Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)
Condition: Primary Immune Deficiency Disorder
MeSH terms: Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes
Intervention: RI-002
Intervention description: Immune Globulin Intravenous (IGIV)
Intervention MeSH terms: Immunoglobulins
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 59.0
Sex: All
Minimum age: 2 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: PIDD, PID, humoral immunity, antibody deficiency

Summary:

      This is a Phase III, multicenter, open-label study of RI-002 administered as an intravenous
      infusion of RI-002 (IGIV) every 21 or 28 days in approximately 60 subjects with Primary
      Immunodeficiency Diseases (PIDD).
    

Eligibility criteria:

        Inclusion Criteria:

        To be eligible to participate in this study, the subjects must meet the following criteria:

          1. Signed a written informed consent or a specific assent form for minors.

          2. Have a diagnosis of primary immunodeficiency disease.

          3. Be ≥ 2 years and ≤ 75 years.

          4. Have body weight ≥ 12 kg at screening.

          5. Have been receiving IGIV at a dose that has not been changed by >50% of the mean dose
             on a mg/kg basis for at least 3 months prior to study entry and have maintained a
             trough serum Immunoglobulin G (IgG) level ≥ 500 mg/dL on the previous 2 assessments
             prior to receiving RI 002. The trough level must be at least 300 mg/dL above the
             pre-treatment serum IgG level.

          6. For female subjects, be of non-childbearing potential or have a negative pregnancy
             test prior to study start and be deemed not at risk of becoming pregnant by adherence
             to a reliable contraceptive method for the duration of the study.

        Exclusion Criteria:

        Subjects must be excluded if they meet any of the following criteria:

          1. Have a known hypersensitivity to immunoglobulin or any excipient in RI-002.

          2. Have a history of a severe anaphylactic or anaphylactoid reaction to blood or any
             blood-derived product.

          3. Have a specific Immunoglobulin A (IgA) deficiency, history of allergic reaction to
             products containing IgA or has demonstrable antibodies to IgA.

          4. Have uncompensated hemodynamically significant congenital or other heart disease.

          5. Have a medical condition that is known to cause secondary immune deficiency.

          6. Have a significant T-cell deficiency or deficiency of granulocyte number or function.

          7. Have significant renal impairment or have a history of acute renal failure.

          8. Have abnormal liver function.

          9. Be receiving chronic anti-coagulation therapy.

         10. Have a history of deep vein thrombosis (DVT), thrombotic or thrombo-embolic event, or
             are at increased risk for thrombotic events.

         11. Current daily use of the following medications:

               -  corticosteroids (> 7.5 mg (or equivalent dose on a mg/kg basis) of prednisone
                  equivalent per day for > 30 days)

               -  immunomodulatory drugs

               -  immunosuppressive drugs (excluding topical pimecrolimus (Elidel) and tacrolimus
                  (Protopic))

         12. Administration of a hyperimmune or specialty high titer immunoglobulin product.

         13. Have uncontrollable arterial hypertension.

         14. Have a history of hemolysis or positive Coombs test while undergoing treatment with
             IGIV therapy.

         15. Be morbidly obese as indicated by a Body Mass Index (BMI) ≥ 40

         16. Have received any blood product (other than Immunoglobulin G) within 3 months prior to
             screening.

         17. Have received any Respiratory Syncytial Virus (RSV) specific products, including
             palivizumab (Synagis®) within 3 months prior to screening.

         18. Have abused alcohol, opiates, psychotropic agents, or other chemicals or drugs within
             the past 12 months.

         19. Have any condition or abnormal laboratory assessment judged by the investigator to
             preclude participation in the study.

         20. Are currently pregnant or nursing.

         21. Have hepatitis A, B, or C.