Primary Immune DeficiencyPhase 3NCT00719680

Extension Study of Subcutaneous Immunoglobulin Human in Patients With Primary Immunodeficiency (PID)

A Phase 3 study testing IgPro20 in people with Primary Immune Deficiency, run by CSL Behring at 4 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

The purpose of this study is to determine whether a long-term use of a new human immunoglobulin G with proline (IgPro) is safe and effective in the treatment of primary immunodeficiency. Read more about this study

Who is it for?: Ages 2 to 75 years
Conditions the study is for: Primary Immune Deficiency
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: 21 participants
Where: 4 sitesUnited States
Study dates: Jun 2008to Jun 2010
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 75 years
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Subjects with primary humoral immunodeficiency who have participated in the study ZLB04_009CR (NCT00419341), namely with a diagnosis of Common Variable Immunodeficiency (CVID) as defined by PAGID (Pan-American Group for Immunodeficiency) and ESID (European Society for Immunodeficiencies) or X-linked Agammaglobulinemia (XLA) as defined by PAGID and ESID
Women of childbearing potential must be using and agree to continue using medically approved contraception and must have a negative pregnancy test at screening
Written informed consent

Who cannot join

Ongoing serious bacterial infection at the time of screening
Malignancies of lymphoid cells such as lymphocytic leukemia, Non-Hodgkin's lymphoma, and immunodeficiency with thymoma
Hypoalbuminemia, protein-losing enteropathies, and any proteinuria (defined by total urine protein concentration > 0.2 g/L)
Other significant medical conditions that could increase the risk to the patient
Females who are pregnant, breast-feeding or planning a pregnancy during the course of the study
A positive result at screening on any of the following viral markers: Human immunodeficiency virus (HIV), Hepatitis C virus (HCV) or Hepatitis B virus (HBV)

Show all 12Show fewer

Aspartate aminotransferase (ASAT) or Alanine aminotransferase (ALAT) concentration > 2.5 times Upper Normal Limit (UNL) at Completion Visit of study ZLB04_009CR (NCT00419341)
Creatinine concentration > 1.5 times UNL at Completion Visit of study ZLB04_009CR (NCT00419341)
Participation in a study with an investigational product other than IgPro20 within 3 months prior to enrollment
Evidence of uncooperative attitude
Any condition that is likely to interfere with evaluation of the study drug or satisfactory conduct of the trial
Subjects who are employees at the investigational site, relatives or spouse of the investigator

Trial locations and contacts

4 sites in 1 country.

United States4 sites

Contact CSL Behring for facility details
Centennial, Colorado, 80112
Contact CSL Behring for facility details
Dallas, Texas, 75230
Contact CSL Behring for facility details
Indianapolis, Indiana, 46202
Contact CSL Behring for facility details
North Palm Beach, Florida, 33408

Other trials in United States

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 (PID)

The purpose of this study is to determine whether a long-term use of a new human immunoglobulin G with proline (IgPro) is safe and effective in the treatment of primary immunodeficiency.

Conditions: Primary Immune Deficiency
Being tested: IgPro20
Keywords: CVID, XLA
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

Study groups in NCT00719680
Study group	Role	What they receive
IgPro20	Experimental	The IgPro20 dose will be the same as in the previous pivotal study ZLB04_009CR (NCT00419341) infused subcutaneously weekly or twice a week (in the latter case, half of a weekly dose will be used) Receives: Biological: IgPro20

What is being measured

The primary outcome is the main question the study is designed to answer.

Annualized Rate of Serious Bacterial Infection (Intention-to-Treat Population)
The annualized rate was based on the total number of infections and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days.
Acute serious bacterial infections included pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess.
Measured: For the duration of the study, up to approximately 104 weeks
Annualized Rate of Serious Bacterial Infection (Per-Protocol Efficacy Population)
The annualized rate was based on the total number of infections and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days.
Acute serious bacterial infections included pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess.
Measured: For the duration of the study, up to approximately 104 weeks

Secondary outcomes (15)

