, and related AEs. Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.
Relatedness and Severity of All AEs (Percentage of Total AEs)
At least possibly related AEs included possibly related AEs, probably related AEs, and related AEs. Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.
Number of Subjects With Any Temporally Associated Adverse Event (AE) Within 24 or 72 Hours After an Infusion
AEs were considered temporally associated if they occurred between the start of infusion and within 24 or 72 hours after the end of infusion.
Rate of Temporally Associated AEs Within 24 or 72 Hours of an Infusion
The rate of AEs was the number of AEs over the number of infusions administered. AEs were considered temporally associated if they occurred between the start of infusion and within 24 or 72 hours after the end of infusion.
Number of Subjects Reporting Mild, Moderate, or Severe Local AEs
In addition to the standard MedDRA System Organ Class (SOC) AE assignments, the category of 'local reactions' was defined to provide the possibility for a combined analysis of local reactions and included AEs of infusion site oedema, infusion site reaction, injection site pain, injection site rash, and injection site reaction. Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.
Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Vital Signs
Vital signs included blood pressure (systolic and diastolic), heart rate, and body temperature.
Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Routine Laboratory Parameters
Routine laboratory parameters included hematology, blood chemistry, and urinalysis parameters.
Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Viral Safety Markers
Viral safety markers included human immunodeficiency virus (HIV)-1, HIV-2, hepatitis A virus (HAV), HBV, HCV, and parvovirus B19.

Full Information

First Posted
July 21, 2008
Last Updated
March 5, 2014
Sponsor
CSL Behring
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1. Study Identification

Unique Protocol Identification Number
NCT00719680
Brief Title
Extension Study of Subcutaneous Immunoglobulin Human in Patients With Primary Immunodeficiency (PID)
Official Title
A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency (PID)
Study Type
Interventional

2. Study Status

Record Verification Date
December 2012
Overall Recruitment Status
Completed
Study Start Date
June 2008 (undefined)
Primary Completion Date
June 2010 (Actual)
Study Completion Date
June 2010 (Actual)

3. Sponsor/Collaborators

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

4. Oversight

5. Study Description

Brief Summary
The purpose of this study is to determine whether a long-term use of a new human immunoglobulin G with proline (IgPro) is safe and effective in the treatment of primary immunodeficiency.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Immune Deficiency
Keywords
CVID, XLA

