CLINICAL PHASE III STUDY TO MONITOR THE SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES, INCLUDING (BUT NOT LIMITED TO) THOSE WHO HAVE COMPLETED THE SCGAM-01 TRIAL
Condition: Primary Immunodeficiency
MeSH terms: Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes
Intervention: Octanorm 16.5%
Intervention description: Human normal immunoglobulin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 27.0
Sex: All
Minimum age: 2 Years
Maximum age: 75 Years
Healthy volunteers: No

Summary:

      Summary for SCGAM-03: Clinical phase III study to monitor the safety, tolerability and
      efficacy of subcutaneous human immunoglobulin (Octanorm) in patients with primary
      immunodeficiency diseases who have completed the SCGAM-01 trial.

      Summary for SCGAM-03 in Canada: Clinical phase III study to monitor the safety, tolerability
      and efficacy of subcutaneous human immunoglobulin (octanorm) in patients with primary
      immunodeficiency diseases, including (but not limited to) those who have completed the
      SCGAM-01 trial
    

Eligibility criteria:

        Inclusion Criteria for SCGAM-03:

          1. Completion of the main study SCGAM-01, with good tolerance of Octanorm (as determined
             by the investigator).

          2. For adult patients: freely given written informed consent. For patients below the
             legal age of majority: freely given written informed consent from parents/legal
             guardians and written informed assent from the child/adolescent in accordance with
             local requirements.

          3. For female patients of child-bearing potential, a negative result in a urine pregnancy
             test conducted at the Screening visit.

          4. Willingness to comply with all aspects of the protocol, including blood sampling, for
             the duration of the study.

        Inclusion Criteria for SCGAM-03 in Canada:

        Either:

        SCGAM-01 patients (United States, Canada):

        1. Completion of the main study SCGAM-01, with good tolerance of octanorm (as determined by
        the investigator).

        Or:

        De novo patients (Canada only):

          1. C-a Age of ≥18 years and ≤75 years.

        1C-b Confirmed diagnosis of PI as defined by ESID and PAGID and requiring immunoglobulin
        replacement therapy due to hypogammaglobulinaemia or agammaglobulinaemia. The exact type of
        PI should be recorded.

          1. C-c Availability of the IgG trough levels of 2 previous SCIG infusions before
             enrolment, and maintenance of ≥5.0 g/L in the trough levels of these 2 previous
             infusions.

        And:

        2. For adult patients: freely given written informed consent. For patients below the legal
        age of majority: freely given written informed consent from parents/legal guardians and
        written informed assent from the child/adolescent in accordance with local requirements.

        3. For female patients of child-bearing potential, a negative result in a urine pregnancy
        test conducted at the Screening Visit.

        4. Willingness to comply with all aspects of the protocol, including blood sampling, for
        the duration of the study.

        Exclusion Criteria for SCGAM-03:

          1. Subject being without any IgG treatment for period greater than approximately 5 weeks
             between the last infusion of Octanorm in the SCGAM-01 study and the first infusion of
             Octanorm in the SCGAM-03 study.

          2. Exposure to blood or any blood product or derivative, other than IgG used for regular
             PID treatment, within the 3 months before the first infusion in this study.

          3. Planned pregnancy during the course of the study.

        Exclusion Criteria for SCGAM-03 in Canada:

          -  Either:

        SCGAM-01 patients (United States, Canada):

        1 Subject being without any IgG treatment for period greater than 5 weeks between the last
        infusion of octanorm in the SCGAM-01 study and the first infusion of octanorm in the
        SCGAM-03 study.

        Or:

        De novo patients (Canada only):

        1C-a Acute infection requiring intravenous antibiotic treatment within 2 weeks prior to and
        during the screening period.

        1C-b Known history of adverse reactions to IgA in other products.

        1C-c Patients with body mass index >40 kg/m2.

        1C-d Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived
        products, or any component of the investigational product (such as Polysorbate 80).

        1C-e Requirement of any routine premedication for IgG administration.

        1C-f History of malignancies of lymphoid cells and immunodeficiency with lymphoma.

        1C-g Severe liver function impairment (ALAT 3 times above upper limit of normal).

        1C-h Known protein-losing enteropathies or proteinuria.

        1C-i Presence of renal function impairment (creatinine >120 μM/L or creatinine >1.35
        mg/dL), or predisposition for acute renal failure (e.g., any degree of pre-existing renal
        insufficiency or routine treatment with known nephritic drugs).

        1C-j Treatment with oral or parenteral steroids for ≥30 days or when given intermittently
        or as bolus at daily doses ≥0.15 mg/kg.

        1C-k Treatment with immunosuppressive or immunomodulatory drugs.

        1C-l Live viral vaccination (such as measles, rubella, mumps and varicella) within the last
        2 months prior to first infusion of octanorm.

        And:

        2. Exposure to blood or any blood product or plasma derivatives, other than SCIG used for
        regular PID treatment, within the 3 months before the first infusion of octanorm in this
        study.

        3. Pregnant or nursing women or planned pregnancy during the course of the study.

        4. Treatment with any investigational medicinal product (other than that of SCGAM-01)
        within 3 months prior to first infusion of octanorm.

        5. Presence of any condition, that is likely to interfere with the evaluation of study
        medication or satisfactory conduct of the trial.

        6. Known or suspected to abuse alcohol, drugs, psychotropic agents or other chemicals
        within the past 12 months prior to first infusion of octanorm.

        7. Known or suspected HIV, HCV, or HBV infection.