Clinical trial NCT00719680: Extension Study of Subcutaneous Immunoglobulin Human in Patients With Primary Immunodeficiency (PID)
Official title: A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency (PID)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Primary Immune Deficiency
Keywords: CVID
XLA
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2008-07-22
Enrollment: 21
Sex: ALL
Minimum age (years): 2
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 purpose of this study is to determine whether a long-term use of a new human immunoglobulin G with proline (IgPro) is safe and effective in the treatment of primary immunodeficiency.


Eligibility criteria: Inclusion Criteria:

* Subjects with primary humoral immunodeficiency who have participated in the study ZLB04_009CR (NCT00419341), namely with a diagnosis of Common Variable Immunodeficiency (CVID) as defined by PAGID (Pan-American Group for Immunodeficiency) and ESID (European Society for Immunodeficiencies) or X-linked Agammaglobulinemia (XLA) as defined by PAGID and ESID
* Women of childbearing potential must be using and agree to continue using medically approved contraception and must have a negative pregnancy test at screening
* Written informed consent

Exclusion Criteria:

* Ongoing serious bacterial infection at the time of screening
* Malignancies of lymphoid cells such as lymphocytic leukemia, Non-Hodgkin's lymphoma, and immunodeficiency with thymoma
* Hypoalbuminemia, protein-losing enteropathies, and any proteinuria (defined by total urine protein concentration \> 0.2 g/L)
* Other significant medical conditions that could increase the risk to the patient
* Females who are pregnant, breast-feeding or planning a pregnancy during the course of the study
* A positive result at screening on any of the following viral markers: Human immunodeficiency virus (HIV), Hepatitis C virus (HCV) or Hepatitis B virus (HBV)
* Aspartate aminotransferase (ASAT) or Alanine aminotransferase (ALAT) concentration \> 2.5 times Upper Normal Limit (UNL) at Completion Visit of study ZLB04_009CR (NCT00419341)
* Creatinine concentration \> 1.5 times UNL at Completion Visit of study ZLB04_009CR (NCT00419341)
* Participation in a study with an investigational product other than IgPro20 within 3 months prior to enrollment
* Evidence of uncooperative attitude
* Any condition that is likely to interfere with evaluation of the study drug or satisfactory conduct of the trial
* Subjects who are employees at the investigational site, relatives or spouse of the investigator

Primary outcomes:
measure: Annualized Rate of Serious Bacterial Infection (Intention-to-Treat Population)
timeFrame: For the duration of the study, up to approximately 104 weeks
measure: Annualized Rate of Serious Bacterial Infection (Per-Protocol Efficacy Population)
timeFrame: For the duration of the study, up to approximately 104 weeks
Secondary outcomes:
measure: Annualized Rate of Any Infection
timeFrame: For the duration of the study, up to approximately 104 weeks
measure: Trough Levels of Total Immunoglobulin G (IgG) Serum Concentrations
timeFrame: Before infusion at Weeks 1, 24, 48, 72, and 96
measure: Number of Days Out of Work / School / Kindergarten / Day Care or Inability to Perform Normal Activities Due to Infection
timeFrame: For the duration of the study, up to approximately 104 weeks
measure: Annualized Rate of Days Out of Work / School / Kindergarten / Day Care or Inability to Perform Normal Activities Due to Infection
timeFrame: For the duration of the study, up to approximately 104 weeks
measure: Number of Days of Hospitalization Due to Infection
timeFrame: For the duration of the study, up to approximately 104 weeks
measure: Annualized Rate of Hospitalization Due to Infection
timeFrame: For the duration of the study, up to approximately 104 weeks
measure: Use of Antibiotics for Infection Prophylaxis and Treatment
timeFrame: For the duration of the study, up to approximately 104 weeks
measure: Rate of All AEs by Relatedness and Severity
timeFrame: For the duration of the study, up to approximately 104 weeks
measure: Relatedness and Severity of All AEs (Percentage of Total AEs)
timeFrame: For the duration of the study, up to approximately 104 weeks
measure: Number of Subjects With Any Temporally Associated Adverse Event (AE) Within 24 or 72 Hours After an Infusion
timeFrame: Within 24 or 72 hours after each infusion
measure: Rate of Temporally Associated AEs Within 24 or 72 Hours of an Infusion
timeFrame: Within 24 or 72 hours after each infusion
measure: Number of Subjects Reporting Mild, Moderate, or Severe Local AEs
timeFrame: For the duration of the study, up to approximately 104 weeks
measure: Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Vital Signs
timeFrame: At weeks 1, 12, 24, 36, 48, 60, 72, 84, and 96
measure: Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Routine Laboratory Parameters
timeFrame: At Week 1, and study completion (approximately 104 weeks)
measure: Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Viral Safety Markers
timeFrame: At Week 1, and study completion (approximately 104 weeks)
Other outcomes:
Locations:
city: Centennial
country: United States
facility: Contact CSL Behring for facility details
state: Colorado
city: North Palm Beach
country: United States
facility: Contact CSL Behring for facility details
state: Florida
city: Indianapolis
country: United States
facility: Contact CSL Behring for facility details
state: Indiana
city: Dallas
country: United States
facility: Contact CSL Behring for facility details
state: Texas
Study officials:
affiliation: CSL Behring
name: Program Director
role: STUDY_DIRECTOR
References:
citation: Jolles S, Borte M, Nelson RP Jr, Rojavin M, Bexon M, Lawo JP, Wasserman RL. Long-term efficacy, safety, and tolerability of Hizentra(R) for treatment of primary immunodeficiency disease. Clin Immunol. 2014 Feb;150(2):161-9. doi: 10.1016/j.clim.2013.10.008. Epub 2013 Oct 26.