Efficacy, Pharmacokinetics, Safety, and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency
Condition: Primary Immunodeficiency
MeSH terms: Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes
Study type: Interventional
Phase: Phase 3

Summary: 
      Approximately 60 subjects will be enrolled in order to have approximately 20 adult subjects
      and 20 pediatric subjects treated with subcutaneously administered Immune Globulin
      Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) who complete the
      entire study. This study will include 3 study stages: Screening/Previous Regimen Phase, IGSC
      20% Treatment Stage 1 (13 IGSC 20% weekly doses), and IGSC 20% Treatment Stage 2 (39 IGSC 20%
      weekly doses). A total of 52 doses of IGSC 20% will be administered with a final follow-up
      visit 1 week after the last dose at Week 53. Subjects/caregivers will be trained on
      self-administration of IGSC 20% by the clinical site personnel.
    

Detailed description: 
      This is a prospective, multi-center, open-label, single-arm, efficacy, PK, safety and
      tolerability study of IGSC 20% in subjects with PI. Approximately 60 subjects will be
      enrolled in order to have approximately 20 adult subjects and 20 pediatric subjects treated
      with subcutaneously administered IGSC 20% who complete the entire study.

      This study will include 3 study stages: Screening/Previous Regimen Phase, IGSC 20% Treatment
      Stage 1 (13 IGSC 20% weekly doses), and IGSC 20% Treatment Stage 2 (39 IGSC 20% weekly
      doses).

      Previous Regimen Phase: Subjects will be infused with their current ongoing ("previous
      regimen") intravenous immune globulin/subcutaneous immune globulin (IVIG/SCIG) regimen
      (pIV/pSC) in the clinic to obtain 2 trough immunoglobulin G (IgG) levels (obtained prior to
      each pIV/pSC infusion) on each subject's "previous regimen".

      20% IGSC Treatment Stage 1: The first dose of IGSC 20% will be administered at the clinical
      site immediately after Baseline assessments are complete (SC#1). All subjects will receive 13
      IGSC 20% infusions at weekly intervals. IgG trough blood levels will be measured at all
      (except SC#3) study visits All other doses of IGSC 20% may be infused at home (once properly
      trained) or in the clinic. The Treatment Stage 1 dose will continue into IGSC 20% Treatment
      Stage 2.

      IGSC 20% Treatment Stage 2: The IGSC 20% dose (mg/kg) will remain constant with no dose
      adjustment permitted in this phase, unless it is absolutely medically necessary. While all
      subjects will have a SC#17 clinic visit and standard assessments, serial pharmacokinetics
      (PK) sampling will only be performed in a subset of adult subjects:
    

Intervention name(s): IGSC 20%
Intervention type(s): Biological
Intervention description(s): Weekly administration of IGSC 20% via intravenous infusion
Other intervention name(s): Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography
Intervention MeSH terms: Immunoglobulins; gamma-Globulins; Immunoglobulins, Intravenous; Rho(D) Immune Globulin
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment