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

Primary Purpose

Primary Immune Deficiency

Status
Completed
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
Immune Globulin Subcutaneous (Human) (SCIG)
Sponsored by
CSL Behring
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Immune Deficiency focused on measuring Immune globulin subcutaneous, SCIG, Primary immunodeficiency, PID

Eligibility Criteria

undefined - 75 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Sites / Locations

  • Study site
  • Study site
  • Study site
  • Study site
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Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

IgPro20

Arm Description

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.
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)
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)
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).
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).
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).
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.
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.

Full Information

First Posted
September 8, 2010
Last Updated
November 25, 2014
Sponsor
CSL Behring
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1. Study Identification

Unique Protocol Identification Number
NCT01199705
Brief Title
Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study)
Official Title
A Multicenter Study of Efficacy, Safety, Tolerability, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency
Study Type
Interventional

2. Study Status

Record Verification Date
March 2013
Overall Recruitment Status
Completed
Study Start Date
September 2010 (undefined)
Primary Completion Date
August 2011 (Actual)
Study Completion Date
November 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CSL Behring

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief 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). In addition, the study will assess the health-related quality of life and pharmacoeconomic aspects related to treatment with IgPro20.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Immune Deficiency
Keywords
Immune globulin subcutaneous, SCIG, Primary immunodeficiency, PID

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
25 (Actual)

8. Arms, Groups, and Interventions

Arm Title
IgPro20
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
Immune Globulin Subcutaneous (Human) (SCIG)
Other Intervention Name(s)
Hizentra
Intervention Description
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.
Primary Outcome Measure Information:
Title
IgG Trough Level
Description
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.
Time Frame
During IVIG period (IV 1, IV 2, IV 3) and during SCIG period at weeks 16, 20, and 24
Secondary Outcome Measure Information:
Title
Number of Infection Episodes (Serious and Non-serious) by Study Period
Description
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.
Time Frame
Up to 36 weeks
Title
Rate of Infection Episodes (Serious and Non-serious) by Study Period, PPS Population
Description
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)
Time Frame
Up to 36 weeks
Title
Rate of Infection Episodes (Serious and Non-serious) by Study Period, FAS Population
Description
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)
Time Frame
Up to 36 weeks
Title
Number of Days Out of Work/School/Kindergarten/Day Care or Unable to Perform Normal Daily Activities Due to Infections by Study Period
Description
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).
Time Frame
Up to 36 weeks
Title
Number of Days of Hospitalization Due to Infections by Study Period
Description
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).
Time Frame
Up to 36 weeks
Title
Duration of Use of Antibiotics for Infection Prophylaxis and Treatment
Description
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).
Time Frame
Up to 36 weeks
Title
Rate of All Adverse Events by Relatedness and Seriousness
Description
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.
Time Frame
For the duration of the study, up to 36 weeks
Title
Rate of Mild, Moderate, or Severe Local Reactions
Description
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.
Time Frame
For the duration of the study, up to 36 weeks
Other Pre-specified Outcome Measures:
Title
Annualized Rate of Serious Bacterial Infections (SBIs), PPS Population
Description
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 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; 12 weeks) SCIG IgPro20 treatment (efficacy; 12 weeks)
Time Frame
Up to 36 weeks
Title
Annualized Rate of Serious Bacterial Infections (SBIs), FAS Population
Description
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 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; 12 weeks) SCIG IgPro20 treatment (efficacy; 12 weeks)
Time Frame
Up to 36 weeks

10. Eligibility

Sex
All
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 Exclusion Criteria: 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yoriyuki Shiga
Organizational Affiliation
CSL Behring K.K.
Official's Role
Study Director
Facility Information:
Facility Name
Study site
City
Nagoya city
State/Province
Aichi Pref.
ZIP/Postal Code
466-8560
Country
Japan
Facility Name
Study site
City
Chiba city
State/Province
Chiba Pref.
ZIP/Postal Code
260-8677
Country
Japan
Facility Name
Study site
City
Gifu city
State/Province
Gifu Pref.
ZIP/Postal Code
501-1194
Country
Japan
Facility Name
Study site
City
Sapporo city
State/Province
Hokkaido
ZIP/Postal Code
060-8648
Country
Japan
Facility Name
Study site
City
Sendai city
State/Province
Miyagi Pref.
ZIP/Postal Code
980-8574
Country
Japan
Facility Name
Study site
City
Fukuoka city
State/Province
Osaka
ZIP/Postal Code
812-8582
Country
Japan
Facility Name
Study site
City
Moriguchi city
State/Province
Osaka
ZIP/Postal Code
570-8507
Country
Japan
Facility Name
Study site
City
Osaka city
State/Province
Osaka
ZIP/Postal Code
534-0021
Country
Japan
Facility Name
Study site
City
Koshigaya city
State/Province
Saitama Pref.
ZIP/Postal Code
343-8555
Country
Japan
Facility Name
Study site
City
Tokorozawa city
State/Province
Saitama Pref.
ZIP/Postal Code
359-8513
Country
Japan

12. IPD Sharing Statement

Citations:
PubMed Identifier
25236916
Citation
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.
Results Reference
background
PubMed Identifier
24504846
Citation
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.
Results Reference
result
Links:
URL
http://www.cslbehring.com/clinical-trials/contact-us.htm?registryRefNum=NCT01199705&registryName=ctgov
Description
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Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study)

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