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Primary Immune DeficiencyPhase 3NCT01199705

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

A Phase 3 study testing Immune Globulin Subcutaneous (Human) (SCIG) in people with Primary Immune Deficiency, run by CSL Behring at 10 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 efficacy, safety, tolerability, and pharmacokinetics of a subcutaneous immune globulin (SCIG; IgPro20) in subjects with primary immunodeficiency (PID). Read more about this study

Who is it for?
Ages Up to 75 years
Conditions the study is for
Primary Immune Deficiency
What is being tested?
Immune Globulin Subcutaneous (Human) (SCIG)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
25 participants
Where
10 sitesJapan
Study dates
Sep 2010to Nov 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 Up to 75 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Diagnosis of PID with hypo- or agammaglobulinemia requiring IgG replacement therapy
  • Intravenous IgG (IVIG) therapy at regular 3- or 4-week intervals at a stable dose for at least 3 doses prior to signing of informed consent
  • Written informed consent

Who cannot join

  • Newly diagnosed PID, i.e., subjects who have not previously received immunoglobulin replacement therapy
  • Ongoing serious bacterial infections (SBIs: pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, or visceral abscess) at the time of screening
  • Ongoing or history of concomitant malignancies of lymphoid cells such as lymphocytic leukemia, non-Hodgkin's lymphoma, and immunodeficiency with thymoma
  • Allergic or other severe reactions to immunoglobulins or other blood products recorded in the past 3 months or at the time of screening
  • Pregnancy or nursing mother
  • A positive result at screening on any of the following viral markers: human immunodeficiency virus-1 (HIV-1), HIV-2, hepatitis C virus, or hepatitis B virus
  • Participation in a study with other investigational product during this study and within 3 months prior to screening
  • Subjects who donated blood (200 mL within one month or 400 mL within 3 months prior to screening), or planning to donate blood during the study

Trial locations and contacts

10 sites in 1 country.

10 sites in 1 country

Japan10 sites

  • Study site

    Chiba, Chiba Pref., 260-8677

  • Study site

    Fukuoka City, Osaka, 812-8582

  • Study site

    Gifu, Gifu Pref., 501-1194

  • Study site

    Koshigaya, Saitama Pref., 343-8555

  • Study site

    Moriguchi, Osaka, 570-8507

  • Study site

    Nagoya, Aichi Pref., 466-8560

  • Study site

    Osaka, Osaka, 534-0021

  • Study site

    Sapporo, Hokkaido, 060-8648

  • Study site

    Sendai, Miyagi Pref., 980-8574

  • Study site

    Tokorozawa, Saitama Pref., 359-8513

Other trials in Japan

About this study

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

The objective of this study is to assess the efficacy, safety, tolerability, and pharmacokinetics of a subcutaneous immune globulin (SCIG; IgPro20) in subjects with primary immunodeficiency (PID). In addition, the study will assess the health-related quality of life and pharmacoeconomic aspects related to treatment with IgPro20.

Being tested
Immune Globulin Subcutaneous (Human) (SCIG)
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) (SCIG)Biological

    Also called Hizentra

    IgPro20 is a 20% (weight per volume [w/v]) liquid formulation of human SCIG. Subjects will receive weekly infusions of IgPro20 at a weekly dosage calculated based on previous IVIG treatment.

Study groups in NCT01199705
Study groupRoleWhat they receive
IgPro20ExperimentalNot described
Receives: Biological: Immune Globulin Subcutaneous (Human) (SCIG)

What is being measured

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

  1. IgG Trough Level

    Geometric means of trough levels measured before 3 intravenous immunoglobulin (IVIG) infusions was compared with those of trough levels measured at steady-state for 3 subcutaneous immunoglobulin (SCIG) infusions (weeks 16, 20 and 24). The ratio of these geometric means was the primary outcome measure.

    Measured: During IVIG period (IV 1, IV 2, IV 3) and during SCIG period at weeks 16, 20, and 24

Secondary outcomes (8)

  1. Number of Infection Episodes (Serious and Non-serious) by Study Period

    Number of infection episodes (serious and non-serious) presented by study period:

    • IVIG treatment: Study subjects were treated with their IVIG therapy with 3- or 4-weekly schedules for 3 dosing cycles (9 to 12 weeks; before being switched to SCIG treatment with IgPro20).
    • SCIG treatment (wash-in/wash-out; weeks 1 to 12): IgPro20 was administered subcutaneously with the first subcutaneous (SC) IgPro20 infusion starting 1 week after the last IVIG dose. Subjects were treated with weekly SC IgPro20 infusions for a 12-week wash-in/wash-out period. The IgPro20 dose was to be equal to the weekly equivalent dose of the previous IVIG therapy.
    • SCIG treatment (efficacy; weeks 13 to 24): After the SCIG wash-in/wash-out treatment, subjects were treated with weekly SC IgPro20 infusions for a 12-week efficacy period. The IgPro20 dose was to be equal to the weekly equivalent dose of the previous IVIG therapy.

