Primary Immune Deficiency DisorderPhase 3NCT01458171

Follow-up Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study)

A Phase 3 study testing Immune globulin subcutaneous (Human) in people with Primary Immune Deficiency Disorder, run by CSL Behring at 9 sites in Japan.

Completed

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

Official record

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Trial summary

The objective of this study is to assess the long-term safety, tolerability, and efficacy of IgPro20 in subjects with primary immunodeficiency (PID) as a follow-up to the pivotal study ZLB06_002CR (NCT01199705). Read more about this study

Who is it for?: Ages Up to 75 years
Conditions the study is for: Primary Immune Deficiency Disorder
What is being tested?: Immune globulin subcutaneous (Human)
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 23 participants
Where: 9 sitesJapan
Study dates: Apr 2011to Apr 2012
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 Up to 75 years
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Subjects who have participated in study ZLB06_002CR and who have tolerated IgPro20 well.
Written informed consent by the subject/parent/legally acceptable representative. Written assent for an underage subject (≥7 years at the time of obtaining informed consent), as far as possible.

Who cannot join

Ongoing serious bacterial infections (SBIs) (pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, or visceral abscess) at the time of the first infusion.
Hypoalbuminemia, protein-losing enteropathies, and any proteinuria (known total urine protein concentration >0.2 g/L or urine protein ++ by dipstick).
Pregnancy or nursing mother.
Participation in a study with an investigational medicinal product (IMP) within 3 months prior to enrollment except for ZLB06_002CR.
Subjects who are planning to donate blood during the study.
Re-entry of subjects previously participating in the current follow-up study.
Known or suspected antibodies to the IMP, or to excipients of the IMP.

Trial locations and contacts

9 sites in 1 country.

City, postcode or hospital

9 sites in 1 country

Japan9 sites

Study site
Bunkyō City, Tokyo Metropolitan, 113-8519
Study site
Chiba, Chiba Pref., 260-8677
Study site
Fukuoka, Fukuoka, 812-8582
Study site
Gifu, Gifu Pref., 502-8558
Study site
Koshigaya, Saitama Pref., 343-8555
Study site
Moriguchi, Osaka, 570-8507
Study site
Nagoya, Aichi Pref., 466-8560
Study site
Sapporo, Hokkaido, 060-8648
Study site
Tokorozawa, Saitama Pref., 359-8513

Other trials in Japan

About this study

Official title: A Multicenter Follow-up Study of Long-term Safety, Tolerability, and Efficacy of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency

Conditions: Primary Immune Deficiency Disorder
Being tested: Immune globulin subcutaneous (Human)
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.

Immune globulin subcutaneous (Human)Biological
Also called Hizentra
IgPro20 is a 20% (weight per volume [w/v]) liquid formulation of human immunoglobulin for subcutaneous (SC) use. Subjects will receive weekly infusions of IgPro20 for a total of 24 weeks at a dose based on the subject's IgPro20 dose in the pivotal study ZLB06_002CR (NCT01199705).

Study groups in NCT01458171
Study group	Role	What they receive
IgPro20	Experimental	Not described Receives: Biological: Immune globulin subcutaneous (Human)

What is being measured

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

Median of the Individual Subject's Rate of Adverse Events (AEs) Per Infusion
The rate was calculated by counting all newly developed or worsened AEs within a subject and dividing by the total number of IgPro20 infusions administered to this subject. Subsequently, the median of these individual AE rates per infusion was calculated. AE rates were classified (i) by severity (mild, moderate, severe) and (ii) by causal relationship to study medication (not related or unlikely related; at least possibly related [i.e., possibly related, probably related, or related]).
Measured: 24 weeks

Secondary outcomes (9)

Overall Rate of AEs Per Infusion
The rate was calculated by counting all newly developed or worsened AEs during the treatment period in all subjects and dividing the total number of AEs by the total number of IgPro20 infusions administered. In addition, individual AEs were classified (i) by severity (mild, moderate, severe) and (ii) by causal relationship to study medication (not related or unlikely related; at least possibly related [i.e., possibly related, probably related, or related]). The AE rates per infusion by severity and causal relationship to study medication were calculated by dividing the number of AEs in each category by the total number of IgPro20 infusions.
Measured: 24 weeks
Number of Subjects With Newly Developing or Worsening AEs
Number of subjects with AEs, overall and classified (i) by severity (mild, moderate, severe) and (ii) by causal relationship to study medication (not related or unlikely related; at least possibly related [i.e., possibly related, probably related, or related]).
Measured: 24 weeks
Percentage of Infusions With Subject-assessed Tolerability of at Least 'Good'
Subjects assessed their overall perception of local tolerability at the infusion site throughout the study in the subject diary within a time window of 24 h to 72 h after the end of the latest infusion by assessing it as "very good", "good", "fair", or "poor". The reported percentage represents the percentage of subjects with local tolerability assessments of "very good" or "good" at any given study infusion.
Measured: 24 to 72 hours after infusion
IgG Trough Level
Serum IgG trough levels at the completion visit compared to the baseline visit of the follow-up study. IgG trough levels at baseline, at the completion visit, and the change from baseline to the completion visit are shown
Measured: 24 weeks
Annualized Rate of Clinically Documented Serious Bacterial Infections (SBIs)
SBIs are defined as bacterial pneumonia, bacteremia and septicemia, osteomyelitis/septic arthritis, bacterial meningitis, or visceral abscess. The annualized rate was based on the total number of SBIs and the total number of subject study days for all subjects in the FAS and PPS and adjusted to 365 days.
Measured: 24 weeks
Number of Infection Episodes (Serious and Non-serious)
Measured: 24 weeks
Number of Days Out of Work/School/Kindergarten/Day Care or Unable to Perform Normal Daily Activities Due to Infections.
Median number of days out of work/school/kindergarten/day care or unable to perform normal daily activities due to infections.
Measured: 24 weeks
Number of Days of Hospitalization Due to Infections.
Median number of days of hospitalization due to infections.
Measured: 24 weeks
Duration of Use of Antibiotics for Infection Prophylaxis and Treatment
Median number of days of use of antibiotics for infection prophylaxis and/or treatment
Measured: 24 weeks

