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

Study Overview

• Status: Completed
• Conditions: Primary Immunodeficiency
• Intervention / Treatment: Drug: Octanorm 16.5%

• Study Type: Interventional
• Enrollment (Actual): 27
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G2V2
      • Octapharma Research Site
• United States
  • California
    • Irvine, California, United States, 92697
      • Octapharma Research Site
    • San Diego, California, United States, 92123
      • Octapharma Research Site
  • Colorado
    • Centennial, Colorado, United States, 80112
      • Octapharma Research Site
  • Nebraska
    • Papillion, Nebraska, United States, 68046
      • Octapharma Research Site
  • Ohio
    • Toledo, Ohio, United States, 43617
      • Octapharma Research Site
  • Texas
    • Frisco, Texas, United States, 75034
      • Octapharma Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Octanorm 16.5%; octanorm 16.5%, human normal immunoglobulin for subcutaneous (SC) administration.	Drug: Octanorm 16.5%; Human normal immunoglobulin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of All Treatment-emergent Adverse Events (TEAEs)	Number of TEAEs	From study start to end, up to 3.5 years
Occurrence of Temporally Associated TEAEs		From study start to end, up to 3.5 years
Number of Temporally Associated TEAEs by Infusion Rate	Number of temporally associated TEAEs by infusion rate. Only includes systemic TEAEs without infections and without infusion site reactions	From study start to end, up to 3.5 years
Local Injection-site Reactions		From study start to end, up to 3.5 years
Blood Pressure	Systolic and diastolic.	From study start to end, up to 3.5 years
Body Temperature		From study start to end, up to 3.5 years
Respiratory Rate		From study start to end, up to 3.5 years
Sodium	Changes in sodium levels from baseline to end of study	From study start to end, up to 3.5 years
Potassium	Changes in potassium levels from baseline to end of study	From study start to end, up to 3.5 years
Blood Glucose	Changes in blood glucose from baseline to end of study	From study start to end, up to 3.5 years
ALAT	Changes in ALAT (alanine transaminase) from baseline to end of study	From study start to end, up to 3.5 years
ASAT	Changes in ASAT (aspartate aminotransferase) from baseline to end of study	From study start to end, up to 3.5 years
LDH	Changes in LDH (lactate dehydrogenase) from baseline to end of study	From study start to end, up to 3.5 years
Total Bilirubin	Changes in total bilirubin from baseline to end of study	From study start to end, up to 3.5 years
Blood Urea Nitrogen	Changes in blood urea nitrogen from baseline to end of study	From study start to end, up to 3.5 years
Creatinine	Changes in creatinine from baseline to end of study	From study start to end, up to 3.5 years
Urine pH	Changes in urine pH from baseline to end of study	From study start to end, up to 3.5 years
Number of Participants With a Change in Urine Glucose	Number of Participants with a Change in Urine Glucose	From study start to end, up to 3.5 years
Number of Participants With a Change in Urine Ketones	Number of Participants With a Change in Urine Ketones at baseline and end of study	From study start to end, up to 3.5 years
Number of Participants With a Change in Urine Leukocytes	Number of participants with a change in urine leukocytes at baseline and end of study	From study start to end, up to 3.5 years
Number of Participants With a Change in Urine Hemoglobin	Number of participants with a change in urine hemoglobin at baseline and end of study	From study start to end, up to 3.5 years
Complete Red Blood Cell Count	Changes in complete red blood cell count from baseline to end of study	From study start to end, up to 3.5 years
Haematocrit	Changes in haematocrit from baseline to end of study	From study start to end, up to 3.5 years
Haemoglobin	Changes in haemoglobin from baseline to end of study	From study start to end, up to 3.5 years
Complete White Blood Cell Count	Changes in complete white blood cell count from baseline to end of study	From study start to end, up to 3.5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Measurement of Trough Total IgG Levels	Measurement of trough total IgG levels from baseline to end of study	From study start to end, up to 3.5 years
Number of Participants With Serious Bacterial Infections (SBIs).	Number of participants with serious bacterial infections	From study start to end, up to 3.5 years
SF-36 Health Survey.	Quality of Life for patients >= age 14 assessed using the Short Form 36 Health survey. Likert like scale. The responses given by patients were combined to create 8 SF-36 scores: physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, mental health 36 questions that fall into 4 Sub scale scoring ranges: Score 1-5: Where 1 is more favorable than 5 Score 1-3: Where 3 is more favorable than 1 Score 1-5: Where 5 is more favorable than 1 Score 1-6: Where 1 is more favorable than 6 The raw subscale scores are converted into a scale score between 0 to 100 using the Quality Metric Health Outcomes™ Scoring Software 2.0 Scale Title of final scales is: Physical and Mental Health Component Summary Scores Range: Lowest = 0 and highest = 100 where a high score equates to a more favorable health state	From study start to end, up to 3.5 years
CHQ-PF50 (Child Health Questionnaire-Parent Form)	Quality of Life for patients ages <14 years assessed using the CHQ-PF50. Measured values represent change in score from baseline to end of study. Two summary scores were derived: physical and psychosocial. In accord with the scoring manual, computed scores were transformed giving each scale a possible range from 0 to 100, with the exception of change in health, with a possible range from 1 to 5. For all CHQ-PF50 scales, higher scores indicated more positive functioning or better health status.	From study start to end, up to 3.5 years

Collaborators and Investigators

• Sponsor: Octapharma

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2016
• Primary Completion (Actual): September 5, 2019
• Study Completion (Actual): September 5, 2019

Study Registration Dates

• First Submitted: December 7, 2018
• First Submitted That Met QC Criteria: April 5, 2019
• First Posted (Actual): April 8, 2019

Study Record Updates

• Last Update Posted (Actual): October 27, 2020
• Last Update Submitted That Met QC Criteria: October 2, 2020
• Last Verified: October 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Genetic Diseases, Inborn; Immunologic Deficiency Syndromes; Primary Immunodeficiency Diseases
• Other Study ID Numbers: SCGAM-03 -

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No