7. Study Design

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

8. Arms, Groups, and Interventions

Arm Title
IgPro20
Arm Type
Experimental
Arm Description
The IgPro20 dose will be the same as in the previous pivotal study ZLB04_009CR (NCT00419341) infused subcutaneously weekly or twice a week (in the latter case, half of a weekly dose will be used)
Intervention Type
Biological
Intervention Name(s)
IgPro20
Other Intervention Name(s)
IgG with Proline
Primary Outcome Measure Information:
Title
Annualized Rate of Serious Bacterial Infection (Intention-to-Treat Population)
Description
The annualized rate was based on the total number of infections and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days. Acute serious bacterial infections included pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess.
Time Frame
For the duration of the study, up to approximately 104 weeks
Title
Annualized Rate of Serious Bacterial Infection (Per-Protocol Efficacy Population)
Description
The annualized rate was based on the total number of infections and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days. Acute serious bacterial infections included pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess.
Time Frame
For the duration of the study, up to approximately 104 weeks
Secondary Outcome Measure Information:
Title
Annualized Rate of Any Infection
Description
The annualized rate was based on the total number of infections and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days.
Time Frame
For the duration of the study, up to approximately 104 weeks
Title
Trough Levels of Total Immunoglobulin G (IgG) Serum Concentrations
Description
Mean of individual median total IgG trough concentration.
Time Frame
Before infusion at Weeks 1, 24, 48, 72, and 96
Title
Number of Days Out of Work / School / Kindergarten / Day Care or Inability to Perform Normal Activities Due to Infection
Time Frame
For the duration of the study, up to approximately 104 weeks
Title
Annualized Rate of Days Out of Work / School / Kindergarten / Day Care or Inability to Perform Normal Activities Due to Infection
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 study days for all subjects in the specified analysis population and adjusted to 365 days.
Time Frame
For the duration of the study, up to approximately 104 weeks
Title
Number of Days of Hospitalization Due to Infection
Time Frame
For the duration of the study, up to approximately 104 weeks
Title
Annualized Rate of Hospitalization Due to Infection
Description
The annualized rate was based on the total number of days of hospitalization due to infection and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days.
Time Frame
For the duration of the study, up to approximately 104 weeks
Title
Use of Antibiotics for Infection Prophylaxis and Treatment
Description
Annualized rate of days with antibiotics for infection prophylaxis and treatment.
Time Frame
For the duration of the study, up to approximately 104 weeks
Title
Rate of All AEs by Relatedness and Severity
Description
The rate of AEs was the number of AEs over the number of infusions administered. At least possibly related AEs included possibly related AEs, probably related AEs, and related AEs. Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.
Time Frame
For the duration of the study, up to approximately 104 weeks
Title
Relatedness and Severity of All AEs (Percentage of Total AEs)
Description
At least possibly related AEs included possibly related AEs, probably related AEs, and related AEs. Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.
Time Frame
For the duration of the study, up to approximately 104 weeks
Title
Number of Subjects With Any Temporally Associated Adverse Event (AE) Within 24 or 72 Hours After an Infusion
Description
AEs were considered temporally associated if they occurred between the start of infusion and within 24 or 72 hours after the end of infusion.
Time Frame
Within 24 or 72 hours after each infusion
Title
Rate of Temporally Associated AEs Within 24 or 72 Hours of an Infusion
Description
The rate of AEs was the number of AEs over the number of infusions administered. AEs were considered temporally associated if they occurred between the start of infusion and within 24 or 72 hours after the end of infusion.
Time Frame
Within 24 or 72 hours after each infusion
Title
Number of Subjects Reporting Mild, Moderate, or Severe Local AEs
Description
In addition to the standard MedDRA System Organ Class (SOC) AE assignments, the category of 'local reactions' was defined to provide the possibility for a combined analysis of local reactions and included AEs of infusion site oedema, infusion site reaction, injection site pain, injection site rash, and injection site reaction. Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.
Time Frame
For the duration of the study, up to approximately 104 weeks
Title
Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Vital Signs
Description
Vital signs included blood pressure (systolic and diastolic), heart rate, and body temperature.
Time Frame
At weeks 1, 12, 24, 36, 48, 60, 72, 84, and 96
Title
Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Routine Laboratory Parameters
Description
Routine laboratory parameters included hematology, blood chemistry, and urinalysis parameters.
Time Frame
At Week 1, and study completion (approximately 104 weeks)
Title
Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Viral Safety Markers
Description
Viral safety markers included human immunodeficiency virus (HIV)-1, HIV-2, hepatitis A virus (HAV), HBV, HCV, and parvovirus B19.
Time Frame
At Week 1, and study completion (approximately 104 weeks)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Subjects with primary humoral immunodeficiency who have participated in the study ZLB04_009CR (NCT00419341), namely with a diagnosis of Common Variable Immunodeficiency (CVID) as defined by PAGID (Pan-American Group for Immunodeficiency) and ESID (European Society for Immunodeficiencies) or X-linked Agammaglobulinemia (XLA) as defined by PAGID and ESID Women of childbearing potential must be using and agree to continue using medically approved contraception and must have a negative pregnancy test at screening Written informed consent Exclusion Criteria: 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 Hypoalbuminemia, protein-losing enteropathies, and any proteinuria (defined by total urine protein concentration > 0.2 g/L) Other significant medical conditions that could increase the risk to the patient Females who are pregnant, breast-feeding or planning a pregnancy during the course of the study A positive result at screening on any of the following viral markers: Human immunodeficiency virus (HIV), Hepatitis C virus (HCV) or Hepatitis B virus (HBV) Aspartate aminotransferase (ASAT) or Alanine aminotransferase (ALAT) concentration > 2.5 times Upper Normal Limit (UNL) at Completion Visit of study ZLB04_009CR (NCT00419341) Creatinine concentration > 1.5 times UNL at Completion Visit of study ZLB04_009CR (NCT00419341) Participation in a study with an investigational product other than IgPro20 within 3 months prior to enrollment Evidence of uncooperative attitude Any condition that is likely to interfere with evaluation of the study drug or satisfactory conduct of the trial 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
Organizational Affiliation
CSL Behring
Official's Role
Study Director
Facility Information:
Facility Name
Contact CSL Behring for facility details
City
Centennial
State/Province
Colorado
ZIP/Postal Code
80112
Country
United States
Facility Name
Contact CSL Behring for facility details
City
North Palm Beach
State/Province
Florida
ZIP/Postal Code
33408
Country
United States
Facility Name
Contact CSL Behring for facility details
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Contact CSL Behring for facility details
City
Dallas
State/Province
Texas
ZIP/Postal Code
75230
Country
United States

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=NCT00719680&registryName=ctgov
Description
Click here to request more information about this study

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Extension Study of Subcutaneous Immunoglobulin Human in Patients With Primary Immunodeficiency (PID)

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