Clinical trial NCT00542997: Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy
Official title: A Multicentre Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Common Variable Immunodeficiency
X-linked Agammaglobulinemia
Autosomal Recessive Agammaglobulinemia
Keywords: Subcutaneous immune globulin
SCIG
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2007-10-12
Enrollment: 51
Sex: ALL
Minimum age (years): 2
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The objective of this study is to assess the efficacy, tolerability, safety and pharmacokinetics of IgPro20 in patients with primary humoral immunodeficiency (PID).

Detailed description: This study consisted of a 12-week wash-in/wash-out period followed by a 28-week efficacy period. During the 28-week efficacy period, subjects visited the study site at least every 4 weeks for efficacy and safety evaluations and additionally recorded details regarding IgPro20 dose and certain aspects of efficacy and safety in a diary. Pharmacokinetic (PK) parameters were assessed in a sub-group of subjects during 1 treatment interval at steady-state (Week 28 ± 1).

Eligibility criteria: 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 European Society for Immunodeficiencies (ESID), X-linked agammaglobulinemia (XLA) as defined by PAGID and ESID, or Autosomal Recessive Agammaglobulinemia
* Chest X-ray or CT scan obtained within 1 year prior to enrolment

Exclusion Criteria:

* Newly diagnosed PID, i.e. subjects who have not previously received immunoglobulin replacement therapy
* 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
* Allergic or other severe reactions to immunoglobulins or other blood products associated with high anti-IgA

Additional criteria may apply and examination by an investigator is required to determine eligibility.

Primary outcomes:
measure: Total Serum IgG Trough Levels
timeFrame: Up to 6 months prior to first IgPro20 treatment (Pre-study treatment) and Week 12 to 17 (IgPro20 treatment)
Secondary outcomes:
measure: Annual Rate of Clinically Documented Serious Bacterial Infections (ITT Population)
timeFrame: Efficacy period: week 12 to week 40 after study start or to the completion visit
measure: Annual Rate of Clinically Documented Serious Bacterial Infections (PPE Population)
timeFrame: Efficacy period: week 12 to week 40 after study start or to the completion visit
measure: Annual Rate of Infection Episodes
timeFrame: Efficacy period: week 12 to week 40 after study start or to the completion visit
measure: Annual Rate of Days Out of Work / School / Kindergarten / Day Care or Unable to Perform Normal Activities Due to Infections
timeFrame: Efficacy period: week 12 to week 40 after study start or to the completion visit
measure: Annual Rate of the Number of Days of Hospitalization Due to Infections
timeFrame: Efficacy period: week 12 to week 40 after study start or to the completion visit
measure: Annual Rate of Antibiotic Use for Infection Prophylaxis and Treatment
timeFrame: Efficacy period: week 12 to week 40 after study start or to the completion visit
Other outcomes:
measure: Maximum Concentration (Cmax) of Total Serum IgG
timeFrame: Week 28 (±1week)
measure: Timepoint of Maximum Concentration (Tmax) of Total Serum IgG
timeFrame: Week 28 (±1week)
measure: Area Under the Concentration-Time Curve (AUC_last) of Total Serum IgG
timeFrame: Week 28 (±1week)
measure: Area Under the Concentration-Time Curve (AUCτ) of Total Serum IgG
timeFrame: Week 28 (±1week)
Locations:
city: Paris
country: France
facility: Study site
city: Berlin
country: Germany
facility: Study site
city: Düsseldorf
country: Germany
facility: Study site
city: Freiburg
country: Germany
facility: Study site
city: Hannover
country: Germany
facility: Study site
city: Leipzig
country: Germany
facility: Study site
city: Mainz
country: Germany
facility: Study site
city: Munich
country: Germany
facility: Study site
city: Brescia
country: Italy
facility: Study site
city: Warsaw
country: Poland
facility: Study site
city: Bucharest
country: Romania
facility: Study site
city: Cluj-Napoca
country: Romania
facility: Study site
city: Timisoara
country: Romania
facility: Study site
city: Barcelona
country: Spain
facility: Study site
city: Sevilla
country: Spain
facility: Study site
city: Göteborg
country: Sweden
facility: Study site
city: Berne
country: Switzerland
facility: Study site
city: Cardiff
country: United Kingdom
facility: Study site
city: London
country: United Kingdom
facility: Study site
Study officials:
affiliation: University Hospital of Wales, Cardiff, UK
name: Stephen Jolles, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Jolles S, Bernatowska E, de Gracia J, Borte M, Cristea V, Peter HH, Belohradsky BH, Wahn V, Neufang-Huber J, Zenker O, Grimbacher B. Efficacy and safety of Hizentra((R)) in patients with primary immunodeficiency after a dose-equivalent switch from intravenous or subcutaneous replacement therapy. Clin Immunol. 2011 Oct;141(1):90-102. doi: 10.1016/j.clim.2011.06.002. Epub 2011 Jun 12.
citation: Borte M, Pac M, Serban M, Gonzalez-Quevedo T, Grimbacher B, Jolles S, Zenker O, Neufang-Hueber J, Belohradsky B. Efficacy and safety of hizentra(R), a new 20% immunoglobulin preparation for subcutaneous administration, in pediatric patients with primary immunodeficiency. J Clin Immunol. 2011 Oct;31(5):752-61. doi: 10.1007/s10875-011-9557-z. Epub 2011 Jun 15.