A Phase 3 interventional study of Immune Globulin Subcutaneous (Human) (SCIG) in Primary Immune Deficiency, sponsored by CSL Behring. Completed at 10 sites in Japan. Open to participants aged Up to 75 Years. Per ClinicalTrials.gov, last updated 2014-12-12.
Sponsored by CSL Behring · Phase 3, Interventional, and Treatment
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.
199 studies on the registry are indexed under Primary Immunodeficiency Diseases; 46 are open to participants now.
This study's enrollment of 25 is below the median of 37 across 121 interventional studies indexed under Primary Immunodeficiency Diseases.
Browse Primary Immunodeficiency Diseases studies →CSL Behring is the lead sponsor of 142 studies on the registry; 18 are open to participants now.
Of its 24 completed or terminated interventional studies of FDA-regulated products, 17 (71%) have results posted.
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Exclusion Criteria:
Biological: Immune Globulin Subcutaneous (Human) (SCIG)
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.
Also known as: Hizentra
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.
Time frame: During IVIG period (IV 1, IV 2, IV 3) and during SCIG period at weeks 16, 20, and 24
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.
Time frame: 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: * 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
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)
Time frame: 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).
Time frame: 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).
Time frame: 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).
Time frame: 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.
Time frame: 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.
Time frame: For the duration of the study, up to 36 weeks
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: * 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
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: * 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
This multicenter study enrolled subjects at nine of the participating study centers in Japan.
| Milestone | IgPro20 |
|---|---|
| Started | 25 |
| Completed | 25 |
| Not completed | 0 |
| Milestone | IgPro20 |
|---|---|
| Started | 25 |
| Completed | 24 |
| Not completed | 1 |
| Withdrew: Transfer of residence | 1 |
| Milestone | IgPro20 |
|---|---|
| Started | 24 |
| Completed | 24 |
| Not completed | 0 |
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.
| Ratio of Geometric Means | IgPro20 - Per Protocol Set (PPS) | IgPro20 - Full Analysis Set (FAS) |
|---|---|---|
| IgG Trough Level | 1.09 (1.06 to 1.13) | 1.11 (1.08 to 1.15) |
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.
| Number of infection episodes | IgPro20 - Per Protocol Set (PPS) | IgPro20 - Full Analysis Set (FAS) |
|---|---|---|
| IVIG Treatment | 19 | 22 |
| SCIG IgPro20 Treatment (Wash-in/Wash-out) | 28 | 32 |
| SCIG IgPro20 Treatment (Efficacy) | 15 | 18 |
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)
| Infections per subject year | IVIG Treatment | SCIG Treatment (Wash-in/Wash-out) | SCIG Treatment (Efficacy) |
|---|---|---|---|
| Rate of Infection Episodes (Serious and Non-serious) by Study Period, PPS Population | 5.74 | 5.79 | 2.98 |
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)
| Infections per subject year | IVIG Treatment | SCIG Treatment (Wash-in/Wash-out) | SCIG Treatment (Efficacy) |
|---|---|---|---|
| Rate of Infection Episodes (Serious and Non-serious) by Study Period, FAS Population | 5.75 | 5.79 | 3.14 |
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)
| SBIs per subject year | IVIG Treatment | SCIG IgPro20 Treatment (Wash-in/Wash-out) | SCIG IgPro20 Treatment (Efficacy) |
|---|---|---|---|
| Annualized Rate of Serious Bacterial Infections (SBIs), PPS Population | 0.00 | 0.00 | 0.00 |
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).
| Days | IgPro20 - Per Protocol Set (PPS) | IgPro20 - Full Analysis Set (FAS) |
|---|---|---|
| IVIG Treatment | 0 (0 to 8) | 0 (0 to 8) |
| SCIG IgPro20 Treatment (Wash-in/Wash-out) | 0 (0 to 9) | 0 (0 to 9) |
| SCIG IgPro20 Treatment (Efficacy) | 0 (0 to 8) | 0 (0 to 8) |
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)
| SBIs per subject year | IVIG Treatment | SCIG IgPro20 Treatment (Wash-in/Wash-out) | SCIG IgPro20 Treatment (Efficacy) |
|---|---|---|---|
| Annualized Rate of Serious Bacterial Infections (SBIs), FAS Population | 0.00 | 0.00 | 0.00 |
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).
