CClinicalTrials.gg
Sign In
CompletedNCT03907241Updated Oct 27, 2020Results posted

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

A Phase 3 interventional study of Octanorm 16.5% in Primary Immunodeficiency, sponsored by Octapharma. Completed at 7 sites in 2 countries. Open to participants aged 2 Years to 75 Years. Per ClinicalTrials.gov, last updated 2020-10-27.

Sponsored by Octapharma · Phase 3, Interventional, and Treatment

From the registry’s dates

  • Registered 2 years 9 months after the study started (first participant enrolled Mar 2016, registered Dec 2018).
Phase
Phase 3
Study type
Interventional
Enrollment
27
Allocation
Not applicable
Ages
2 Years to 75 Years
Sex
All
01

Study 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

02

Conditions studied

  • Primary Immunodeficiency
03

In context

Primary Immunodeficiency Diseases

199 studies on the registry are indexed under Primary Immunodeficiency Diseases; 46 are open to participants now.

This study's enrollment of 27 is below the median of 37 across 121 interventional studies indexed under Primary Immunodeficiency Diseases.

Browse Primary Immunodeficiency Diseases studies →

Lead sponsor

Octapharma is the lead sponsor of 69 studies on the registry; 8 are open to participants now.

Of its 16 completed or terminated interventional studies of FDA-regulated products, 14 (88%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility 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:

  1. 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.
  1. For female patients of child-bearing potential, a negative result in a urine pregnancy test conducted at the Screening Visit.
  1. 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:

  1. 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.
  1. Pregnant or nursing women or planned pregnancy during the course of the study.
  1. Treatment with any investigational medicinal product (other than that of SCGAM-01) within 3 months prior to first infusion of octanorm.
  1. Presence of any condition, that is likely to interfere with the evaluation of study medication or satisfactory conduct of the trial.
  1. Known or suspected to abuse alcohol, drugs, psychotropic agents or other chemicals within the past 12 months prior to first infusion of octanorm.
  1. Known or suspected HIV, HCV, or HBV infection.
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
27 participants (actual)

Study arms

  • Experimental
    Octanorm 16.5%

    octanorm 16.5%, human normal immunoglobulin for subcutaneous (SC) administration.

    Drug: Octanorm 16.5%

Interventions

  • DrugOctanorm 16.5%

    Human normal immunoglobulin

06

What researchers measure

Primary outcomes

  1. Occurrence of All Treatment-emergent Adverse Events (TEAEs)

    Number of TEAEs

    Time frame: From study start to end, up to 3.5 years

  2. Occurrence of Temporally Associated TEAEs

    Time frame: From study start to end, up to 3.5 years

  3. 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

    Time frame: From study start to end, up to 3.5 years

  4. Local Injection-site Reactions

    Time frame: From study start to end, up to 3.5 years

  5. Blood Pressure

    Systolic and diastolic.

    Time frame: From study start to end, up to 3.5 years

  6. Body Temperature

    Time frame: From study start to end, up to 3.5 years

  7. Respiratory Rate

    Time frame: From study start to end, up to 3.5 years

  8. Sodium

    Changes in sodium levels from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  9. Potassium

    Changes in potassium levels from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  10. Blood Glucose

    Changes in blood glucose from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  11. ALAT

    Changes in ALAT (alanine transaminase) from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  12. ASAT

    Changes in ASAT (aspartate aminotransferase) from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  13. LDH

    Changes in LDH (lactate dehydrogenase) from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  14. Total Bilirubin

    Changes in total bilirubin from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  15. Blood Urea Nitrogen

    Changes in blood urea nitrogen from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  16. Creatinine

    Changes in creatinine from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  17. Urine pH

    Changes in urine pH from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  18. Number of Participants With a Change in Urine Glucose

    Number of Participants with a Change in Urine Glucose

    Time frame: From study start to end, up to 3.5 years

  19. Number of Participants With a Change in Urine Ketones

    Number of Participants With a Change in Urine Ketones at baseline and end of study

    Time frame: From study start to end, up to 3.5 years

  20. Number of Participants With a Change in Urine Leukocytes

    Number of participants with a change in urine leukocytes at baseline and end of study

    Time frame: From study start to end, up to 3.5 years

  21. Number of Participants With a Change in Urine Hemoglobin

    Number of participants with a change in urine hemoglobin at baseline and end of study

    Time frame: From study start to end, up to 3.5 years

  22. Complete Red Blood Cell Count

    Changes in complete red blood cell count from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  23. Haematocrit

    Changes in haematocrit from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  24. Haemoglobin

    Changes in haemoglobin from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  25. Complete White Blood Cell Count

    Changes in complete white blood cell count from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

Secondary outcomes

  1. Measurement of Trough Total IgG Levels

    Measurement of trough total IgG levels from baseline to end of study

    Time frame: From study start to end, up to 3.5 years

  2. Number of Participants With Serious Bacterial Infections (SBIs).

    Number of participants with serious bacterial infections

    Time frame: From study start to end, up to 3.5 years

  3. 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

    Time frame: From study start to end, up to 3.5 years

  4. 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.

