Clinical trial NCT01199705: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Primary Immune Deficiency
Keywords: Immune globulin subcutaneous
SCIG
Primary immunodeficiency
PID
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2010-09-13
Enrollment: 25
Sex: ALL
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: IgG Trough Level
timeFrame: During IVIG period (IV 1, IV 2, IV 3) and during SCIG period at weeks 16, 20, and 24
Secondary outcomes:
measure: Number of Infection Episodes (Serious and Non-serious) by Study Period
timeFrame: Up to 36 weeks
measure: Rate of Infection Episodes (Serious and Non-serious) by Study Period, PPS Population
timeFrame: Up to 36 weeks
measure: Rate of Infection Episodes (Serious and Non-serious) by Study Period, FAS Population
timeFrame: Up to 36 weeks
measure: Number of Days Out of Work/School/Kindergarten/Day Care or Unable to Perform Normal Daily Activities Due to Infections by Study Period
timeFrame: Up to 36 weeks
measure: Number of Days of Hospitalization Due to Infections by Study Period
timeFrame: Up to 36 weeks
measure: Duration of Use of Antibiotics for Infection Prophylaxis and Treatment
timeFrame: Up to 36 weeks
measure: Rate of All Adverse Events by Relatedness and Seriousness
timeFrame: For the duration of the study, up to 36 weeks
measure: Rate of Mild, Moderate, or Severe Local Reactions
timeFrame: For the duration of the study, up to 36 weeks
Other outcomes:
measure: Annualized Rate of Serious Bacterial Infections (SBIs), PPS Population
timeFrame: Up to 36 weeks
measure: Annualized Rate of Serious Bacterial Infections (SBIs), FAS Population
timeFrame: Up to 36 weeks
Locations:
city: Nagoya city
country: Japan
facility: Study site
state: Aichi Pref.
city: Chiba city
country: Japan
facility: Study site
state: Chiba Pref.
city: Gifu city
country: Japan
facility: Study site
state: Gifu Pref.
city: Sapporo city
country: Japan
facility: Study site
state: Hokkaido
city: Sendai city
country: Japan
facility: Study site
state: Miyagi Pref.
city: Fukuoka city
country: Japan
facility: Study site
state: Osaka
city: Moriguchi city
country: Japan
facility: Study site
state: Osaka
city: Osaka city
country: Japan
facility: Study site
state: Osaka
city: Koshigaya city
country: Japan
facility: Study site
state: Saitama Pref.
city: Tokorozawa city
country: Japan
facility: Study site
state: Saitama Pref.
Study officials:
affiliation: CSL Behring K.K.
name: Yoriyuki Shiga
role: STUDY_DIRECTOR
References:
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.
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.