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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1116-6379 | Other Identifier | WHO Universal Trial Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| IgPro20 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Immune Globulin Subcutaneous (Human) (SCIG) | Biological | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | During IVIG period (IV 1, IV 2, IV 3) and during SCIG period at weeks 16, 20, and 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Infection Episodes (Serious and Non-serious) by Study Period | Number of infection episodes (serious and non-serious) presented by study period:
|
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Rate of Serious Bacterial Infections (SBIs), PPS Population | 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:
|
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Yoriyuki Shiga | CSL Behring K.K. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Study site | Nagoya | Aichi Pref. | 466-8560 | Japan | ||
| Study site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 25236916 | Background | 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. | |
| 24504846 | Result | 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. |
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Screening took place 3 to 4 weeks prior to or at the first intravenous immunoglobulin (IVIG) infusion in the IVIG period of the study.
This multicenter study enrolled subjects at nine of the participating study centers in Japan.
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| ID | Title | Description |
|---|---|---|
| FG000 | IgPro20 | Immune Globulin Subcutaneous (Human): IgPro20 is a 20% (weight per volume [w/v]) liquid formulation of human immunoglobulin for subcutaneous use. Subjects will receive weekly infusions of IgPro20 at a weekly dosage calculated based on previous IVIG treatment. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| IVIG Treatment |
| |||||||||||||
| SCIG Treatment (Wash-in/Wash-out) |
| |||||||||||||
| SCIG Treatment (Efficacy) |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | IgPro20 | Immune Globulin Subcutaneous (Human): IgPro20 is a 20% (weight per volume [w/v]) liquid formulation of human immunoglobulin for subcutaneous use. Subjects will receive weekly infusions of IgPro20 at a weekly dosage calculated based on previous IVIG treatment. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | The PPS comprised all subjects with the disease under study who fulfilled the protocol-specified criteria for a) immunoglobulin treatment prior to and during the study, b) availability of evaluable serum IgG levels, and c) dose stability. The FAS comprised all subjects treated with IgPro20 during the efficacy period who had the disease under study. | Posted | Number | 90% Confidence Interval | Ratio of Geometric Means | During IVIG period (IV 1, IV 2, IV 3) and during SCIG period at weeks 16, 20, and 24 |
|
For the duration of the study, up to 36 weeks
The safety data set comprised all subjects treated with the study drug. All SAEs are presented including a pre-treatment SAE of gastroenteritis. In Other AEs, non-serious AEs starting at or after the first study drug infusion are presented. A total of 75 IVIG and 584 SCIG infusions of IgPro20 were administered to 25 subjects during the study.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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). |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bacterial infection | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dental caries | Gastrointestinal disorders | MedDRA (14.1) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trial Disclosure Manager | CSL Behring | Use email contact | clinicaltrials@cslbehring.com |
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| ID | Term |
|---|---|
| D000081207 | Primary Immunodeficiency Diseases |
| ID | Term |
|---|---|
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007153 | Immunologic Deficiency Syndromes |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| D005719 | gamma-Globulins |
| C558471 | Hizentra |
| ID | Term |
|---|---|
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
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|
| Up to 36 weeks |
| 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:
| Up to 36 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:
| Up to 36 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). | Up to 36 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). | Up to 36 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). | Up to 36 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. | For the duration of the study, up to 36 weeks |
| 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. | For the duration of the study, up to 36 weeks |
| Up to 36 weeks |
| Annualized Rate of Serious Bacterial Infections (SBIs), FAS Population | 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:
| Up to 36 weeks |
| Chiba |
| Chiba Pref. |
| 260-8677 |
| Japan |
| Study site | Gifu | Gifu Pref. | 501-1194 | Japan |
| Study site | Sapporo | Hokkaido | 060-8648 | Japan |
| Study site | Sendai | Miyagi Pref. | 980-8574 | Japan |
| Study site | Fukuoka City | Osaka | 812-8582 | Japan |
| Study site | Moriguchi | Osaka | 570-8507 | Japan |
| Study site | Osaka | Osaka | 534-0021 | Japan |
| Study site | Koshigaya | Saitama Pref. | 343-8555 | Japan |
