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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03907241
• Date of registration: 07/12/2018
• Prospective Registration: No
• Primary sponsor: Octapharma
• Public title: 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
• Scientific title: 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
• Date of first enrolment: March 1, 2016
• Target sample size: 27
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03907241
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Canada, United States

Key inclusion & exclusion 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.



Age minimum: 2 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Primary Immunodeficiency

Intervention(s)

Drug: Octanorm 16.5%

Primary Outcome(s)

ASAT [Time Frame: From study start to end, up to 3.5 years]

Number of Participants With a Change in Urine Glucose [Time Frame: From study start to end, up to 3.5 years]

Blood Pressure [Time Frame: From study start to end, up to 3.5 years]

Haematocrit [Time Frame: From study start to end, up to 3.5 years]

Number of Temporally Associated TEAEs by Infusion Rate [Time Frame: From study start to end, up to 3.5 years]

Occurrence of Temporally Associated TEAEs [Time Frame: From study start to end, up to 3.5 years]

Potassium [Time Frame: From study start to end, up to 3.5 years]

Respiratory Rate [Time Frame: From study start to end, up to 3.5 years]

Urine pH [Time Frame: From study start to end, up to 3.5 years]

Blood Glucose [Time Frame: From study start to end, up to 3.5 years]

Complete White Blood Cell Count [Time Frame: From study start to end, up to 3.5 years]

Haemoglobin [Time Frame: From study start to end, up to 3.5 years]

Body Temperature [Time Frame: From study start to end, up to 3.5 years]

LDH [Time Frame: From study start to end, up to 3.5 years]

ALAT [Time Frame: From study start to end, up to 3.5 years]

Blood Urea Nitrogen [Time Frame: From study start to end, up to 3.5 years]

Sodium [Time Frame: From study start to end, up to 3.5 years]

Total Bilirubin [Time Frame: From study start to end, up to 3.5 years]

Number of Participants With a Change in Urine Hemoglobin [Time Frame: From study start to end, up to 3.5 years]

Number of Participants With a Change in Urine Ketones [Time Frame: From study start to end, up to 3.5 years]

Number of Participants With a Change in Urine Leukocytes [Time Frame: From study start to end, up to 3.5 years]

Complete Red Blood Cell Count [Time Frame: From study start to end, up to 3.5 years]

Creatinine [Time Frame: From study start to end, up to 3.5 years]

Local Injection-site Reactions [Time Frame: From study start to end, up to 3.5 years]

Occurrence of All Treatment-emergent Adverse Events (TEAEs) [Time Frame: From study start to end, up to 3.5 years]

Secondary Outcome(s)

Number of Participants With Serious Bacterial Infections (SBIs). [Time Frame: From study start to end, up to 3.5 years]

Measurement of Trough Total IgG Levels [Time Frame: From study start to end, up to 3.5 years]

SF-36 Health Survey. [Time Frame: From study start to end, up to 3.5 years]

CHQ-PF50 (Child Health Questionnaire-Parent Form) [Time Frame: From study start to end, up to 3.5 years]

Secondary ID(s)

SCGAM-03 -

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 22/09/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03907241