Common Variable ImmunodeficiencyPhase 4NCT00520494
Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency
A Phase 4 study testing Vivaglobin in people with Common Variable Immunodeficiency and Agammaglobulinemia, run by CSL Behring at 6 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 70 years
- Conditions the study is for
- Common Variable Immunodeficiency, Agammaglobulinemia
- What is being tested?
- Vivaglobin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 6 sitesin 4 countries
- Study dates
- Mar 2007to Oct 2008
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
6 sites in 4 countries.
6 sites in 4 countries
Canada2 sites
Contact CSL Behring for facility details
Edmonton, Alberta, T6G 2B7
Contact CSL Behring for facility details
Montreal, Quebec, H3H 1P3
Italy2 sites
Contact CSL Behring for facility details
Brescia, 25123
Contact CSL Behring for facility details
Roma, 00186
About this study
Official title: A Multicenter Study on the Efficacy and Safety of Vivaglobin® in Previously Untreated Patients (PUPs) With Primary Immunodeficiency (PID)
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.
- Being tested
- Vivaglobin
- 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)
- Medical subject headings
- Common Variable Immunodeficiency, Agammaglobulinemia, Primary Immunodeficiency Diseases, Bruton type agammaglobulinemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VivaglobinDrug
Human normal immunoglobulin G (IgG) for subcutaneous (SC) use.
| Study group | Role | What they receive |
|---|---|---|
| Vivaglobin | Experimental | 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. Receives: Drug: Vivaglobin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Patients Achieving Immunoglobulin G (IgG) Levels ≥ 5 g/L on Day 12
Measured: On Day 12
Secondary outcomes (19)
Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 19
Measured: On Day 19
Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 26
Measured: On Day 26
IgG Increase (Change From Baseline) on Day 12
Measured: 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".
Measured: For the duration of the study, up to approximately 25 weeks
Total Serum IgG Trough Levels on Day 12
Measured: On Day 12
Total Serum IgG Trough Levels at Week 25
Measured: At Week 25
Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles on Day 12
Measured: On Day 12
Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles at Week 25
Measured: At Week 25
Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae On Day 12
Measured: On Day 12
Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae at Week 25
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: 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.
Measured: At the screening visit, before and after infusions (Days 1 to 5), and at the completion visit (Week 25)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Aug 2007 (estimated)
- Study startMar 2007
- Primary completionOct 2008
- Study completionOct 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Common Variable ImmunodeficiencyStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Michael Borte, MD, Principal investigator, Klinik für Kinder-und Jugendmedizin am Städtischen Klinikum St. Georg, Leipzig, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00520494
- Sponsor's protocol ID
- ZLB06_005CR
- Other IDs
- 2006-006522-25, 1461
- Status last verified
- Jun 2013
- First submitted
- 23 Aug 2007
- Last updated
- 20 Jun 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 21932110
Study at a glance
From the registry record, last updated by the sponsor on 20 Jun 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Study on the Efficacy and Safety of Vivaglobin® in Previously Untreated Patients (PUPs) With Primary Immunodeficiency (PID)
- ClinicalTrials.gov ID
- NCT00520494
- Acronym / sponsor ID
- ZLB06_005CR
- Keywords
- Previously Untreated Patient (PUP), Primary Immunodeficiency (PID), CVID, XLA, Subcutaneous immunoglobulin (SCIG), IgG trough level, Quality of life, Common variable immunodeficiency (CVID)
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Mar 2007
- Primary completion
- Oct 2008 (actual)
- Study completion
- Oct 2008 (actual)
- First posted
- 24 Aug 2007 (estimated)
- Results
- Posted 3 May 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vivaglobin | Experimental | Drug: VivaglobinVivaglobin: 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. |
Who may qualify
- Ages
- 1 year to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VivaglobinHuman normal immunoglobulin G (IgG) for subcutaneous (SC) use. | Drug | Vivaglobin |
Primary outcome
Proportion of Patients Achieving Immunoglobulin G (IgG) Levels ≥ 5 g/L on Day 12
Time frame: On Day 12
19 secondary outcomes
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
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
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)
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)
Time frame: At study completion, approximately Week 25
Number of Patients With Adverse Events (AEs) by Severity and Relatedness
Time frame: For the duration of the study, up to approximately 25 weeks
Rate of AEs by Severity and Relatedness
Time frame: For the duration of the study, up to approximately 25 weeks
Number of Patients With Local Reactions by Severity and Relatedness
Time frame: For the duration of the study, up to approximately 25 weeks
Rate of Local Reactions by Severity and Relatedness
Time frame: For the duration of the study, up to approximately 25 weeks
Number of Patients With Clinically Relevant Changes in Routine Laboratory Parameters
Time frame: At Weeks 12 and 25
Number of Patients With Clinically Relevant Changes in Vital Signs
Time frame: At the screening visit, before and after infusions (Days 1 to 5), and at the completion visit (Week 25)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jun 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 21932110Borte 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael Borte, MD principal investigator
Klinik für Kinder-und Jugendmedizin am Städtischen Klinikum St. Georg, Leipzig, Germany
6 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Vivaglobin in people with Common Variable Immunodeficiency and Agammaglobulinemia, run by CSL Behring at 6 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Jun 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Canada, Italy, Germany, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 May 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of Patients Achieving Immunoglobulin G (IgG) Levels ≥ 5 g/L on Day 12 (time frame: On Day 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00520494, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jun 2013.