Extension Study of Subcutaneous Immunoglobulin Human in Patients With Primary Immunodeficiency (PID) - Full Text View

Purpose

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.

Condition	Intervention	Phase
Primary Immune Deficiency	Biological: IgPro20	Phase 3

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency (PID)

Resource links provided by NLM:

Genetics Home Reference related topics: complement factor I deficiency

MedlinePlus related topics: Bacterial Infections

Drug Information available for: Proline

U.S. FDA Resources

Further study details as provided by CSL Behring:

Primary Outcome Measures:

Annualized Rate of Serious Bacterial Infection (Intention-to-Treat Population) [ Time Frame: For the duration of the study, up to approximately 104 weeks ] [ Designated as safety issue: No ]
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) [ Time Frame: For the duration of the study, up to approximately 104 weeks ] [ Designated as safety issue: No ]
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 [ Time Frame: For the duration of the study, up to approximately 104 weeks ] [ Designated as safety issue: No ]
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 [ Time Frame: Before infusion at Weeks 1, 24, 48, 72, and 96 ] [ Designated as safety issue: No ]
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 [ Time Frame: For the duration of the study, up to approximately 104 weeks ] [ Designated as safety issue: No ]
Annualized Rate 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 ] [ Designated as safety issue: No ]
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 [ Time Frame: For the duration of the study, up to approximately 104 weeks ] [ Designated as safety issue: No ]
Annualized Rate of Hospitalization Due to Infection [ Time Frame: For the duration of the study, up to approximately 104 weeks ] [ Designated as safety issue: No ]
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 [ Time Frame: For the duration of the study, up to approximately 104 weeks ] [ Designated as safety issue: No ]
Annualized rate of days with antibiotics for infection prophylaxis and treatment.
Rate of All AEs by Relatedness and Severity [ Time Frame: For the duration of the study, up to approximately 104 weeks ] [ Designated as safety issue: Yes ]
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) [ Time Frame: For the duration of the study, up to approximately 104 weeks ] [ Designated as safety issue: Yes ]
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 [ Time Frame: Within 24 or 72 hours after each infusion ] [ Designated as safety issue: Yes ]
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 [ Time Frame: Within 24 or 72 hours after each infusion ] [ Designated as safety issue: Yes ]
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 [ Time Frame: For the duration of the study, up to approximately 104 weeks ] [ Designated as safety issue: Yes ]
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 [ Time Frame: At weeks 1, 12, 24, 36, 48, 60, 72, 84, and 96 ] [ Designated as safety issue: Yes ]
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 [ Time Frame: At Week 1, and study completion (approximately 104 weeks) ] [ Designated as safety issue: Yes ]
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 [ Time Frame: At Week 1, and study completion (approximately 104 weeks) ] [ Designated as safety issue: Yes ]
Viral safety markers included human immunodeficiency virus (HIV)-1, HIV-2, hepatitis A virus (HAV), HBV, HCV, and parvovirus B19.

Enrollment:	21
Study Start Date:	June 2008
Study Completion Date:	June 2010
Primary Completion Date:	June 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: IgPro20 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)	Biological: IgPro20 Other Name: IgG with Proline

Eligibility

Ages Eligible for Study:	2 Years to 75 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00719680

Locations

United States, Colorado
Contact CSL Behring for facility details
Centennial, Colorado, United States, 80112
United States, Florida
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North Palm Beach, Florida, United States, 33408
United States, Indiana
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Indianapolis, Indiana, United States, 46202
United States, Texas
Contact CSL Behring for facility details
Dallas, Texas, United States, 75230

Sponsors and Collaborators

CSL Behring

Investigators

Study Director:

Program Director

CSL Behring

More Information

Additional Information:

Click here to request more information about this study This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	CSL Behring
ClinicalTrials.gov Identifier:	NCT00719680 History of Changes
Other Study ID Numbers:	IgPro20_3001, 1473
Study First Received:	July 21, 2008
Results First Received:	November 27, 2012
Last Updated:	December 20, 2012
Health Authority:	United States: Food and Drug Administration

Keywords provided by CSL Behring:

CVID
XLA

Additional relevant MeSH terms:

Immunologic Deficiency Syndromes
Immune System Diseases

ClinicalTrials.gov processed this record on May 21, 2013