Annualized Rate of Any Infection
The annualized rate was based on the total number of infections and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days.
Measured: For the duration of the study, up to approximately 104 weeks
Trough Levels of Total Immunoglobulin G (IgG) Serum Concentrations
Mean of individual median total IgG trough concentration.
Measured: Before infusion at Weeks 1, 24, 48, 72, and 96
Number of Days Out of Work / School / Kindergarten / Day Care or Inability to Perform Normal Activities Due to Infection
Measured: For the duration of the study, up to approximately 104 weeks
Annualized Rate of Days Out of Work / School / Kindergarten / Day Care or Inability to Perform Normal Activities Due to Infection
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 study days for all subjects in the specified analysis population and adjusted to 365 days.
Measured: For the duration of the study, up to approximately 104 weeks
Number of Days of Hospitalization Due to Infection
Measured: For the duration of the study, up to approximately 104 weeks
Annualized Rate of Hospitalization Due to Infection
The annualized rate was based on the total number of days of hospitalization due to infection and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days.
Measured: For the duration of the study, up to approximately 104 weeks
Use of Antibiotics for Infection Prophylaxis and Treatment
Annualized rate of days with antibiotics for infection prophylaxis and treatment.
Measured: For the duration of the study, up to approximately 104 weeks
Rate of All AEs by Relatedness and Severity
The rate of AEs was the number of AEs over the number of infusions administered.
At least possibly related AEs included possibly related AEs, probably related AEs, and related AEs.
Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.
Measured: For the duration of the study, up to approximately 104 weeks
Relatedness and Severity of All AEs (Percentage of Total AEs)
At least possibly related AEs included possibly related AEs, probably related AEs, and related AEs.
Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.
Measured: For the duration of the study, up to approximately 104 weeks
Number of Subjects With Any Temporally Associated Adverse Event (AE) Within 24 or 72 Hours After an Infusion
AEs were considered temporally associated if they occurred between the start of infusion and within 24 or 72 hours after the end of infusion.
Measured: Within 24 or 72 hours after each infusion
Rate of Temporally Associated AEs Within 24 or 72 Hours of an Infusion
The rate of AEs was the number of AEs over the number of infusions administered.
AEs were considered temporally associated if they occurred between the start of infusion and within 24 or 72 hours after the end of infusion.
Measured: Within 24 or 72 hours after each infusion
Number of Subjects Reporting Mild, Moderate, or Severe Local AEs
In addition to the standard MedDRA System Organ Class (SOC) AE assignments, the category of 'local reactions' was defined to provide the possibility for a combined analysis of local reactions and included AEs of infusion site oedema, infusion site reaction, injection site pain, injection site rash, and injection site reaction.
Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.
Measured: For the duration of the study, up to approximately 104 weeks
Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Vital Signs
Vital signs included blood pressure (systolic and diastolic), heart rate, and body temperature.
Measured: At weeks 1, 12, 24, 36, 48, 60, 72, 84, and 96
Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Routine Laboratory Parameters
Routine laboratory parameters included hematology, blood chemistry, and urinalysis parameters.
Measured: At Week 1, and study completion (approximately 104 weeks)
Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Viral Safety Markers
Viral safety markers included human immunodeficiency virus (HIV)-1, HIV-2, hepatitis A virus (HAV), HBV, HCV, and parvovirus B19.
Measured: At Week 1, and study completion (approximately 104 weeks)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jun 2008Jun 2010

First posted22 Jul 2008 (estimated)
Study startJun 2008
Primary completionJun 2010
Study completionJun 2010

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 Immune DeficiencyStudies 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: 21 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00719680
Sponsor's protocol ID: IgPro20_3001
Other IDs: 1473
Status last verified: Dec 2012
First submitted: 21 Jul 2008
Last updated: 2 Apr 2014
Results posted: Yes, on ClinicalTrials.gov

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

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Leukapheresis Procedures to Obtain Plasma and Lymphocytes for Research Studies on Antiretroviral Naive HIV-1 Research Participants
HIV
RecruitingObservational study
Natural History, Management, and Genetics of the Hyperimmunoglobulin E Recurrent Infection Syndrome (HIES)
Infections, Pneumonia
RecruitingObservational study
Genetic Basis of Immunodeficiency
Severe Combined Immunodeficiency
RecruitingObservational study
Detection and Characterization of Infections and Infection Susceptibility
Immune Deficiencies

All Primary Immune Deficiency trials

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 (PID)
ClinicalTrials.gov ID: NCT00719680
Acronym / sponsor ID: IgPro20_3001
Conditions: Primary Immune Deficiency
Keywords: CVID, XLA
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 21 participants (actual)
Lead sponsor: CSL BehringIndustry (pharma, biotech, devices)
Study start: Jun 2008
Primary completion: Jun 2010 (actual)
Study completion: Jun 2010 (actual)
First posted: 22 Jul 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.

The purpose of this study is to determine whether a long-term use of a new human immunoglobulin G with proline (IgPro) is safe and effective in the treatment of primary immunodeficiency.

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: IgPro20The IgPro20 dose will be the same as in the previous pivotal study ZLB04_009CR (NCT00419341) infused subcutaneously weekly or twice a week (in the latter case, half of a weekly dose will be used)

Who may qualify

Ages: 2 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (3 listed)

Subjects with primary humoral immunodeficiency who have participated in the study ZLB04_009CR (NCT00419341), namely with a diagnosis of Common Variable Immunodeficiency (CVID) as defined by PAGID (Pan-American Group for Immunodeficiency) and ESID (European Society for Immunodeficiencies) or X-linked Agammaglobulinemia (XLA) as defined by PAGID and ESID
Women of childbearing potential must be using and agree to continue using medically approved contraception and must have a negative pregnancy test at screening
Written informed consent

Key exclusion criteria (12 listed)

