Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (EU Extension Study)

Primary Immunodeficiency (PID) Clinical Trial

Official title:

A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency (IgPro20 EU Extension Study)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00751621
• Other study ID #: ZLB07_002CR
• Secondary ID: 14722008-000830-
• Status: Completed
• Phase: Phase 3
• First received: September 11, 2008
• Last updated: March 5, 2014
• Start date: August 2008
• Est. completion date: December 2011

Study information

• Verified date: December 2012
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)Germany: Paul-Ehrlich-InstitutRomania: National Medicines AgencySpain: Spanish Agency of MedicinesSweden: Medical Products AgencySwitzerland: SwissmedicUnited Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial Summary

This study is a continuation of the study ZLB06_001CR with the objective of assessing efficacy, tolerability, safety of IgPro, as well as long-term health-related quality of life in patients with PID.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: December 2011
• Est. primary completion date: December 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 2 Years to 65 Years

Eligibility

Inclusion Criteria:

• Subjects with primary humoral immunodeficiency, namely with a diagnosis of Common variable immunodeficiency (CVID) as defined by the Pan-American Group for Immunodeficiency (PAGID) and the European Society for Immunodeficiencies (ESID), or X-linked agammaglobulinemia (XLA) as defined by PAGID and ESID, or autosomal recessive agammaglobulinemia who have participated in the study ZLB06_001CR and who have tolerated IgPro well

• Written informed consent

Exclusion Criteria:

• Hypoalbuminemia, protein-losing enteropathies, and any proteinuria (defined as total urine protein concentration > 0.2g/L)

• Other significant medical conditions that could increase the risk to the subject

• Females who are pregnant, breast feeding or planning a pregnancy during the course of the study

• Participation in a study with an investigational medicinal product within three months prior to enrollment, except for ZLB06_001CR

• Evidence of uncooperative attitude

• Any condition that is likely to interfere with evaluation of the IMP or satisfactory conduct of the study

• Subjects who are employees at the investigational site, relatives or spouse of the investigator

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 Immunodeficiency (PID)

Intervention

Biological:
• IgPro20

Locations

Country	Name	City	State
France	Study Site	Paris
Germany	Study Site	Berlin
Germany	Study Site	Freiburg
Germany	Study Site	Leipzig
Germany	Study Site	Mainz
Poland	Study Site	Warsaw
Romania	Study Site	Cluj-Napoca
Romania	Study Site	Timisoara
Spain	Study Site	Barcelona
Spain	Study Site	Sevilla
Sweden	Study Site	Göteborg
Switzerland	Study Site	Bern
United Kingdom	Study Site	London

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Countries where clinical trial is conducted

France,  Germany,  Poland,  Romania,  Spain,  Sweden,  Switzerland,  United Kingdom, 

References & Publications (1)

Jolles S, Borte M, Nelson RP Jr, Rojavin M, Bexon M, Lawo JP, Wasserman RL. Long-term efficacy, safety, and tolerability of Hizentra® for treatment of primary immunodeficiency disease. Clin Immunol. 2014 Feb;150(2):161-9. doi: 10.1016/j.clim.2013.10.008. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Total Serum IgG Trough Levels	The IgG trough values per subject were aggregated to a median value, and then median values across subjects were summarized using descriptive statistics.	Up to 42 months	No
Secondary	Annualized Rate of Clinically Documented Serious Bacterial Infections (SBIs)	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 analysis population and adjusted to 365 days.; Potential SBIs included bacterial pneumonia, bacteremia and septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess. If an adverse event (AE) was identified as a potential SBI, the AE was adjudicated by the Medical Monitor and Investigator to determine if the event fulfilled the predefined criteria for SBIs.	Up to 42 months	No
Secondary	Annualized Rate of Infection Episodes	The annualized rate was based on the total number of infection episodes occurring during the study divided by the total number of subject study days for all subjects in the specified analysis population and adjusted to 365 days.	Up to 42 months	No
Secondary	Number of Infection Episodes	Total number of infections for the specified analysis population	Up to 42 months	No
Secondary	Annualized Rate of Days Out of Work / School / Kindergarten / Day Care or Unable to Perform Normal Activities Due to Infections	The annualized rate was based on the total number of days out of work / school / kindergarten / day care or inability to perform normal activities due to infection, and the total number of subject diary days for all subjects in the specified analysis population and adjusted to 365 days.	Up to 42 months	No
Secondary	Number of Days Out of Work / School / Kindergarten / Day Care or Unable to Perform Normal Activities Due to Infections	Total number of days out of work / school / kindergarten / day care or unable to perform normal activities due to infections, for the specified analysis population	Up to 42 months	No
Secondary	Annualized Rate of Hospitalization Due to Infections	The annualized rate was based on the total number of days of hospitalization due to infections and the total number of subject diary days for all subjects in the specified analysis population and adjusted to 365 days.	Up to 42 months	No
Secondary	Number of Days of Hospitalization Due to Infections	Total number of days of hospitalization due to infections for the specified analysis population	Up to 42 months	No
Secondary	Use of Antibiotics for Infection Prophylaxis and Treatment	Annualized rate of days with antibiotics for infection prophylaxis and treatment. The annualized rate was based on the total number of days of antibiotic use for infection prophylaxis and treatment in the efficacy period, and the total number of subject study days for all subjects in the specified analysis population, and adjusted to 365 days.	Up to 42 months	No
Secondary	Health Related Quality of Life (Short Form 36 Health Survey)	The Short Form 36 Health Survey (SF-36) is a 36-item questionnaire that measures generic health concepts that are relevant across age, disease, and treatment groups. The questions are grouped into eight domains: physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role-emotional, and mental health. Scores range from 0 to 100, with higher scores indicating a better health state.	At baseline and at the last available post-baseline observation for each subject (up to 42 months)	No
Secondary	Clinically Relevant Changes in Vital Signs From Baseline to the Completion Visit.	The total number of subjects with clinically relevant changes in vital signs from baseline to the completion visit. Vital signs included heart rate, systolic blood pressure, diastolic blood pressure, and body temperature.	At baseline (data either from Infusion 40 or the completion visit of study ZLB06_001CR), and at completion (up to 42 months).	Yes
Secondary	Clinically Significant Abnormal Changes in Routine Laboratory Parameters Between Baseline and the Completion Visit.	The total number of subjects with clinically significant abnormal changes in routine laboratory parameters between baseline and the completion visit. Routine laboratory parameters included haematology, serum chemistry and urinalysis.	At baseline (data either from Infusion 40 or the completion visit of study ZLB06_001CR), and at completion (up to 42 months).	Yes
Secondary	Rate, Severity and Relatedness of Any Adverse Events (AEs) Per Infusion	The rate of AEs was the number of AEs over the number of infusions administered. Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities. At least possibly related AEs included possibly related AEs, probably related AEs, and related AEs.	Up to 42 months	Yes

External Links

•   Click here to request more information about this study