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

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03907241

Enrollment

Registered

2019-04-08

Start date

2016-03-01

Completion date

2019-09-05

Last updated

2020-10-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Primary Immunodeficiency

Brief 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

Interventions

DRUGOctanorm 16.5%

Human normal immunoglobulin

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

2 Years to 75 Years

Healthy volunteers

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.

Design outcomes

Primary

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

Secondary

Measure	Time frame	Description
Number of Participants With Serious Bacterial Infections (SBIs).	From study start to end, up to 3.5 years	Number of participants with serious bacterial infections
SF-36 Health Survey.	From study start to end, up to 3.5 years	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
CHQ-PF50 (Child Health Questionnaire-Parent Form)	From study start to end, up to 3.5 years	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.
Measurement of Trough Total IgG Levels	From study start to end, up to 3.5 years	Measurement of trough total IgG levels from baseline to end of study

Countries

Canada, United States

Participant flow

Participants by arm

Arm	Count
Age >=2 to <6 Octanorm 16.5%	2
Age >=6 to <12 Octanorm 16.5%	4
Age >=12 to <17 Octanorm 16.5%	4
Age >=17 to <=75 Octanorm 16.5%	17
Total	27

Baseline characteristics

Characteristic	Age >=2 to <6	Age >=6 to <12	Age >=12 to <17	Age >=17 to <=75	Total
ABO Rhesus Blood Type AB Blood Type	0 Participants	0 Participants	1 Participants	1 Participants	2 Participants
ABO Rhesus Blood Type A Blood Type	1 Participants	2 Participants	2 Participants	8 Participants	13 Participants
ABO Rhesus Blood Type O Blood Type	1 Participants	2 Participants	1 Participants	8 Participants	12 Participants
Age, Continuous	6.50 years	9.00 years	14.25 years	56.12 years	39.26 years
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants	4 Participants	4 Participants	17 Participants	27 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Asian	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Black or African American	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) More than one race	0 Participants	0 Participants	0 Participants	1 Participants	1 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants	0 Participants	0 Participants	1 Participants	1 Participants
Race (NIH/OMB) White	2 Participants	4 Participants	4 Participants	15 Participants	25 Participants
Sex: Female, Male Female	0 Participants	2 Participants	2 Participants	13 Participants	17 Participants
Sex: Female, Male Male	2 Participants	2 Participants	2 Participants	4 Participants	10 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk	EG002 affected / at risk	EG003 affected / at risk
deaths Total, all-cause mortality	0 / 2	0 / 4	0 / 4	0 / 17
other Total, other adverse events	2 / 2	4 / 4	4 / 4	14 / 17
serious Total, serious adverse events	0 / 2	1 / 4	2 / 4	4 / 17