Primary Immunodeficiency (PID)Phase 3NCT00751621
Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (EU Extension Study)
A Phase 3 study testing IgPro20 in people with Primary Immunodeficiency (PID), run by CSL Behring at 13 sites in 8 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 to 65 years
- Conditions the study is for
- Primary Immunodeficiency (PID)
- What is being tested?
- IgPro20
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 13 sitesin 8 countries
- Study dates
- Aug 2008to Dec 2011
- Phase
- Phase 3
- 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 2 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
13 sites in 8 countries.
13 sites in 8 countries
Germany4 sites
Study Site
Berlin, 13353
Study Site
Freiburg im Breisgau, 79095
Study Site
Leipzig, 04129
Study Site
Mainz, 55131
About this study
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)
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.
- Conditions
- Primary Immunodeficiency (PID)
- Being tested
- IgPro20
- Keywords
- Immune globulin subcutaneous, SCIG, primary immunodeficiency, PID
- Medical subject headings
- Primary Immunodeficiency Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IgPro20Biological
Also called IgG with Proline (IgPro), Hizentra
| Study group | Role | What they receive |
|---|---|---|
| IgPro20 | Experimental | 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). Receives: Biological: IgPro20 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 42 months
Secondary outcomes (12)
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.
Measured: 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.
Measured: Up to 42 months
Number of Infection Episodes
Total number of infections for the specified analysis population
Measured: 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.
Measured: Up to 42 months
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
Measured: 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.
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Up to 42 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2008 (estimated)
- Study startAug 2008
- Primary completionDec 2011
- Study completionDec 2011
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 Primary Immunodeficiency (PID)Studies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Program Director, Clinical R&D, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00751621
- Sponsor's protocol ID
- ZLB07_002CR
- Other IDs
- 1472, 2008-000830-30, 2008
- Status last verified
- Dec 2012
- First submitted
- 11 Sep 2008
- Last updated
- 2 Apr 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Jolles S, Borte M, Nelson RP Jr, Rojavin M, Bexon M, Lawo JP, Wasserman RL. Long-term efficacy, safety, and tolerability of Hizentra(R) for treatment of primary immunodeficiency disease. Clin Immunol. 2014 Feb;150(2):161-9. doi: 10.1016/j.clim.2013.10.008. Epub 2013 Oct 26.
PubMed 24412910
Study at a glance
From the registry record, last updated by the sponsor on 2 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT00751621
- Acronym / sponsor ID
- ZLB07_002CR
- Conditions
- Primary Immunodeficiency (PID)
- Keywords
- Immune globulin subcutaneous, SCIG, primary immunodeficiency, PID
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Aug 2008
- Primary completion
- Dec 2011 (actual)
- Study completion
- Dec 2011 (actual)
- First posted
- 12 Sep 2008 (estimated)
- Results
- Posted 25 Jan 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IgPro20 | Experimental | Biological: IgPro20Subcutaneous (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). |
Who may qualify
- Ages
- 2 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (7 listed)
- 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
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 |
|---|---|---|
| IgPro20Also: IgG with Proline (IgPro), Hizentra | Biological | IgPro20 |
Primary outcome
Total Serum IgG Trough Levels
Time frame: Up to 42 months
The IgG trough values per subject were aggregated to a median value, and then median values across subjects were summarized using descriptive statistics.
12 secondary outcomes
Annualized Rate of Clinically Documented Serious Bacterial Infections (SBIs)
Time frame: Up to 42 months
Annualized Rate of Infection Episodes
Time frame: Up to 42 months
Number of Infection Episodes
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
Time frame: Up to 42 months
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
Annualized Rate of Hospitalization Due to Infections
Time frame: Up to 42 months
Number of Days of Hospitalization Due to Infections
Time frame: Up to 42 months
Use of Antibiotics for Infection Prophylaxis and Treatment
Time frame: Up to 42 months
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)
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).
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).
Rate, Severity and Relatedness of Any Adverse Events (AEs) Per Infusion
Time frame: Up to 42 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Apr 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 24412910Jolles S, Borte M, Nelson RP Jr, Rojavin M, Bexon M, Lawo JP, Wasserman RL. Long-term efficacy, safety, and tolerability of Hizentra(R) for treatment of primary immunodeficiency disease. Clin Immunol. 2014 Feb;150(2):161-9. doi: 10.1016/j.clim.2013.10.008. Epub 2013 Oct 26.
Related trials for Primary Immunodeficiency (PID)
RecruitingObservational study
Studies of Disorders With Increased Susceptibility to Fungal Infections
Fungal Infections, Primary Immune Deficiencies
RecruitingObservational study
COVID-19 in PID Survey
Primary Immune Deficiency, COVID
Not yet recruitingPhase 4
A Study of TAK-664 in People With Primary Immunodeficiency Diseases Who Have Not Yet Been Treated With Immunoglobulins
Primary Immunodeficiency Diseases (PID)
RecruitingObservational study
A Database Survey to Evaluate the Safety of Immune Globulin Subcutaneous (Human), 20% Solution in Participants With Primary Immunodeficiency
Primary Immunodeficiency Diseases (PID)
Active, not recruitingPhase 2
A Study to Evaluate Tabelecleucel in Participants With Epstein Barr Virus (EBV) Associated Diseases
Epstein-Barr Virus (EBV)-Associated Diseases, EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (EBV+ PID LPD)
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.
Program Director, Clinical R&D study director
CSL Behring
13 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing IgPro20 in people with Primary Immunodeficiency (PID), run by CSL Behring at 13 sites in 8 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 2 inclusion and 7 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 2 Apr 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in Germany, Romania, Spain, France, Poland and 3 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 25 Jan 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?
Total Serum IgG Trough Levels (time frame: Up to 42 months). 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 NCT00751621, 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 2 Apr 2014.