    Time frame: From study start to end, up to 3.5 years

07

Results

Posted Sep 22, 2020

Participant flow

Participant flow — Overall Study
MilestoneOctanorm 16.5%
Started27
Completed23
Not completed4

Outcome measures

PrimaryOccurrence of All Treatment-emergent Adverse Events (TEAEs)

Number of TEAEs

Time frame:
From study start to end, up to 3.5 years
Reported as:
Number · events
Occurrence of All Treatment-emergent Adverse Events (TEAEs)
eventsAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
TEAEs excluding infections81922155
Infections8122277
PrimaryOccurrence of Temporally Associated TEAEs
Time frame:
From study start to end, up to 3.5 years
Reported as:
Number · events
Occurrence of Temporally Associated TEAEs
eventsAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Temporally Associated TEAEs excluding infections271372
Temporally Associated Infections561145
PrimaryNumber 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

Time frame:
From study start to end, up to 3.5 years
Reported as:
Count of units · infusions
Number of Temporally Associated TEAEs by Infusion Rate
infusionsAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
missing0001
10 to <20ml/h1000
20 to <30ml/hr1100
30 to <40ml/hr0606
40 to <50ml/hr00928
50 to <60ml/hr0043
60 to <70ml/hr0002
70 to <80ml/hr0004
80 to <90ml/hr0006
90 to <100ml/hr00022
PrimaryLocal Injection-site Reactions
Time frame:
From study start to end, up to 3.5 years
Reported as:
Number · infusion site reactions
Local Injection-site Reactions
infusion site reactionsAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Local Injection-site Reactions0435
PrimaryBlood Pressure

Systolic and diastolic.

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · mmHg
Blood Pressure
mmHgAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Systolic Blood Pressure at Screening100.00 ± 2.828105.00 ± 7.789116.60 ± 6.758126.21 ± 15.817
Systolic Blood Pressure End of Study102.50 ± 0.707103.00 ± 12.302114.00 ± 16.104127.24 ± 12.553
Diastolic Blood Pressure at Screening63.00 ± 11.31462.25 ± 6.55168.50 ± 2.38078.79 ± 9.407
Diastolic Blood Pressure at End of Study58.50 ± 13.43563.75 ± 6.70270.25 ± 12.84273.71 ± 8.130
PrimaryBody Temperature
Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · degrees Celsius
Body Temperature
degrees CelsiusAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Temperature at Screening36.85 ± 0.07136.98 ± 0.28736.63 ± 0.35936.56 ± 0.298
Temperature at End of Study36.55 ± 0.35436.58 ± 0.37736.65 ± 0.12936.57 ± 0.355
PrimaryRespiratory Rate
Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · breaths/minute
Respiratory Rate
breaths/minuteAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Respiratory Rate at Screening23.50 ± 6.36419.00 ± 3.83016.00 ± 0.00016.07 ± 2.129
Respiratory Rate at End of Study17.50 ± 2.12115.50 ± 1.00017.75 ± 2.63016.47 ± 2.625
PrimarySodium

Changes in sodium levels from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · mmol/L
Sodium
mmol/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Sodium0.50 ± 0.7070.25 ± 1.2581.75 ± 2.2170.41 ± 2.238
PrimaryPotassium

Changes in potassium levels from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · mmol/L
Potassium
mmol/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Potassium0.35 ± 0.3540.25 ± 0.3000.18 ± 0.340-0.05 ± 0.726
PrimaryBlood Glucose

Changes in blood glucose from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · mmol/L
Blood Glucose
mmol/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Blood Glucose-1.03 ± 1.7663.49 ± 9.0170.22 ± 0.371-0.69 ± 1.672
PrimaryALAT

Changes in ALAT (alanine transaminase) from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · U/L
ALAT
U/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
ALAT-2.50 ± 2.121-5.75 ± 8.016-1.50 ± 16.1350.71 ± 4.074
PrimaryASAT

Changes in ASAT (aspartate aminotransferase) from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · U/L
ASAT
U/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
ASAT-3.00 ± 7.071-4.00 ± 5.354-3.00 ± 11.6330.59 ± 5.885
PrimaryLDH

