A Multicentre Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- CSL Behring
- Enrollment
- 51
- Locations
- 20
- Primary Endpoint
- Total Serum IgG Trough Levels
Study Overview
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).
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 2 Years to 65 Years (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
IgPro20
Intervention: Human Normal Immunoglobulin for Subcutaneous Administration (IGSC) (Biological)
Outcomes
Primary Outcomes
Total Serum IgG Trough Levels
Time Frame: Up to 6 months prior to first IgPro20 treatment (Pre-study treatment) and Week 12 to 17 (IgPro20 treatment)
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.
Secondary Outcomes
- Annual Rate of the Number of Days of Hospitalization Due to Infections(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)(Efficacy period: week 12 to week 40 after study start or to the completion visit)
- Annual Rate of Clinically Documented Serious Bacterial Infections (ITT Population)(Efficacy period: week 12 to week 40 after study start or to the completion visit)
- Annual Rate of Infection Episodes(Efficacy period: week 12 to week 40 after study start or to the completion visit)
- Annual Rate of Days Out of Work / School / Kindergarten / Day Care or Unable to Perform Normal Activities Due to Infections(Efficacy period: week 12 to week 40 after study start or to the completion visit)
- Annual Rate of Antibiotic Use for Infection Prophylaxis and Treatment(Efficacy period: week 12 to week 40 after study start or to the completion visit)