The objective of this study is to assess the efficacy and safety of Vivaglobin in previously untreated patients (PUPs) with primary immunodeficiency (PID) over a 25-week observation period. The purpose is to investigate whether PUPs will respond to subcutaneous immunoglobulin (SCIG) treatment with adequate trough levels without first receiving immunoglobulins by the intravenous route by demonstrating that 100 mg immunoglobulin G/kg body weight (IgG/kg bw) administered on 5 consecutive days (i.e. resulting in a total dose of 500 mg IgG/kg bw) results in an IgG increase to ≥ 5 g/L on Day 12 after initiation of SCIG therapy.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
18
Human normal immunoglobulin G (IgG) for subcutaneous (SC) use.
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Edmonton, Alberta, Canada
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Montreal, Quebec, Canada
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Leipzig, Germany
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Brescia, Italy
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Roma, Italy
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Madrid, Spain
Proportion of Patients Achieving Immunoglobulin G (IgG) Levels ≥ 5 g/L on Day 12
Time frame: On Day 12
Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 19
Time frame: On Day 19
Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 26
Time frame: On Day 26
IgG Increase (Change From Baseline) on Day 12
Time frame: Baseline to Day 12
Overall Rate of Infections
Annualized rate of any infection. The annualized rate was based on the total number of infections and the total number of patient study days for all patients in the specified analysis population and adjusted to 365 days. Infections were classified as all AEs with the system organ class "infections and infestations".
Time frame: For the duration of the study, up to approximately 25 weeks
Total Serum IgG Trough Levels on Day 12
Time frame: On Day 12
Total Serum IgG Trough Levels at Week 25
Time frame: At Week 25
Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles on Day 12
Time frame: On Day 12
Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles at Week 25
Time frame: At Week 25
Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae On Day 12
Time frame: On Day 12
Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae at Week 25
Time frame: At Week 25
Use of Antibiotics for Infection Prophylaxis and Treatment
Number of patients. Medications were classified as antibiotics according to the anatomic therapeutic chemical code.
Time frame: For the duration of the study, up to approximately 25 weeks
Quality of Life as Measured by the Adapted Short Form-36 Health Survey (SF-36; Age ≥ 14 Years)
The 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 study completion, approximately Week 25
Quality of Life as Measured by the Child Health Questionnaire Parent Form-50 (CHQ-PF50; Age ≤ 13 Years)
The CHQ-PF50 is a 50-item questionnaire that measures generic health concepts and is suitable for patients younger than 14 years of age. The questions are grouped into 15 domains: global health, physical functioning, role/social limitations - emotional/behavioral, role/social limitations - physical, bodily pain, behavior, global behavior, mental health, self esteem, general health perceptions, change in health, parental impact - emotional, parental impact - time, family activities, and family cohesion. Scores range from 0 to 100, with higher scores indicating a better health state.
Time frame: At study completion, approximately Week 25
Number of Patients With Adverse Events (AEs) by Severity and Relatedness
Mild AE: Did not interfere with activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities. Not related: Explained by factors not involving the drug, no temporal relationship; Possibly related: Occurred within a reasonable time of administration, could also be explained by concurrent disease or other drugs; Probably related: Compelling temporal relationship, could not be explained concurrent disease/other drugs; Related AE: Compelling temporal relationship, known/suspected response to the drug confirmed by improvement on stopping.
Time frame: For the duration of the study, up to approximately 25 weeks
Rate of AEs by Severity and Relatedness
The rate was the number of AEs over the number of infusions administered. Mild AE: Did not interfere with activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities. Not related: Explained by factors not involving the drug, no temporal relationship; Possibly related: Occurred within a reasonable time of administration, could also be explained by concurrent disease or other drugs; Probably related: Compelling temporal relationship, could not be explained concurrent disease/other drugs; Related AE: Compelling temporal relationship, known/suspected response to the drug confirmed by improvement on stopping.
Time frame: For the duration of the study, up to approximately 25 weeks
Number of Patients With Local Reactions by Severity and Relatedness
Local reactions included: infusion site erythema, infusion site pain, infusion site pruritus, infusion site rash, infusion site reaction, infusion site swelling, injection site bruising, injection site erythema, injection site irritation, injection site pruritus, injection site swelling, edema peripheral, tenderness, erythema, pruritus, and skin swelling. Mild AE: Did not interfere with activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities. Not related: Explained by factors not involving the drug, no temporal relationship; Possibly related: Occurred within a reasonable time of administration, could also be explained by concurrent disease or other drugs; Probably related: Compelling temporal relationship, could not be explained concurrent disease/other drugs; Related AE: Compelling temporal relationship, known/suspected response to the drug confirmed by improvement on stopping.
Time frame: For the duration of the study, up to approximately 25 weeks
Rate of Local Reactions by Severity and Relatedness
The rate was the number of local reactions over the number of infusions administered. Local reactions included: * infusion site: erythema, pain, pruritus, rash, reaction, swelling; * injection site: bruising, erythema, irritation, pruritus, swelling; * edema peripheral; * tenderness; * erythema; * pruritus; and * skin swelling. Mild AE: Did not interfere with activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities. Not related: Explained by factors not involving the drug, no temporal relationship; Possibly related: Occurred within a reasonable time of administration, could also be explained by concurrent disease or other drugs; Probably related: Compelling temporal relationship, could not be explained concurrent disease/other drugs; Related AE: Compelling temporal relationship, known/suspected response to the drug confirmed by improvement on stopping.
Time frame: For the duration of the study, up to approximately 25 weeks
Number of Patients With Clinically Relevant Changes in Routine Laboratory Parameters
Laboratory parameters included hematology, serum chemistry, and urinalysis parameters, and were assessed at screening, Week 12 (hematology and serum chemistry) and at the completion visit (approximately Week 25).
Time frame: At Weeks 12 and 25
Number of Patients With Clinically Relevant Changes in Vital Signs
Vital signs included heart rate, systolic blood pressure, diastolic blood pressure, and body temperature.
Time frame: At the screening visit, before and after infusions (Days 1 to 5), and at the completion visit (Week 25)
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