Efficacy, Pharmacokinetics, Safety, and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency
Condition: Primary Immunodeficiency
MeSH terms: Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes
Intervention: IGSC 20%
Intervention description: Weekly administration of IGSC 20% via intravenous infusion
Intervention MeSH terms: Immunoglobulins; gamma-Globulins; Immunoglobulins, Intravenous; Rho(D) Immune Globulin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 61.0
Sex: All
Minimum age: 2 Years
Maximum age: 75 Years
Healthy volunteers: No

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.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Pre-existing diagnosis of PI with features of hypogammaglobulinemia requiring IgG
             replacement therapy

          -  No serious bacterial infection within the 3 months prior to Screening and has no
             serious bacterial infections (SBIs) up to the time of the Baseline Visit

          -  Currently on IgG replacement therapy (stable regimen [dose and dosing interval] via IV
             or SC infusion) for ≥ 3 consecutive months at a dosage of at least 200 mg/kg per
             infusion

          -  Documented (within previous 3 months) of an IgG trough level of ≥ 500 mg/dL on current
             IgG replacement therapy regimen

          -  Screening/pre-Baseline trough IgG levels must be ≥ 500 mg/dL.

        Exclusion Criteria:

          -  Known serious adverse reaction to immunoglobulin or any severe anaphylactic reaction
             to blood or any blood-derived product

          -  History of blistering skin disease, clinically significant thrombocytopenia, bleeding
             disorder, diffuse rash, recurrent skin infections, or other disorders where SC therapy
             would be contraindicated during the study

          -  Isolated IgG subclass deficiency, isolated specific antibody deficiency disorder, or
             transient hypogammaglobulinemia of infancy

          -  Nephrotic syndrome, and/or a history of acute renal failure, and/or severe renal
             impairment, and/or is on dialysis

          -  Known previous infection with or clinical signs and symptoms consistent with current
             hepatitis B virus or hepatitis C virus infection

          -  History of (year prior to Screening or 2 episodes in lifetime) or current diagnosis of
             deep venous thrombosis or thromboembolism (e.g., myocardial infarction,
             cerebrovascular accident, or transient ischemic attack)

          -  Acquired medical condition known to cause secondary immune deficiency, such as chronic
             lymphocytic leukemia, lymphoma, multiple myeloma, chronic or recurrent neutropenia
             (absolute neutrophil count less than 1000/μL [1.0 x 10^9/L]), or human
             immunodeficiency virus infection/acquired immune deficiency syndrome

          -  HIV positive by nucleic acid amplification technology based on a Screening blood
             sample

          -  Uncontrolled arterial hypertension (adult subjects: systolic blood pressure > 160 mmHg
             and/or diastolic blood pressure > 100 mmHg)

          -  Receiving any of the following medications: (a) immunosuppressants including
             chemotherapeutic agents, (b) immunomodulators, (c) long-term systemic corticosteroids
             defined as daily dose > 1 mg of prednisone equivalent/kg/day for > 30 days Note:
             Intermittent courses of corticosteroids of not more than 10 days would not exclude a
             subject. Inhaled or topical corticosteroids are allowed.