Changes in LDH (lactate dehydrogenase) from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · U/L
LDH
U/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
LDH23.50 ± 60.104-110.50 ± 131.751-52.50 ± 41.348-8.47 ± 26.779
PrimaryTotal Bilirubin

Changes in total bilirubin from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · umol/L
Total Bilirubin
umol/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Total Bilirubin0.86 ± 1.2090.94 ± 1.0951.58 ± 2.3090.52 ± 2.793
PrimaryBlood Urea Nitrogen

Changes in blood urea nitrogen from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · mmol/L
Blood Urea Nitrogen
mmol/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Blood Urea Nitrogen0.36 ± 1.010-2.12 ± 3.1240.09 ± 1.468-0.49 ± 1.154
PrimaryCreatinine

Changes in creatinine from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · umol/L
Creatinine
umol/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Creatinine-4.86 ± 5.626-27.72 ± 54.8374.20 ± 6.431-0.62 ± 10.959
PrimaryUrine pH

Changes in urine pH from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · pH
Urine pH
pHAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Urine pH0.00 ± 1.4140.13 ± 2.056-0.50 ± 0.913-0.34 ± 0.889
PrimaryNumber of Participants With a Change in Urine Glucose

Number of Participants with a Change in Urine Glucose

Time frame:
From study start to end, up to 3.5 years
Reported as:
Count of participants · Participants
Number of Participants With a Change in Urine Glucose
ParticipantsAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
From Normal to Normal24415
From High Non-CS to Normal0001
Missing0001
PrimaryNumber of Participants With a Change in Urine Ketones

Number of Participants With a Change in Urine Ketones at baseline and end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Count of participants · Participants
Number of Participants With a Change in Urine Ketones
ParticipantsAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
From Normal to Normal24413
From Normal to High Non-CS0001
From High Non-CS to Normal0002
From High Non-CS to Missing0001
PrimaryNumber of Participants With a Change in Urine Leukocytes

Number of participants with a change in urine leukocytes at baseline and end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Count of participants · Participants
Number of Participants With a Change in Urine Leukocytes
ParticipantsAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
From Normal to Normal23414
From Normal to High Non-CS0001
From Normal to Missing0102
PrimaryNumber of Participants With a Change in Urine Hemoglobin

Number of participants with a change in urine hemoglobin at baseline and end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Count of participants · Participants
Number of Participants With a Change in Urine Hemoglobin
ParticipantsAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
From Normal to Normal24414
From Normal to High Non-CS0001
From High Non-CS to Normal0001
From High Non-CS to Missing0001
PrimaryComplete Red Blood Cell Count

Changes in complete red blood cell count from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · 10^12 cells/L
Complete Red Blood Cell Count
10^12 cells/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Complete Red Blood Cell Count0.14 ± 0.311-0.06 ± 0.2560.17 ± 0.3210.01 ± 0.287
PrimaryHaematocrit

Changes in haematocrit from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · L/L
Haematocrit
L/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Haematocrit0.02 ± 0.0340.00 ± 0.0130.02 ± 0.0320.00 ± 0.030
PrimaryHaemoglobin

Changes in haemoglobin from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · g/L
Haemoglobin
g/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Haemoglobin8.00 ± 7.071-2.08 ± 4.1984.25 ± 11.6731.29 ± 8.513
PrimaryComplete White Blood Cell Count

Changes in complete white blood cell count from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · 10^9 cells/L
Complete White Blood Cell Count
10^9 cells/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Complete White Blood Cell Count1.45 ± 1.626-0.05 ± 1.0340.40 ± 1.655-0.24 ± 1.722
SecondaryMeasurement of Trough Total IgG Levels

Measurement of trough total IgG levels from baseline to end of study

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · g/L
Measurement of Trough Total IgG Levels
g/LAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
IgG at Screening10.99 ± 2.14311.92 ± 3.35212.17 ± 2.73113.17 ± 3.236
IgG at End of Study12.50 ± 3.04815.45 ± 7.3599.38 ± 1.84213.28 ± 3.336
SecondaryNumber of Participants With Serious Bacterial Infections (SBIs).

Number of participants with serious bacterial infections

Time frame:
From study start to end, up to 3.5 years
Reported as:
Count of participants · Participants
Number of Participants With Serious Bacterial Infections (SBIs).
ParticipantsAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
Number of Participants With Serious Bacterial Infections (SBIs).0001
SecondarySF-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

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · score on a scale
SF-36 Health Survey.
score on a scaleAge >=12 to <17Age >=17 to <=75
Physical Health Score12.390.42 ± 6.922
Mental Health Score-1.120.38 ± 10.877
SecondaryCHQ-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.

