Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in PID Patients
Condition: Primary Immunodeficiency Disease
MeSH terms: Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes
Intervention: Kedrion IVIG 10%
Intervention description: Kedrion intravenous immunoglobulin (IVIg) 10%
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 47.0
Sex: All
Minimum age: 2 Years
Maximum age: 70 Years
Healthy volunteers: No
Keywords: Immunoglobulin; Kedrion IVIg10%; IgG antibodies; Agammaglobulinemia; Hypogammaglobulinemia

Summary:

      The purpose of this study was to assess efficacy and safety of Kedrion Immunoglobulin 10%
      (KIg10) in participants with Primary Immunodeficiency (PID).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Written informed consent/assent obtained from participant/participant's parent(s) or
             legally acceptable representative indicating that they understand the purpose of and
             procedures required for the study and are willing to participate in it.

          -  Confirmed clinical diagnosis of a PID, requiring treatment with IVIg and have
             documented agammaglobulinemia (defined as the total absence of one or more classes of
             antibodies) or hypogammaglobulinemia (defined as low levels of one or more classes
             [that is at least 2 standard deviations under the mean level per age])

          -  Male or female, ages 2 to 70 years at screening

          -  Received 200 to 800 mg/kg of a commercially available IVIg therapy in the range of 21-
             or 28-day intervals (±3 days or ±4 days, respectively) for at least 3 infusion cycles
             prior to screening

          -  At least 2 documented IgG trough levels while receiving an IVIg, of greater than or
             equals to (>=) 6 gram per liter (g/L) obtained at 2 infusion cycles within 12 months
             (1 must be within 6 months) prior to Informed Consent Form (ICF) signature

          -  Participant is willing to comply all requirements of the protocol.

          -  Females of child-bearing potential with a negative urine pregnancy test and who agree
             to employ adequate birth control measures during the study

          -  Authorization to access personal health information

          -  Participant previously participating in a clinical trial with another experimental
             IVIg may be enrolled if they have received stable commercially available IVIg therapy
             for at least 3 infusion cycles (21 or 28 days) prior to screening

          -  Participant currently on treatment with any subcutaneous immunoglobulin (SCIG) can be
             enrolled if they are switched to stable commercially available IVIg therapy for at
             least 3 infusion cycles (21 or 28 days) prior to screening

        Exclusion Criteria:

          -  Newly diagnosed PID and and naïve to IgG replacement therapy

          -  Dysgammaglobulinemia (defined as a deficiency in one or more classes of antibodies,
             but not severe enough to require substitutive therapy) or isolated IgG subclass
             deficiency or profound primary T-cell deficiency (defined as the absence or severe
             reduction of T lymphocytes [CD3+ <300 cells per cubic millimeter (cell/mm3)] and an
             absent or particularly low proliferative response [10% of the lower normal range] to
             phytohaemagglutinin P [PHA])

          -  Has history of severe or serious reactions or hypersensitivity to IVIg or other
             injectable forms of IgG

          -  Has history of thrombotic events (including deep vein thrombosis, cerebrovascular
             accident, pulmonary embolism, transient ischemic attacks, or myocardial infarction),
             as defined by at least 1 event in participant's lifetime

          -  Has IgA deficiency with documented antibodies to IgA

          -  Have received blood products that have not undergone viral inactivation measures
             within 12 months prior to Informed Consent Form (ICF) signature

          -  Has significant protein losing enteropathy, nephrotic syndrome, or lymphangiectasia

          -  Has an acute infection as documented by culture or diagnostic imaging and/or a body
             temperature >= 38 degree Celsius (°C) (>=100.4 degree Fahrenheit (°F) within 7 days
             prior to screening

          -  Has acquired immunodeficiency syndrome (AIDS) and/or hepatitis B/C active disease at
             ICF signature

          -  Has levels of Alanine aminotransferase (ALT) or aspartate aminotransferase (AST), 2.5
             times of the upper limit of normal for the laboratory designated for the study

          -  Using an implanted venous access device

          -  Has profound anemia (haemoglobin less than10 gram per deciliter [g/dl]) or persistent
             severe neutropenia (<= 1000 neutrophils per millimeter cube (mm^3) or lymphopenia of
             less than 500 cells per microliter

          -  Have severe chronic condition such as renal failure (creatinine concentration > 2.0
             times the upper limit of normal) with proteinuria, congestive heart failure (New York
             Heart Association III/IV), cardiomyopathy, cardiac arrhythmia associated with
             thromboembolic events (e.g., atrial fibrillation), unstable or advanced ischemic heart
             disease, hyper viscosity, or any other condition that the Investigator believes is
             likely to interfere with evaluation of the study drug or with satisfactory conduct of
             the trial. Normal values for serum creatinine are the following: a) Female (18+
             years): 45 - 84 micromole per liter (mcmol/L) or 0.51 - 0.95 milligrams per deciliter
             (mg/dl); b) Male (18+ years): 59 - 103 mcmol/L or 0.67 - 1.17 mg/dl

          -  Has history of a malignant disease other than properly treated carcinoma in situ of
             the cervix or basal cell or squamous cell carcinoma of the skin within 24 months prior
             to ICF signature

          -  Has history of pharmacoresistant epilepsy or multiple episodes of migraine (defined as
             at least 1 episode within 6 months of ICF signature) not completely controlled by
             medication

          -  Participants must not be receiving steroids (oral or parenteral daily dose of >= 0.15
             milligram per kilogram per day (mg/kg/day) of prednisone or equivalent) or other
             immunosuppressive drugs or chemotherapy

          -  Females who are pregnant, breast feeding or planning a pregnancy during the course of
             the study. Women who become pregnant during the study will be withdrawn from the study

          -  Has participated in another clinical study within 3 weeks prior to study ICF signature

          -  Has history of drug or alcohol abuse within the 6 months before screening

          -  Has Employed or a direct relative of an employee of the CRO, the study site, or the
             Sponsor

          -  Previously treated under this protocol

          -  Unable to provide informed consent or provide informed consent by a legally authorized
             representative