Ongoing serious bacterial infection at the time of screening
Malignancies of lymphoid cells such as lymphocytic leukemia, Non-Hodgkin's lymphoma, and immunodeficiency with thymoma
Hypoalbuminemia, protein-losing enteropathies, and any proteinuria (defined by total urine protein concentration > 0.2 g/L)
Other significant medical conditions that could increase the risk to the patient
Females who are pregnant, breast-feeding or planning a pregnancy during the course of the study

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	Biological	IgPro20

Primary outcome

Annualized Rate of Serious Bacterial Infection (Intention-to-Treat Population)
Time frame: For the duration of the study, up to approximately 104 weeks
The annualized rate was based on the total number of infections and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days. Acute serious bacterial infections included pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess.
Annualized Rate of Serious Bacterial Infection (Per-Protocol Efficacy Population)
Time frame: For the duration of the study, up to approximately 104 weeks
The annualized rate was based on the total number of infections and the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days. Acute serious bacterial infections included pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess.

15 secondary outcomes

Annualized Rate of Any Infection
Time frame: For the duration of the study, up to approximately 104 weeks
Trough Levels of Total Immunoglobulin G (IgG) Serum Concentrations
Time frame: Before infusion at Weeks 1, 24, 48, 72, and 96
Number of Days Out of Work / School / Kindergarten / Day Care or Inability to Perform Normal Activities Due to Infection
Time frame: For the duration of the study, up to approximately 104 weeks
Annualized Rate of Days Out of Work / School / Kindergarten / Day Care or Inability to Perform Normal Activities Due to Infection
Time frame: For the duration of the study, up to approximately 104 weeks
Number of Days of Hospitalization Due to Infection
Time frame: For the duration of the study, up to approximately 104 weeks
Annualized Rate of Hospitalization Due to Infection
Time frame: For the duration of the study, up to approximately 104 weeks
Use of Antibiotics for Infection Prophylaxis and Treatment
Time frame: For the duration of the study, up to approximately 104 weeks
Rate of All AEs by Relatedness and Severity
Time frame: For the duration of the study, up to approximately 104 weeks
Relatedness and Severity of All AEs (Percentage of Total AEs)
Time frame: For the duration of the study, up to approximately 104 weeks
Number of Subjects With Any Temporally Associated Adverse Event (AE) Within 24 or 72 Hours After an Infusion
Time frame: Within 24 or 72 hours after each infusion
Rate of Temporally Associated AEs Within 24 or 72 Hours of an Infusion
Time frame: Within 24 or 72 hours after each infusion
Number of Subjects Reporting Mild, Moderate, or Severe Local AEs
Time frame: For the duration of the study, up to approximately 104 weeks
Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Vital Signs
Time frame: At weeks 1, 12, 24, 36, 48, 60, 72, 84, and 96
Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Routine Laboratory Parameters
Time frame: At Week 1, and study completion (approximately 104 weeks)
Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Viral Safety Markers
Time frame: At Week 1, and study completion (approximately 104 weeks)

Results and safety

Results posted to the registry on 25 Jan 2013, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
IgPro20	21	16

Primary outcome: Annualized Rate of Serious Bacterial Infection (Intention-to-Treat Population)

Time frame: For the duration of the study, up to approximately 104 weeks. Number. Unit: infections per subject year

Measure	IgPro20 n = 21
Value	0.06

Population analysed: The Intention-to-Treat (ITT) population comprised all subjects treated with study medication during any study period.

Primary outcome: Annualized Rate of Serious Bacterial Infection (Per-Protocol Efficacy Population)

Time frame: For the duration of the study, up to approximately 104 weeks. Number. Unit: infections per subject year

Measure	IgPro20 n = 18
Value	0.03

Population analysed: The Per-Protocol Efficacy population comprised all subjects who completed at least 48 weeks of the efficacy period that started with the first IgPro20 dose in this study.

15 secondary outcomes are reported in the full results.

Adverse events

Time frame: From the time of informed consent until study completion, up to approximately 121 weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
IgPro20	4 of 21 (19%)	21 of 21 (100%)	Not reported

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 Immune Deficiency

RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Leukapheresis Procedures to Obtain Plasma and Lymphocytes for Research Studies on Antiretroviral Naive HIV-1 Research Participants
HIV
RecruitingObservational study
Natural History, Management, and Genetics of the Hyperimmunoglobulin E Recurrent Infection Syndrome (HIES)
Infections, Pneumonia
RecruitingObservational study
Genetic Basis of Immunodeficiency
Severe Combined Immunodeficiency
RecruitingObservational study
Detection and Characterization of Infections and Infection Susceptibility
Immune Deficiencies

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 study director

CSL Behring

4 sites in 1 country: 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 Immune Deficiency, run by CSL Behring at 4 sites in United States.

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 3 inclusion and 12 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 4 sites in United States. 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?

Annualized Rate of Serious Bacterial Infection (Intention-to-Treat Population) (time frame: For the duration of the study, up to approximately 104 weeks). The study lists 2 primary outcomes in all. 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 NCT00719680, 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.