A Study of NewGam, Human Immunoglobulin 10%, in Patients With Primary Immunodeficiency Diseases
Condition: Primary Immunodeficiency Diseases
MeSH terms: Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes
Intervention: NewGam
Intervention description: The dose of NewGam, solvent/detergent treated human normal immunoglobulin 10%, remained the same throughout the study, as long as minimum trough levels of serum immunoglobulin G (IgG) was above 5 g/L. If serum IgG trough levels dropped to 5 g/L or less, the dose was to be adjusted at the investigator's discretion. NewGam was supplied as a solution for infusion.
Intervention MeSH terms: Immunoglobulins
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 51.0
Sex: All
Minimum age: 2 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: PID

Summary:

      The purpose of this study was to determine the efficacy of NewGam in preventing serious
      bacterial infections and to determine the pharmacokinetic profile of NewGam. The safety of
      NewGam and its effect on quality of life were also evaluated.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Age of ≥ 2 years and ≤ 75 years.

          -  Confirmed diagnosis of common variable immunodeficiency (CVID) or X-linked
             agammaglobulinemia (XLA).

          -  Previously treated with a commercial immune globulin intravenous (human) every 21-28
             days for at least 6 infusion intervals at a constant dose between 200 and 800 mg/kg
             body weight.

        Exclusion Criteria:

          -  Acute infection requiring intravenous antibiotic treatment within 2 weeks prior to and
             during the screening period.

          -  Exposure to blood or any blood product or derivative, other than commercially
             available intravenous immunoglobulin (IVIG), within the past 3 months prior to
             enrollment.

          -  Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived
             products, or any component of the investigational product.

          -  Requirement of any routine pre-medication for IVIG infusion.

          -  Severe liver function impairment (alanine aminotransferase [ALAT] 3x > upper limit of
             normal).

          -  Presence of renal function impairment (creatinine > 120 μmol/L), or predisposition for
             acute renal failure (eg, any degree of pre-existing renal insufficiency or routine
             treatment with known nephritic drugs).

          -  History of autoimmune hemolytic anemia.

          -  History of diabetes mellitus.

          -  Congestive heart failure New York Heart Association (NYHA) class III or IV.

          -  Non-controlled arterial hypertension (systolic blood pressure > 160 mmHg or diastolic
             blood pressure > 90 mmHg).

          -  History of deep vein thrombosis or thrombotic complications of IVIG therapy.

          -  A positive result at screening on any of the following viral markers: human
             immunodeficiency virus (HIV), hepatitis C virus (HCV), hepatitis B virus (HBV).

          -  Treatment with steroids (oral or parenteral, long-term, ie, 30 days or more, not
             intermittent or burst, daily, ≥ 0.15 mg of prednisone or equivalent/kg/day),
             immunosuppressive or immunomodulatory drugs.

          -  Planned vaccination during the study period.

          -  Treatment with any investigational agent within 3 months prior to enrollment.

          -  Known or suspected to abuse alcohol, drugs, psychotropic agents or other chemicals
             within the past 12 months prior to enrollment.

          -  Pregnant or nursing women.