Back to resultsEfficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency
Primary Purpose
Common Variable Immunodeficiency, Agammaglobulinemia
Status
Completed
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Vivaglobin
Sponsored by
About this trial
This is an interventional treatment trial for Common Variable Immunodeficiency focused on measuring Previously Untreated Patient (PUP), Primary Immunodeficiency (PID), CVID, XLA, Subcutaneous immunoglobulin (SCIG), IgG trough level, Quality of life, Common variable immunodeficiency (CVID), X-linked agammaglobulinemia (XLA)
Eligibility Criteria
Key Inclusion Criteria:
- Written informed consent, age-adapted
- Male or female aged 1 to 70 years
- Diagnosis of primary humoral immunodeficiency
- No prior immunoglobulin substitution therapy
- IgG level of <5 g/L at screening
- Women of childbearing potential must use medically approved contraception and must have a negative urine pregnancy test at screening
Key Exclusion Criteria:
- Evidence of serious infection between screening and first treatment
- Bleeding disorders that require medical treatments
- Any medical disorder causing secondary immune disorders, autoimmune neutropenia, or a clinically significant defect in cell mediated immunity
- Any condition likely to interfere with evaluation of the study drug or satisfactory conduct of the trial
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
- Contact CSL Behring for facility details
- Contact CSL Behring for facility details
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Vivaglobin
Arm Description
Vivaglobin: 16% (160 mg/mL) liquid formulation of human IgG for SC use. Loading dose: 100 mg/kg for 5 consecutive days; maintenance dose: 100 mg/kg 1 to 2 times a week for 24 weeks.
Outcomes
Primary Outcome Measures
Proportion of Patients Achieving Immunoglobulin G (IgG) Levels ≥ 5 g/L on Day 12
Secondary Outcome Measures
Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 19
Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 26
IgG Increase (Change From Baseline) on 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".
Total Serum IgG Trough Levels on Day 12
Total Serum IgG Trough Levels at Week 25
Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles on Day 12
Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles at Week 25
Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae On Day 12
Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae 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.
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.
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.
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.
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.
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.
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.
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).
Number of Patients With Clinically Relevant Changes in Vital Signs
Vital signs included heart rate, systolic blood pressure, diastolic blood pressure, and body temperature.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00520494
Brief Title
Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency
Official Title
A Multicenter Study on the Efficacy and Safety of Vivaglobin® in Previously Untreated Patients (PUPs) With Primary Immunodeficiency (PID)
Study Type
Interventional
2. Study Status
Record Verification Date
June 2013
Overall Recruitment Status
Completed
Study Start Date
March 2007 (undefined)
Primary Completion Date
October 2008 (Actual)
Study Completion Date
October 2008 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CSL Behring
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Common Variable Immunodeficiency, Agammaglobulinemia
Keywords
Previously Untreated Patient (PUP), Primary Immunodeficiency (PID), CVID, XLA, Subcutaneous immunoglobulin (SCIG), IgG trough level, Quality of life, Common variable immunodeficiency (CVID), X-linked agammaglobulinemia (XLA)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
18 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Vivaglobin
Arm Type
Experimental
Arm Description
Vivaglobin: 16% (160 mg/mL) liquid formulation of human IgG for SC use. Loading dose: 100 mg/kg for 5 consecutive days; maintenance dose: 100 mg/kg 1 to 2 times a week for 24 weeks.
Intervention Type
Drug
Intervention Name(s)
Vivaglobin
Intervention Description
Human normal immunoglobulin G (IgG) for subcutaneous (SC) use.
Primary Outcome Measure Information:
Title
Proportion of Patients Achieving Immunoglobulin G (IgG) Levels ≥ 5 g/L on Day 12
Time Frame
On Day 12
Secondary Outcome Measure Information:
Title
Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 19
Time Frame
On Day 19
Title
Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 26
Time Frame
On Day 26
Title
IgG Increase (Change From Baseline) on Day 12
Time Frame
Baseline to Day 12
Title
Overall Rate of Infections
Description
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
Title
Total Serum IgG Trough Levels on Day 12
Time Frame
On Day 12
Title
Total Serum IgG Trough Levels at Week 25
Time Frame
At Week 25
Title
Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles on Day 12
Time Frame
On Day 12
Title
Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles at Week 25
Time Frame
At Week 25
Title
Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae On Day 12
Time Frame
On Day 12
Title
Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae at Week 25
Time Frame
At Week 25
Title
Use of Antibiotics for Infection Prophylaxis and Treatment
Description
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
Title
Quality of Life as Measured by the Adapted Short Form-36 Health Survey (SF-36; Age ≥ 14 Years)
Description
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
Title
Quality of Life as Measured by the Child Health Questionnaire Parent Form-50 (CHQ-PF50; Age ≤ 13 Years)
Description
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
Title
Number of Patients With Adverse Events (AEs) by Severity and Relatedness
Description
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
Title
Rate of AEs by Severity and Relatedness
Description
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
Title
Number of Patients With Local Reactions by Severity and Relatedness
Description
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
Title
Rate of Local Reactions by Severity and Relatedness
Description
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
Title
Number of Patients With Clinically Relevant Changes in Routine Laboratory Parameters
Description
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
Title
Number of Patients With Clinically Relevant Changes in Vital Signs
Description
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)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Written informed consent, age-adapted
Male or female aged 1 to 70 years
Diagnosis of primary humoral immunodeficiency
No prior immunoglobulin substitution therapy
IgG level of <5 g/L at screening
Women of childbearing potential must use medically approved contraception and must have a negative urine pregnancy test at screening
Key Exclusion Criteria:
Evidence of serious infection between screening and first treatment
Bleeding disorders that require medical treatments
Any medical disorder causing secondary immune disorders, autoimmune neutropenia, or a clinically significant defect in cell mediated immunity
Any condition likely to interfere with evaluation of the study drug or satisfactory conduct of the trial
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Michael Borte, MD
Organizational Affiliation
Klinik für Kinder-und Jugendmedizin am Städtischen Klinikum St. Georg, Leipzig, Germany
Official's Role
Principal Investigator
Facility Information:
Facility Name
Contact CSL Behring for facility details
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 2B7
Country
Canada
Facility Name
Contact CSL Behring for facility details
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3H 1P3
Country
Canada
Facility Name
Contact CSL Behring for facility details
City
Leipzig
ZIP/Postal Code
04129
Country
Germany
Facility Name
Contact CSL Behring for facility details
City
Brescia
ZIP/Postal Code
25123
Country
Italy
Facility Name
Contact CSL Behring for facility details
City
Roma
ZIP/Postal Code
00186
Country
Italy
Facility Name
Contact CSL Behring for facility details
City
Madrid
ZIP/Postal Code
28007
Country
Spain
12. IPD Sharing Statement
Citations:
PubMed Identifier
21932110
Citation
Borte M, Quinti I, Soresina A, Fernandez-Cruz E, Ritchie B, Schmidt DS, McCusker C. Efficacy and safety of subcutaneous vivaglobin(R) replacement therapy in previously untreated patients with primary immunodeficiency: a prospective, multicenter study. J Clin Immunol. 2011 Dec;31(6):952-61. doi: 10.1007/s10875-011-9588-5. Epub 2011 Sep 20.
Results Reference
result
Links:
URL
http://www.cslbehring.com/clinical-trials/contact-us.htm?registryRefNum=NCT00520494®istryName=ctgov
Description
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Learn more about this trial
Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency
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