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

Primary Immune Deficiency Disorder Clinical Trial

Official title:

A Multicenter Follow-up Study of Long-term Safety, Tolerability, and Efficacy of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01458171
• Other study ID #: ZLB07_001CR
• Status: Completed
• Phase: Phase 3
• First received: October 12, 2011
• Last updated: February 27, 2013
• Start date: April 2011
• Est. completion date: April 2012

Study information

• Verified date: February 2013
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Health authority: Japan: Pharmaceuticals and Medical Devices Agency
• Study type: Interventional

Clinical Trial Summary

The objective of this study is to assess the long-term safety, tolerability, and efficacy of IgPro20 in subjects with primary immunodeficiency (PID) as a follow-up to the pivotal study ZLB06_002CR (NCT01199705).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 23
• Est. completion date: April 2012
• Est. primary completion date: February 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 75 Years

Eligibility

Inclusion Criteria:

• Subjects who have participated in study ZLB06_002CR and who have tolerated IgPro20 well.

• Written informed consent by the subject/parent/legally acceptable representative. Written assent for an underage subject (=7 years at the time of obtaining informed consent), as far as possible.

Exclusion Criteria:

• Ongoing serious bacterial infections (SBIs) (pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, or visceral abscess) at the time of the first infusion.

• Hypoalbuminemia, protein-losing enteropathies, and any proteinuria (known total urine protein concentration >0.2 g/L or urine protein ++ by dipstick).

• Pregnancy or nursing mother.

• Participation in a study with an investigational medicinal product (IMP) within 3 months prior to enrollment except for ZLB06_002CR.

• Subjects who are planning to donate blood during the study.

• Re-entry of subjects previously participating in the current follow-up study.

• Known or suspected antibodies to the IMP, or to excipients of the IMP.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Immunologic Deficiency Syndromes
• Primary Immune Deficiency Disorder

Intervention

Biological:
• Immune globulin subcutaneous (Human)
IgPro20 is a 20% (weight per volume [w/v]) liquid formulation of human immunoglobulin for subcutaneous (SC) use. Subjects will receive weekly infusions of IgPro20 for a total of 24 weeks at a dose based on the subject's IgPro20 dose in the pivotal study ZLB06_002CR (NCT01199705).

Locations

Country	Name	City	State
Japan	Study site	Bunkyo-ku	Tokyo Metropolitan
Japan	Study site	Chiba city	Chiba Pref.
Japan	Study site	Fukuoka city	Fukuoka
Japan	Study site	Gifu city	Gifu Pref.
Japan	Study site	Koshigaya city	Saitama Pref.
Japan	Study site	Moriguchi city	Osaka
Japan	Study site	Nagoya city	Aichi Pref.
Japan	Study site	Sapporo city	Hokkaido
Japan	Study site	Tokorozawa city	Saitama Pref.

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Rate of Infection Episodes (Serious and Non-serious)	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 FAS population and the PPS population and adjusted to 365 days.	24 weeks	No
Primary	Median of the Individual Subject's Rate of Adverse Events (AEs) Per Infusion	The rate was calculated by counting all newly developed or worsened AEs within a subject and dividing by the total number of IgPro20 infusions administered to this subject. Subsequently, the median of these individual AE rates per infusion was calculated. AE rates were classified (i) by severity (mild, moderate, severe) and (ii) by causal relationship to study medication (not related or unlikely related; at least possibly related [i.e., possibly related, probably related, or related]).	24 weeks	Yes
Secondary	Overall Rate of AEs Per Infusion	The rate was calculated by counting all newly developed or worsened AEs during the treatment period in all subjects and dividing the total number of AEs by the total number of IgPro20 infusions administered. In addition, individual AEs were classified (i) by severity (mild, moderate, severe) and (ii) by causal relationship to study medication (not related or unlikely related; at least possibly related [i.e., possibly related, probably related, or related]). The AE rates per infusion by severity and causal relationship to study medication were calculated by dividing the number of AEs in each category by the total number of IgPro20 infusions.	24 weeks	Yes
Secondary	Number of Subjects With Newly Developing or Worsening AEs	Number of subjects with AEs, overall and classified (i) by severity (mild, moderate, severe) and (ii) by causal relationship to study medication (not related or unlikely related; at least possibly related [i.e., possibly related, probably related, or related]).	24 weeks	Yes
Secondary	Percentage of Infusions With Subject-assessed Tolerability of at Least 'Good'	Subjects assessed their overall perception of local tolerability at the infusion site throughout the study in the subject diary within a time window of 24 h to 72 h after the end of the latest infusion by assessing it as "very good", "good", "fair", or "poor". The reported percentage represents the percentage of subjects with local tolerability assessments of "very good" or "good" at any given study infusion.	24 to 72 hours after infusion	No
Secondary	IgG Trough Level	Serum IgG trough levels at the completion visit compared to the baseline visit of the follow-up study. IgG trough levels at baseline, at the completion visit, and the change from baseline to the completion visit are shown	24 weeks	No
Secondary	Annualized Rate of Clinically Documented Serious Bacterial Infections (SBIs)	SBIs are defined as bacterial pneumonia, bacteremia and septicemia, osteomyelitis/septic arthritis, bacterial meningitis, or visceral abscess. The annualized rate was based on the total number of SBIs and the total number of subject study days for all subjects in the FAS and PPS and adjusted to 365 days.	24 weeks	No
Secondary	Number of Infection Episodes (Serious and Non-serious)		24 weeks	No
Secondary	Number of Days Out of Work/School/Kindergarten/Day Care or Unable to Perform Normal Daily Activities Due to Infections.	Median number of days out of work/school/kindergarten/day care or unable to perform normal daily activities due to infections.	24 weeks	No
Secondary	Number of Days of Hospitalization Due to Infections.	Median number of days of hospitalization due to infections.	24 weeks	No
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	24 weeks	No