| Days | IgPro20 - Per Protocol Set (PPS) | IgPro20 - Full Analysis Set (FAS) |
|---|---|---|
| IVIG Treatment | 0 (0 to 1) | 0 (0 to 1) |
| SCIG IgPro20 Treatment (Wash-in/Wash-out) | 0 (0 to 0) | 0 (0 to 0) |
| SCIG IgPro20 Treatment (Efficacy) | 0 (0 to 3) | 0 (0 to 3) |
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).
| Days | IgPro20 - Per Protocol Set (PPS) | IgPro20 - Full Analysis Set (FAS) |
|---|---|---|
| IVIG Treatment | 48.5 (2 to 64) | 48.0 (2 to 64) |
| SCIG IgPro20 Treatment (Wash-in/Wash-out) | 49.0 (3 to 86) | 35.5 (2 to 86) |
| SCIG IgPro20 Treatment (Efficacy) | 71.0 (6 to 85) | 71.0 (6 to 85) |
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.
| AEs per infusion | IVIG Treatment | SCIG Treatment |
|---|---|---|
| All AEs | 0.653 | 0.457 |
| At Least Possibly Related AEs | 0.027 | 0.296 |
| Serious AEs | 0 | 0.002 |
| At Least Possibly Related and Serious AEs | 0 | 0 |
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.
| AEs per infusion | IVIG Treatment | SCIG Treatment |
|---|---|---|
| Mild Local Reactions | 0 | 0.274 |
| Moderate Local Reactions | 0 | 0 |
| Severe Local Reactions | 0 | 0 |
Collected over For the duration of the study, up to 36 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| IVIG Treatment | — | 1/25 (4%) | 20/25 (80%) |
| SCIG Treatment | — | 1/25 (4%) | 24/25 (96%) |
| Event | IVIG Treatment | SCIG Treatment |
|---|---|---|
| Bacterial infectionInfections and infestations | 0/25 | 1/25 |
| GastroenteritisInfections and infestations | 1/25 | 0/25 |
| Event | IVIG Treatment | SCIG Treatment |
|---|---|---|
| Local reactionsGeneral disorders | 0/25 | 20/25 |
| NasopharyngitisInfections and infestations | 5/25 | 11/25 |
| Upper respiratory tract infectionInfections and infestations | 4/25 | 5/25 |
| InfluenzaInfections and infestations | 4/25 | 4/25 |
| Dental cariesGastrointestinal disorders | 1/25 | 3/25 |
| BronchitisInfections and infestations | 0/25 | 3/25 |
| GastroenteritisInfections and infestations | 1/25 | 3/25 |
| SinusitisInfections and infestations | 0/25 | 3/25 |
| Arthropod biteInjury, poisoning and procedural complications | 0/25 | 3/25 |
| HeadacheNervous system disorders | 3/25 | 1/25 |
| Age, Continuous(Years) | IgPro20 |
|---|---|
| Mean | 20.6 ± 13.23 |
| Age, Customized(Participants) | IgPro20 |
|---|---|
| < 2 years | 0 |
| ≥ 2 to < 12 years | 7 |
| ≥ 12 to ≤ 16 years | 4 |
| > 16 to < 65 years | 14 |
| ≥ 65 years | 0 |
| Sex: Female, Male(Participants) | IgPro20 |
|---|---|
| Female | 9 |
| Male | 16 |
| Primary Immunodeficiency Type(Participants) | IgPro20 |
|---|---|
| Common Variable Immunodeficiency (CVID) | 10 |
| X-Linked Agammaglobulinemia (XLA) | 13 |
| Autosomal Recessive Agammaglobulinemia (ARAG) | 1 |
| Hyper-Immunoglobulin M (IgM) Syndrome | 1 |
This study is completed, as verified in Mar 2013. You cannot join it, but the record below documents what was studied.
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