| Study site | Tokorozawa | Saitama Pref. | 359-8513 | Japan |
|
| Years |
|
| Age, Customized | Number | Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Primary Immunodeficiency Type | Number | Participants |
|
| OG001 | IgPro20 - Full Analysis Set (FAS) | The FAS comprised all subjects treated with IgPro20 during the SCIG efficacy period (weeks 13 to 24) who had the disease under study. |
|
|
| Secondary | Number of Infection Episodes (Serious and Non-serious) by Study Period | Number of infection episodes (serious and non-serious) presented by study period:
| Posted | Number | Number of infection episodes | Up to 36 weeks |
|
|
|
| Secondary | 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:
| The PPS comprised all subjects with the disease under study who fulfilled the protocol-specified criteria for a) immunoglobulin treatment prior to and during the study, b) availability of evaluable serum IgG levels, and c) dose stability. | Posted | Number | Infections per subject year | Up to 36 weeks | Subject Study Days | Participants |
|
|
|
| Secondary | 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:
| The FAS comprised all subjects treated with IgPro20 during the efficacy period who had the disease under study. | Posted | Number | Infections per subject year | Up to 36 weeks | Subject Study Days | Participants |
|
|
|
| Other Pre-specified | Annualized Rate of Serious Bacterial Infections (SBIs), PPS Population | 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:
| The PPS comprised all subjects with the disease under study who fulfilled the protocol-specified criteria for a) immunoglobulin treatment prior to and during the study, b) availability of evaluable serum IgG levels, and c) dose stability. | Posted | Number | SBIs per subject year | Up to 36 weeks | Subject Study Days | Participants |
|
|
|
|
| Secondary | 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). | Posted | Median | Full Range | Days | Up to 36 weeks |
|
|
|
| Other Pre-specified | Annualized Rate of Serious Bacterial Infections (SBIs), FAS Population | 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:
| The FAS comprised all subjects treated with IgPro20 during the efficacy period who had the disease under study. | Posted | Number | SBIs per subject year | Up to 36 weeks | Subject Study Days | Participants |
|
|
|
|
| Secondary | 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). | Posted | Median | Full Range | Days | Up to 36 weeks |
|
|
|
| Secondary | 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). | Posted | Median | Full Range | Days | Up to 36 weeks |
|
|
|
| Secondary | 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. | The safety data set (SDS) comprised all subjects treated with the study drug. | Posted | Number | AEs per infusion | For the duration of the study, up to 36 weeks | Infusions | Participants |
|
|
|
| Secondary | 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. | The SDS comprised all subjects treated with the study drug. | Posted | Number | AEs per infusion | For the duration of the study, up to 36 weeks | Infusions | Participants |
|
|
|
| 1 |
| 25 |
| 20 |
| 25 |
| EG001 | SCIG Treatment | IgPro20 was administered subcutaneously with the first 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 followed by a 12-week efficacy period. The IgPro20 dose was to be equal to the weekly equivalent dose of the previous IVIG IgG treatment. | 1 | 25 | 24 | 25 |
| Gastroenteritis | Infections and infestations | MedDRA (14.1) | Systematic Assessment | This SAE occurred between screening and the first IVIG dose and was thus non-treatment-emergent. |
|
| Vomiting | Gastrointestinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Local reactions | General disorders | Systematic Assessment | Local reactions cover MedDRA PTs: infusion site: discomfort, erythema, haemorrhage, induration, inflammation, pain, pruritus, swelling; injection site: erythema, extravasation, induration, irritation, pain, pruritus, swelling; puncture site reaction. |
|
| Bronchitis | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Conjunctivitis infective | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA (14.1) | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA (14.1) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (14.1) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (14.1) | Systematic Assessment |
|
| Malaise | General disorders | MedDRA (14.1) | Systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
CSL agreements and restrictions on publishing may vary with individual investigators; however, CSL will not prohibit any investigator from publishing. CSL supports the publication of results from all centers of a multi-center trial and generally requires that reports based on single-site data not precede the primary publication of the entire clinical trial.
| D000602 |
| Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| SCIG IgPro20 Treatment (Efficacy) |
|
| 0.00 |
| 1-Sided |
| 99 |
| 0.953 |
| No |
| Superiority or Other |
| Annualized Rate | 0.00 | 1-Sided | 99 | 0.914 | No | Superiority or Other |
| SCIG IgPro20 Treatment (Efficacy) |
|
| 0.00 |
| 1-Sided |
| 99 |
| 0.834 |
| No |
| Superiority or Other |
| Annualized Rate | 0.00 | 1-Sided | 99 | 0.802 | No | Superiority or Other |
| SCIG IgPro20 Treatment (Efficacy) |
|
| SCIG IgPro20 Treatment (Efficacy) |
|
| Serious AEs |
|
| At Least Possibly Related and Serious AEs |
|
| Severe Local Reactions |
|