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

CSL Behring40 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Primary Immunodeficiency (PID)

Interventions

IgPro20BIOLOGICAL

Eligibility

Sex: ALLMin age: 2 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (13)

Study Site

Paris, France

Study Site

Berlin, Germany

Study Site

Freiburg im Breisgau, Germany

Study Site

Leipzig, Germany

Outcomes

Primary Outcomes

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.

Time frame: Up to 42 months

Secondary Outcomes

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.

Time frame: Up to 42 months

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.

Time frame: Up to 42 months

Number of Infection Episodes

Total number of infections for the specified analysis population

Time frame: Up to 42 months

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.

Time frame: Up to 42 months

Data from ClinicalTrials.gov

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Study Site

Mainz, Germany

Study Site

Warsaw, Poland

Study Site

Cluj-Napoca, Romania

Study Site

Timișoara, Romania

Study Site

Barcelona, Spain

Study Site

Seville, Spain

...and 3 more locations

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

Time frame: Up to 42 months

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.

Time frame: Up to 42 months

Number of Days of Hospitalization Due to Infections

Total number of days of hospitalization due to infections for the specified analysis population

Time frame: Up to 42 months

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.

Time frame: Up to 42 months

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.

Time frame: At baseline and at the last available post-baseline observation for each subject (up to 42 months)

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.

Time frame: At baseline (data either from Infusion 40 or the completion visit of study ZLB06_001CR), and at completion (up to 42 months).

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.

Time frame: At baseline (data either from Infusion 40 or the completion visit of study ZLB06_001CR), and at completion (up to 42 months).

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.

Time frame: Up to 42 months