    Measured: Up to 36 weeks

  2. Rate of Infection Episodes (Serious and Non-serious) by Study Period, PPS Population

    The annualized rate of infection episodes (serious and non-serious) was based on the total number of infection episodes and the total number of subject study days for all subjects in the specified study periods (listed below) and analysis population and adjusted to 365 days.

    Study periods:

    • IVIG treatment (up to 12 weeks)
    • SCIG IgPro20 treatment (wash-in/wash-out period) (12 weeks)
    • SCIG IgPro20 treatment (efficacy) (12 weeks)

    Measured: Up to 36 weeks

  3. Rate of Infection Episodes (Serious and Non-serious) by Study Period, FAS Population

    The annualized rate of infection episodes (serious and non-serious) was based on the total number of infection episodes and the total number of subject study days for all subjects in the specified study periods (listed below) and analysis population and adjusted to 365 days.

    Study periods:

    • IVIG treatment (up to 12 weeks)
    • SCIG IgPro20 treatment (wash-in/wash-out period) (12 weeks)
    • SCIG IgPro20 treatment (efficacy) (12 weeks)

    Measured: Up to 36 weeks

  4. Number of Days Out of Work/School/Kindergarten/Day Care or Unable to Perform Normal Daily Activities Due to Infections by Study Period

    Median number of days out of work/school/kindergarten/day care or unable to perform normal daily activities due to infections, presented by study period: IVIG treatment (up to 12 weeks), SCIG IgPro20 treatment (wash-in/wash-out; 12 weeks), and SCIG IgPro20 treatment (efficacy; 12 weeks).

    Measured: Up to 36 weeks

  5. Number of Days of Hospitalization Due to Infections by Study Period

    Median number of days of hospitalization due to infections, presented by study period: IVIG treatment (up to 12 weeks), SCIG IgPro20 treatment (wash-in/wash-out; 12 weeks), and SCIG IgPro20 treatment (efficacy; 12 weeks).

    Measured: Up to 36 weeks

  6. Duration of Use of Antibiotics for Infection Prophylaxis and Treatment

    Median number of days of use of antibiotics for infection prophylaxis and/or treatment, presented by study period: IVIG treatment (up to 12 weeks), SCIG IgPro20 treatment (wash-in/wash-out; 12 weeks), and SCIG IgPro20 treatment (efficacy; 12 weeks).

    Measured: Up to 36 weeks

  7. Rate of All Adverse Events by Relatedness and Seriousness

    The rate of adverse events (AEs) was the number of treatment-emergent AEs over the number of infusions administered. At least possibly related AEs included possibly related AEs, probably related AEs, and related AEs.

    Measured: For the duration of the study, up to 36 weeks

  8. Rate of Mild, Moderate, or Severe Local Reactions

    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 discomfort, infusion site erythema, infusion site haemorrhage, infusion site induration, infusion site inflammation, infusion site pain, infusion site pruritus, infusion site swelling, injection site erythema, injection site extravasation, injection site induration, injection site irritation, injection site pain, injection site pruritus, injection site swelling, and puncture site reaction.

    Mild AE: Symptoms are easily tolerated and there is no interference with daily activities; Moderate AE: Discomfort enough to cause some interference with daily activities; Severe AE: Incapacitating with inability to work or do usual activity.

    Measured: For the duration of the study, up to 36 weeks

Timeline

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

  1. First posted13 Sep 2010 (estimated)
  2. Study startSep 2010
  3. Primary completionAug 2011
  4. Study completionNov 2011

Resources

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
25 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01199705
Sponsor's protocol ID
ZLB06_002CR
Other IDs
U1111-1116-6379
Status last verified
Mar 2013
First submitted
8 Sep 2010
Last updated
12 Dec 2014
Data monitoring committee
No

Publications

  • Igarashi A, Kanegane H, Kobayashi M, Miyawaki T, Tsutani K. Cost-minimization analysis of IgPro20, a subcutaneous immunoglobulin, in Japanese patients with primary immunodeficiency. Clin Ther. 2014 Nov 1;36(11):1616-24. doi: 10.1016/j.clinthera.2014.08.007. Epub 2014 Sep 16.

    PubMed 25236916

  • Kanegane H, Imai K, Yamada M, Takada H, Ariga T, Bexon M, Rojavin M, Hu W, Kobayashi M, Lawo JP, Nonoyama S, Hara T, Miyawaki T. Efficacy and safety of IgPro20, a subcutaneous immunoglobulin, in Japanese patients with primary immunodeficiency diseases. J Clin Immunol. 2014 Feb;34(2):204-11. doi: 10.1007/s10875-013-9985-z. Epub 2014 Feb 7.

    PubMed 24504846

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Immune Globulin Subcutaneous (Human) (SCIG) in people with Primary Immune Deficiency, run by CSL Behring at 10 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 3 inclusion and 8 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 12 Dec 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 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 20 Mar 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?

IgG Trough Level (time frame: During IVIG period (IV 1, IV 2, IV 3) and during SCIG period at weeks 16, 20, and 24). 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 NCT01199705, which also lists 2 linked publications.

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 12 Dec 2014.

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