Timeline

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

Apr 2011Apr 2012

First posted24 Oct 2011 (estimated)
Study startApr 2011
Primary completionFeb 2012
Study completionApr 2012

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.
Other trials for Primary Immune Deficiency DisorderStudies 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: 23 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01458171
Sponsor's protocol ID: ZLB07_001CR
Status last verified: Feb 2013
First submitted: 12 Oct 2011
Last updated: 9 Apr 2013
Results posted: Yes, on ClinicalTrials.gov

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

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Study at a glance

From the registry record, last updated by the sponsor on 9 Apr 2013. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multicenter Follow-up Study of Long-term Safety, Tolerability, and Efficacy of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency
ClinicalTrials.gov ID: NCT01458171
Acronym / sponsor ID: ZLB07_001CR
Conditions: Primary Immune Deficiency Disorder
Keywords: Immune globulin subcutaneous, SCIG, Primary immunodeficiency, PID
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 23 participants (actual)
Lead sponsor: CSL BehringIndustry (pharma, biotech, devices)
Study start: Apr 2011
Primary completion: Feb 2012 (actual)
Study completion: Apr 2012 (actual)
First posted: 24 Oct 2011 (estimated)
Results: Posted 9 Apr 2013

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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: Immune globulin subcutaneous (Human)

Who may qualify

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

Key inclusion criteria (2 listed)

Subjects who have participated in study ZLB06_002CR and who have tolerated IgPro20 well.
Written informed consent by the subject/parent/legally acceptable representative. Written assent for an underage subject (≥7 years at the time of obtaining informed consent), as far as possible.

Key exclusion criteria (7 listed)

Ongoing serious bacterial infections (SBIs) (pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, or visceral abscess) at the time of the first infusion.
Hypoalbuminemia, protein-losing enteropathies, and any proteinuria (known total urine protein concentration >0.2 g/L or urine protein ++ by dipstick).
Pregnancy or nursing mother.
Participation in a study with an investigational medicinal product (IMP) within 3 months prior to enrollment except for ZLB06_002CR.
Subjects who are planning to donate blood during 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
Immune globulin subcutaneous (Human)Also: HizentraIgPro20 is a 20% (weight per volume [w/v]) liquid formulation of human immunoglobulin for subcutaneous (SC) use. Subjects will receive weekly infusions of IgPro20 for a total of 24 weeks at a dose based on the subject's IgPro20 dose in the pivotal study ZLB06_002CR (NCT01199705).	Biological	IgPro20

Primary outcome

Median of the Individual Subject's Rate of Adverse Events (AEs) Per Infusion
Time frame: 24 weeks
The rate was calculated by counting all newly developed or worsened AEs within a subject and dividing by the total number of IgPro20 infusions administered to this subject. Subsequently, the median of these individual AE rates per infusion was calculated. AE rates were classified (i) by severity (mild, moderate, severe) and (ii) by causal relationship to study medication (not related or unlikely related; at least possibly related [i.e., possibly related, probably related, or related]).

9 secondary outcomes

Overall Rate of AEs Per Infusion
Time frame: 24 weeks
Number of Subjects With Newly Developing or Worsening AEs
Time frame: 24 weeks
Percentage of Infusions With Subject-assessed Tolerability of at Least 'Good'
Time frame: 24 to 72 hours after infusion
IgG Trough Level
Time frame: 24 weeks
Annualized Rate of Clinically Documented Serious Bacterial Infections (SBIs)
Time frame: 24 weeks
Number of Infection Episodes (Serious and Non-serious)
Time frame: 24 weeks
Number of Days Out of Work/School/Kindergarten/Day Care or Unable to Perform Normal Daily Activities Due to Infections.
Time frame: 24 weeks
Number of Days of Hospitalization Due to Infections.
Time frame: 24 weeks
Duration of Use of Antibiotics for Infection Prophylaxis and Treatment
Time frame: 24 weeks

Results and safety

Results posted to the registry on 9 Apr 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	23	22

Primary outcome: Median of the Individual Subject's Rate of Adverse Events (AEs) Per Infusion

Time frame: 24 weeks. Median (Full Range). Unit: AEs per infusion

Measure	IgPro20 n = 23
All AEs	0.167 (0 to 1.71)
Mild AEs	0.125 (0 to 1.71)
Moderate AEs	0 (0 to 0.04)
Severe AEs	0 (0 to 1.00)
Not related or unlikely related AEs	0.125 (0 to 0.75)
At least possibly related AEs	0 (0 to 1.00)

Population analysed: The All Treated (AT) set comprised all subjects receiving at least 1 IgPro20 infusion.

9 secondary outcomes are reported in the full results.

Adverse events

Time frame: For the duration of the study, that is, 24 weeks.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
IgPro20	1 of 23 (4.3%)	20 of 23 (87%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2013.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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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.

Midori Kobayashi study director

CSL Behring K.K.

9 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 Immune globulin subcutaneous (Human) in people with Primary Immune Deficiency Disorder, run by CSL Behring at 9 sites in Japan.

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 9 Apr 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in Japan. 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 9 Apr 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?

Median of the Individual Subject's Rate of Adverse Events (AEs) Per Infusion (time frame: 24 weeks). 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 NCT01458171.

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 9 Apr 2013.