Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)

Inclusion Criteria

To be eligible to participate in this study, the subjects must meet the following criteria:

• Signed a written informed consent or a specific assent form for minors.
• Have a diagnosis of primary immunodeficiency disease.
• Be ≥ 2 years and ≤ 75 years.
• Have body weight ≥ 12 kg at screening.
• 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.
• 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:

• Have a known hypersensitivity to immunoglobulin or any excipient in RI-002.
• Have a history of a severe anaphylactic or anaphylactoid reaction to blood or any blood-derived product.
• Have a specific Immunoglobulin A (IgA) deficiency, history of allergic reaction to products containing IgA or has demonstrable antibodies to IgA.
• Have uncompensated hemodynamically significant congenital or other heart disease.
• Have a medical condition that is known to cause secondary immune deficiency.
• Have a significant T-cell deficiency or deficiency of granulocyte number or function.
• Have significant renal impairment or have a history of acute renal failure.
• Have abnormal liver function.
• Be receiving chronic anti-coagulation therapy.
• Have a history of deep vein thrombosis (DVT), thrombotic or thrombo-embolic event, or are at increased risk for thrombotic events.
• 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))
• Administration of a hyperimmune or specialty high titer immunoglobulin product.
• Have uncontrollable arterial hypertension.
• Have a history of hemolysis or positive Coombs test while undergoing treatment with IGIV therapy.
• Be morbidly obese as indicated by a Body Mass Index (BMI) ≥ 40
• Have received any blood product (other than Immunoglobulin G) within 3 months prior to screening.
• Have received any Respiratory Syncytial Virus (RSV) specific products, including palivizumab (Synagis®) within 3 months prior to screening.
• Have abused alcohol, opiates, psychotropic agents, or other chemicals or drugs within the past 12 months.
• Have any condition or abnormal laboratory assessment judged by the investigator to preclude participation in the study.
• Are currently pregnant or nursing.
• Have hepatitis A, B, or C.