Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy

CSL Behring51 enrolled

Overview

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

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).

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Common Variable Immunodeficiency X-linked Agammaglobulinemia Autosomal Recessive Agammaglobulinemia

Interventions

Human Normal Immunoglobulin for Subcutaneous Administration (IGSC)BIOLOGICAL

IgPro20 is a liquid formulation of normal human IgG at a concentration of 20% administered as a SC infusion at weekly intervals. The initial weekly dose was determined based on subjects' previous treatment. Dose adjustments could be performed during the wash-in/wash-out period at the discretion of the investigator.

Eligibility

Sex: ALLMin age: 2 YearsMax age: 65 Years

Medical Language ↔ Plain English

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.

Locations (19)

Study site

Paris, France

Study site

Berlin, Germany

Study site

Düsseldorf, Germany

Outcomes

Primary Outcomes

Total Serum IgG Trough Levels

Total IgG trough levels for IgPro20 treatment at steady state were compared with documented trough level data for IgG treatment received prior to enrolling in the study (either subcutaneous or intravenous IgG). For this purpose, 6 consecutive IgPro20 trough values (obtained prior to infusions 12 to 17) per subject were aggregated to the subject's median value and then median values across subjects were summarised using descriptive statistics. The same procedure was applied to pre-study treatment using the 3 most recent IgG trough values ≥ 5 g/L obtained prior to the first IgPro20 infusion.

Time frame: Up to 6 months prior to first IgPro20 treatment (Pre-study treatment) and Week 12 to 17 (IgPro20 treatment)

Secondary Outcomes

Annual Rate of Clinically Documented Serious Bacterial Infections (ITT Population)

Serious bacterial infections (SBIs) included bacterial pneumonia, bacteraemia/septicaemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess. Diagnosis of the SBIs was based on the presence of predefined clinical signs and symptoms as well as on laboratory parameters. The annual rate was calculated based on the total number of SBIs and the total number of study days during the efficacy period for all subjects in the ITT population and adjusted to 365 days.

Time frame: Efficacy period: week 12 to week 40 after study start or to the completion visit

Annual Rate of Clinically Documented Serious Bacterial Infections (PPE Population)

Time frame: Efficacy period: week 12 to week 40 after study start or to the completion visit

Data from ClinicalTrials.gov

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