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
- Conditions
- Primary Immunodeficiency
- Interventions
- Drug: Octanorm 16.5%
- Registration Number
- NCT03907241
- Lead Sponsor
- Octapharma
- 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
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 27
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Octanorm 16.5% Octanorm 16.5% octanorm 16.5%, human normal immunoglobulin for subcutaneous (SC) administration.
- Primary Outcome Measures
Name Time Method Creatinine From study start to end, up to 3.5 years Changes in creatinine from baseline to end of study
Occurrence of Temporally Associated TEAEs From study start to end, up to 3.5 years 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
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 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
Haemoglobin From study start to end, up to 3.5 years Changes in haemoglobin from baseline to end of study
Sodium From study start to end, up to 3.5 years Changes in sodium levels 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
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
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
Occurrence of All Treatment-emergent Adverse Events (TEAEs) From study start to end, up to 3.5 years Number of TEAEs
Respiratory Rate From study start to end, up to 3.5 years ASAT From study start to end, up to 3.5 years Changes in ASAT (aspartate aminotransferase) 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
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
Blood Pressure From study start to end, up to 3.5 years Systolic and diastolic.
Potassium From study start to end, up to 3.5 years Changes in potassium levels from baseline to 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
Local Injection-site Reactions From study start to end, up to 3.5 years Body Temperature From study start to end, up to 3.5 years LDH From study start to end, up to 3.5 years Changes in LDH (lactate dehydrogenase) 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
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 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
- Secondary Outcome Measures
Name Time Method 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
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
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 stateCHQ-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.
Trial Locations
- Locations (1)
Octapharma Research Site
🇨🇦Edmonton, Alberta, Canada