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

Title 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 Title 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

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

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.