Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency
Condition: Primary Immunodeficiency
MeSH terms: Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes
Intervention: GAMUNEX-C; GAMUNEX-C
Intervention description: GAMUNEX-C Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified, Intravenous Administration: 200-600 mg/kg per intravenous infusion every 3-4 weeks; GAMUNEX-C Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified, Subcutaneous Administration: weekly subcutaneous infusion at a mg/kg dose based on subject's intravenous dose and dosing interval x 1.37 conversion factor
Intervention MeSH terms: Immunoglobulins, Intravenous; gamma-Globulins; Rho(D) Immune Globulin; Immunoglobulin G
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Crossover Assignment
Masking: None (Open Label)
Enrollment: 12.0
Sex: All
Minimum age: 2 Years
Maximum age: 16 Years
Healthy volunteers: No

Summary:

      The purpose of this open-label study is to evaluate the pharmacokinetics, safety, and
      tolerability of subcutaneously (SC; under the skin) administered GAMUNEX-C compared to
      intravenously (IV; through the vein) administered GAMUNEX-C in subjects 2-16 years of age
      with Primary Immunodeficiency.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Aged 2-16 years old, inclusive.

          -  Documented and confirmed pre-existing diagnosis of PI with features of
             hypogammaglobulinemia requiring immunoglobulin replacement.

          -  Currently on IgG replacement therapy with a serum IgG trough concentration of ≥ 500
             mg/dL at the Screening Visit.

          -  Adequate normal skin to allow for SC infusions.

          -  Signs an assent form, if applicable (per Institutional Review Board [IRB]
             requirements). Parent or legal guardian must sign an informed consent form.

          -  Females of childbearing potential must have a negative urine pregnancy test result and
             must practice an effective form of contraception (which may include abstinence).

        Exclusion Criteria:

          -  History of anaphylaxis or severe systemic response to an immunoglobulin or blood
             product.

          -  History of blistering skin disease, clinically significant thrombocytopenia, bleeding
             disorder, recurrent skin infections or other disorders where subcutaneous therapy
             could be contraindicated.

          -  Has a specific antibody deficiency disorder, IgG subclass deficiency, or transient
             hypogammaglobulinemia of infancy.

          -  History of severe adverse reaction to parenteral products containing immunoglobulin A
             (IgA).

          -  Significant proteinuria and/or has a history of acute renal failure and/or severe
             renal impairment (serum creatinine more than 2.5 times the upper limit of normal [ULN]
             for age and gender) and/or is on dialysis.

          -  Known substance or prescription drug abuse in the past 12 months.

          -  Acquired medical condition that is known to cause secondary immune deficiency.

          -  Receiving any of the following medications: systemic corticosteroids (long term daily,
             >1 mg of prednisone equivalent/kg/day for >30 days) (intermittent courses would not
             exclude subject); immunosuppressants (i.e., antimetabolites and systemic calcineurin
             inhibitors; NOTE: inhaled steroids are allowed); or immunomodulators.

          -  Non-controlled arterial hypertension at a level of ≥ the 90th percentile blood
             pressure (either systolic or diastolic) for age and height (based on
             http://www.nhlbi.nih.gov/guidelines/hypertension/child_tbl.pdf ).

          -  History or current diagnosis of thrombotic episodes; venous thrombus that occurred in
             association with a medical device > 2 years prior to screening are allowed.

          -  Currently receiving anti-coagulation therapy.

          -  History of Kawasaki disease.

          -  Participated in another clinical trial involving exposure to an investigational
             product or device within 30 days prior to screening (imaging studies without
             investigative treatments are permitted) or has received any investigational blood
             product within the previous 3 months.

          -  Unable or unwilling to comply with any aspect of the protocol, including blood
             sampling and completion of the Infusion Site Reactions pages in the SC Infusion Diary.

          -  In the opinion of the Investigator the subject may have compliance problems with the
             protocol and the procedures of the protocol.

          -  Pregnant or lactating.

          -  Clinical evidence of any significant acute or chronic disease that, in the opinion of
             the Investigator, may interfere with successful completion of the study.