Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects
Condition: Primary Immune Deficiency Disease
MeSH terms: Primary Immunodeficiency Diseases; Deficiency Diseases; Immunologic Deficiency Syndromes
Intervention: Flebogamma 5% DIF
Intervention description: Intravenous Immune Globulin (Human)
Intervention MeSH terms: gamma-Globulins; Immunoglobulins, Intravenous; Rho(D) Immune Globulin
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 24.0
Sex: All
Minimum age: 2 Years
Maximum age: 16 Years
Healthy volunteers: No
Keywords: CVID, XLA, hyper IgM syndrome, Wiskott-Aldrich Syndrome

Summary:

      This is a multi-center, open-label study to assess the efficacy and safety of Flebogamma 5%
      DIF in the pediatric population.
    

Eligibility criteria:

        Inclusion Criteria:

          -  The subject is between 2 and 16 years old, of either sex, belonging to any ethnic
             group, and above a minimum weight of 10 kg (this weight is based on the amount of
             blood required for testing).

          -  The subject has a primary immunodeficiency disease, (e.g., common variable
             immunodeficiency, X-linked and autosomal forms of agammaglobulinemia, hyper-IgM
             syndrome, Wiskott-Aldrich Syndrome).

          -  The subject has been receiving licensed IGIV replacement therapy at a dose that has
             not changed by + 50% of the mean dose on a mg/kg basis for at least 3 months before
             study entry and has maintained a trough level at least 300 mg/dL above baseline serum
             IgG levels.

          -  Trough levels of IgG, dose of IGIV, and treatment intervals for the last 2 consecutive
             routine IGIV treatments must be documented for each subject before the first infusion
             in this study can be administered.

          -  If a subject is an adolescent female (> 12 years of age) who is or becomes sexually
             active, she must have a negative result on a pregnancy test (HCG-based assay).

          -  The subject, if old enough (generally 6 years to 16) has signed an informed Child
             Assent form and the subject's parent or legal guardian has signed an informed consent
             form, both approved by the Institutional Review Board.

        Exclusion Criteria:

          -  Adult patient (> 17 years old).

          -  The subject has a history of any severe anaphylactic reaction to blood or any
             blood-derived product.

          -  The subject is known to be intolerant to any component of the products, such as
             sorbitol (i.e., intolerance to fructose).

          -  The subject has selective IgA deficiency or has demonstrable antibodies to IgA.

          -  The subject is currently receiving, or has received, any investigational agent within
             the prior 3 months.

          -  The subject has been exposed to blood or any blood product or derivative within the
             last 6 months, other than a commercially available IGIV.

          -  The adolescent subject is pregnant or is nursing.

          -  The subject, at screening, has levels greater than 2.5 times the upper limit of normal
             as defined at the central laboratory for pediatric patients of any of the following:

               -  ALT

               -  AST

               -  LDH

          -  The subject has a severe pre-existing renal impairment (defined by serum creatinine
             greater than 2 times the ULN or BUN greater than 2.5 times the ULN for that
             laboratory, or any subject who is on dialysis) or any history of acute renal failure.

          -  The subject has a history of DVT or thrombotic complications of IGIV therapy.

          -  The subject suffers from any acute or chronic medical condition (e.g., renal disease
             or predisposing conditions for renal disease, coronary artery disease, or protein
             losing state) that, in the opinion of the Investigator, may interfere with the conduct
             of the study.

          -  The subject has an acquired medical condition such as lymphocytic leukemia, chronic or
             recurrent neutropenia (ANC less than 1000), or AIDS known to cause secondary immune
             deficiency, or is s/p hematopoetic stem cell transplantation.

          -  The subject is receiving the following medication:

               -  Systemic corticosteroids (long-term, i.e., not intermittent or burst, daily, > 1
                  mg of prednisone equivalent/kg/day). Topical steroids (ie- nasal, inhaled for
                  asthma, and/or skin preparations for eczema) are not exclusionary.

               -  Immunosuppressive drugs

               -  Immunomodulatory drugs

          -  The subject has non-controlled arterial hypertension (systolic blood pressure > 160 mm
             Hg and/or diastolic blood pressure > 100 mm Hg).

          -  The subject has anemia (hemoglobin < 10 g/dL) at screening.

          -  The subject and/or parent/legal guardian of the subject is unlikely to adhere to the
             protocol requirements of the study or is likely to be uncooperative or unable to
             provide from the patient a storage serum sample at the screening visit. (Please note,
             a pre-treatment serum sample to be stored at -94° F (-70º C) for possible future
             testing is absolutely required).