Time frame:
From study start to end, up to 3.5 years
Reported as:
Mean · score on a scale
CHQ-PF50 (Child Health Questionnaire-Parent Form)
score on a scaleAge >=2 to <6Age >=6 to <12Age >=12 to <17
Physical Summary Score-0.79 ± 5.0320.94 ± 6.8002.76 ± 2.683
Psychosocial Summary-9.07 ± 12.3610.50 ± 2.6799.92 ± 20.860

Adverse events

Collected over From study start to end, up to 3.5 years. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Age >=2 to <60/2 (0%)0/2 (0%)2/2 (100%)
Age >=6 to <120/4 (0%)1/4 (25%)4/4 (100%)
Age >=12 to <170/4 (0%)2/4 (50%)4/4 (100%)
Age >=17 to <=750/17 (0%)4/17 (23.5%)14/17 (82.4%)
Most frequent serious events
Showing 10 of 14
Most frequent serious events
EventAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
SeizureNervous system disorders0/21/40/40/17
Pulmonary embolismRespiratory, thoracic and mediastinal disorders0/20/41/40/17
Status asthmaticusRespiratory, thoracic and mediastinal disorders0/20/41/40/17
Irritable bowel syndromeGastrointestinal disorders0/20/40/41/17
PyrexiaGeneral disorders0/20/40/41/17
DiverticulitisInfections and infestations0/20/40/41/17
Escherichia bacteraemiaInfections and infestations0/20/40/41/17
Infected biteInfections and infestations0/20/40/41/17
Subdural haematomaInjury, poisoning and procedural complications0/20/40/41/17
HyponatraemiaMetabolism and nutrition disorders0/20/40/41/17
Most frequent other events
Showing 10 of 157
Most frequent other events
EventAge >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75
BronchitisInfections and infestations1/20/43/43/17
TicPsychiatric disorders1/20/40/40/17
HeadacheNervous system disorders0/22/41/42/17
AsthmaRespiratory, thoracic and mediastinal disorders0/22/41/41/17
Nasal congestionRespiratory, thoracic and mediastinal disorders1/20/40/40/17
Sleep apnoea syndromeRespiratory, thoracic and mediastinal disorders1/21/40/40/17
NauseaGastrointestinal disorders1/20/41/41/17
VomitingGastrointestinal disorders1/21/41/40/17
Dermatitis contactSkin and subcutaneous tissue disorders1/20/40/42/17
Acute sinusitisInfections and infestations1/21/41/41/17

Baseline characteristics

Age, Continuous
Age, Continuous(years)Age >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75Total
Median6.50 (6.0 to 7.0)9.00 (7.0 to 11.0)14.25 (13.0 to 15.0)56.12 (25.0 to 73.0)39.26 (6.0 to 73.0)
Sex: Female, Male
Sex: Female, Male(Participants)Age >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75Total
Female0221317
Male222410
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Age >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75Total
Hispanic or Latino00000
Not Hispanic or Latino2441727
Unknown or Not Reported00000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Age >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75Total
American Indian or Alaska Native00000
Asian00000
Native Hawaiian or Other Pacific Islander00000
Black or African American00000
White2441525
More than one race00011
Unknown or Not Reported00011
ABO Rhesus Blood Type
ABO Rhesus Blood Type(Participants)Age >=2 to <6Age >=6 to <12Age >=12 to <17Age >=17 to <=75Total
A Blood Type122813
AB Blood Type00112
O Blood Type121812
08

Study locations

7 sites
  • Octapharma Research Site
    Irvine, California 92697, United States
  • Octapharma Research Site
    San Diego, California 92123, United States
  • Octapharma Research Site
    Centennial, Colorado 80112, United States
  • Octapharma Research Site
    Papillion, Nebraska 68046, United States
  • Octapharma Research Site
    Toledo, Ohio 43617, United States
  • Octapharma Research Site
    Frisco, Texas 75034, United States
  • Octapharma Research Site
    Edmonton, Alberta T6G2V2, Canada
09

References and documents

Study documents

  • Study protocol · Oct 25, 2017
  • Statistical analysis plan · Jul 17, 2019
  • Study protocol · Oct 4, 2018

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 27, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03907241
Lead sponsor
Octapharma
Responsible party
Sponsor
First posted
Apr 8, 2019
Start date
Mar 1, 2016
Primary completion
Sep 5, 2019
Completion
Sep 5, 2019
Results posted
Sep 22, 2020
Last update
Oct 27, 2020

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Oct 2020. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Primary Immunodeficiency Diseases→

Other studies from this sponsor

Octapharma

Start the discussion