Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (EU Extension Study) - Full Text View

Purpose

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.

Condition	Intervention	Phase
Primary Immunodeficiency (PID)	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 (IgPro20 EU Extension Study)

Resource links provided by NLM:

Genetics Home Reference related topics: complement factor I deficiency

Drug Information available for: Proline

U.S. FDA Resources

Further study details as provided by CSL Behring:

Primary Outcome Measures:

Total Serum IgG Trough Levels [ Time Frame: Up to 42 months ] [ Designated as safety issue: No ]
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) [ Time Frame: Up to 42 months ] [ Designated as safety issue: No ]
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 [ Time Frame: Up to 42 months ] [ Designated as safety issue: No ]
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 [ Time Frame: Up to 42 months ] [ Designated as safety issue: No ]
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 [ Time Frame: Up to 42 months ] [ 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 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 [ Time Frame: Up to 42 months ] [ Designated as safety issue: No ]
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 [ Time Frame: Up to 42 months ] [ Designated as safety issue: No ]
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 [ Time Frame: Up to 42 months ] [ Designated as safety issue: No ]
Total number of days of hospitalization due to infections for the specified analysis population
Use of Antibiotics for Infection Prophylaxis and Treatment [ Time Frame: Up to 42 months ] [ Designated as safety issue: No ]
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) [ Time Frame: At baseline and at the last available post-baseline observation for each subject (up to 42 months) ] [ Designated as safety issue: No ]
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. [ Time Frame: At baseline (data either from Infusion 40 or the completion visit of study ZLB06_001CR), and at completion (up to 42 months). ] [ Designated as safety issue: Yes ]
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. [ Time Frame: At baseline (data either from Infusion 40 or the completion visit of study ZLB06_001CR), and at completion (up to 42 months). ] [ Designated as safety issue: Yes ]
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 [ Time Frame: Up to 42 months ] [ Designated as safety issue: Yes ]
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.

Enrollment:	40
Study Start Date:	August 2008
Study Completion Date:	December 2011
Primary Completion Date:	December 2011 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: IgPro20 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).	Biological: IgPro20 Other Names: IgG with Proline (IgPro) Hizentra

Eligibility

Ages Eligible for Study:	2 Years to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00751621

Locations

France
Study Site
Paris, France, 75743
Germany
Study Site
Berlin, Germany, 13353
Study Site
Freiburg, Germany, 79095
Study Site
Leipzig, Germany, 04129
Study Site
Mainz, Germany, 55131
Poland
Study Site
Warsaw, Poland
Romania
Study Site
Cluj-Napoca, Romania, 400162
Study Site
Timisoara, Romania, 300011
Spain
Study Site
Barcelona, Spain, 08036
Study Site
Sevilla, Spain, 41013
Sweden
Study Site
Göteborg, Sweden, 41685
Switzerland
Study Site
Bern, Switzerland, 3010
United Kingdom
Study Site
London, United Kingdom, EC1A7BE

Sponsors and Collaborators

CSL Behring

Investigators

Study Director:

Program Director, Clinical R&D

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:	NCT00751621 History of Changes
Other Study ID Numbers:	ZLB07_002CR, 1472, 2008-000830-30
Study First Received:	September 11, 2008
Results First Received:	December 19, 2012
Last Updated:	December 19, 2012
Health Authority:	France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) Germany: Paul-Ehrlich-Institut Romania: National Medicines Agency Spain: Spanish Agency of Medicines Sweden: Medical Products Agency Switzerland: Swissmedic United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by CSL Behring:

Immune globulin subcutaneous
SCIG
primary immunodeficiency
PID

Additional relevant MeSH terms:

Immunologic Deficiency Syndromes
Immune System Diseases
Antibodies
Immunoglobulins

Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 19, 2013