Trial Outcomes & Findings for Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases (NCT NCT03988426)
NCT ID: NCT03988426
Last Updated: 2020-03-10
Results Overview
Serious Bacterial Infections defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess
COMPLETED
PHASE3
25 participants
Primary Treatment Period (24 Weeks)
2020-03-10
Participant Flow
Participant milestones
| Measure |
Participants Administered Octanorm
All patients in the study received Octanorm as study drug. Dosage of octanorm was based on the previous dosage of IVIG for each patient: by dividing the previous IVIG dosage by the number of weeks between IVIG administrations. Octanorm was to be administered every week (+/- 2 days) as subcutaneous administration. Administration were either done at the study site (for the first training sessions and then every 4th infusions) or at home (administered by the patient or a caregiver)
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|---|---|
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Overall Study
STARTED
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25
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Overall Study
COMPLETED
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24
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Overall Study
NOT COMPLETED
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1
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases
Baseline characteristics by cohort
| Measure |
All Patients
n=25 Participants
Full analysis set (FAS population) included all patients who received at least one administration of the study drug and for whom any post-baseline data was available
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Age, Continuous
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35.24 years
n=99 Participants
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Sex: Female, Male
Female
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15 Participants
n=99 Participants
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Sex: Female, Male
Male
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10 Participants
n=99 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=99 Participants
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Race (NIH/OMB)
Asian
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0 Participants
n=99 Participants
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Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=99 Participants
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Race (NIH/OMB)
Black or African American
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0 Participants
n=99 Participants
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Race (NIH/OMB)
White
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25 Participants
n=99 Participants
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Race (NIH/OMB)
More than one race
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0 Participants
n=99 Participants
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Race (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=99 Participants
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Region of Enrollment
Russia
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25 participants
n=99 Participants
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Height
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170 centimeters
STANDARD_DEVIATION 8.68 • n=99 Participants
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Weight
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66.49 kilograms
STANDARD_DEVIATION 10.54 • n=99 Participants
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BMI (Body Mass Index)
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23.02 kg/m^2
STANDARD_DEVIATION 3.48 • n=99 Participants
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PRIMARY outcome
Timeframe: Primary Treatment Period (24 Weeks)Serious Bacterial Infections defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess
Outcome measures
| Measure |
Octanorm
n=24 Participants
Octanorm: Octanorm
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|---|---|
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Number of Serious Bacterial Infections Per Person-Year on Treatment
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0 SBI per patient year
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SECONDARY outcome
Timeframe: Primary Treatment Period (24 Weeks)The number of patients with all infections of any kind or seriousness.
Outcome measures
| Measure |
Octanorm
n=24 Participants
Octanorm: Octanorm
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|---|---|
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Number of Patients With Other Infections
Number of Patients With Infections
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14 Participants
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Number of Patients With Other Infections
Number of Patients with 0 Infections
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10 Participants
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Number of Patients With Other Infections
Number of Patients with 1 Infection
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9 Participants
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Number of Patients With Other Infections
Number of Patients with 2 Infections
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2 Participants
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Number of Patients With Other Infections
Number of Patients with 3 Infections
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0 Participants
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Number of Patients With Other Infections
Number of Patients with 4 Infections
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2 Participants
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Number of Patients With Other Infections
Number of Patients with 5 Infections
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1 Participants
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SECONDARY outcome
Timeframe: Primary Treatment Period (24 Weeks)For other infections, the Medical Dictionary for Regulatory Activities (MedDRA) preferred term was used to determine the type of infection. They were grouped into the following categories as determined by a medical expert: Ear infections, eye infections, infections of the gastrointestinal tract, infections of the genitourinary tract, upper respiratory tract infections, lower respiratory tract infections, infections of the skin, and infections not elsewhere classified.
Outcome measures
| Measure |
Octanorm
n=24 Participants
Octanorm: Octanorm
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|---|---|
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Number of Other Infections
Number of Mild Infections
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17 Infections
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Number of Other Infections
Number of Moderate Infections
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9 Infections
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Number of Other Infections
Number of Severe Infections
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0 Infections
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SECONDARY outcome
Timeframe: Primary Treatment Period (24 Weeks) and Whole Treatment Period (up to 36 Weeks)Since infections were reported as adverse events, the time to resolution of an infection was the time from the start date of the infection adverse event to the end date of the infection adverse event.
Outcome measures
| Measure |
Octanorm
n=24 Participants
Octanorm: Octanorm
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|---|---|
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Time to Resolution of Infections
Primary Treatment Period
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9.53 days
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Time to Resolution of Infections
Whole Treatment Period
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10.32 days
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SECONDARY outcome
Timeframe: Primary Treatment Period (24 Weeks)Number of patients using antibiotics during the whole treatment period (36 weeks) grouped per number of days with antibiotic usage.
Outcome measures
| Measure |
Octanorm
n=24 Participants
Octanorm: Octanorm
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|---|---|
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Number of Participants Using Antibiotics From 0 to > 20 Days
Patients with 0 Treatment Days
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14 Participants
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Number of Participants Using Antibiotics From 0 to > 20 Days
Patients with 5 Treatment Days
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2 Participants
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Number of Participants Using Antibiotics From 0 to > 20 Days
Patients with 7 Treatment Days
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1 Participants
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Number of Participants Using Antibiotics From 0 to > 20 Days
Patients with 8 Treatment Days
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2 Participants
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Number of Participants Using Antibiotics From 0 to > 20 Days
Patients with >20 Treatment Days
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5 Participants
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SECONDARY outcome
Timeframe: Primary Treatment Period (24 Weeks)The number of antibiotic treatment episodes per person-year of treatment was calculated by the following formula: Total number of antibiotic treatment episodes / patient-years of Octanorm treatment
Outcome measures
| Measure |
Octanorm
n=24 Participants
Octanorm: Octanorm
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|---|---|
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Annual Rate of Antibiotic Use
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1.73 treatment episodes per person-year
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SECONDARY outcome
Timeframe: Primary Treatment Period (24 Weeks)Number of days spent in hospital due to infection
Outcome measures
| Measure |
Octanorm
n=24 Participants
Octanorm: Octanorm
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|---|---|
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Hospitalizations Due to Infection
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0 days
Standard Deviation 0
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SECONDARY outcome
Timeframe: Primary Treatment Period (24 Weeks)Annual Rate of Hospitalizations due to Infection
Outcome measures
| Measure |
Octanorm
n=24 Participants
Octanorm: Octanorm
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|---|---|
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Rate of Hospitalizations Due to Infection
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0 hospitalizations/person-year
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SECONDARY outcome
Timeframe: Primary Treatment Period (24 Weeks) and Whole Treatment Period (up to 36 Weeks)Number of episodes of fever
Outcome measures
| Measure |
Octanorm
n=24 Participants
Octanorm: Octanorm
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|---|---|
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Episodes of Fever
Primary Treatment Period
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6 episodes of fever
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Episodes of Fever
Whole Treatment Period
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8 episodes of fever
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SECONDARY outcome
Timeframe: Primary Treatment Period (24 Weeks)The number of episodes of fever per person-year of treatment was calculated by the following formula: Total number of episodes of fever / patient-years of Octanorm treatment
Outcome measures
| Measure |
Octanorm
n=24 Participants
Octanorm: Octanorm
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|---|---|
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Rate of Episodes of Fever
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0.55 episodes of fever per person-year
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SECONDARY outcome
Timeframe: Primary Treatment Period (24 Weeks)Total number of patients who missed days from work or study due to infections or treatment thereof.
Outcome measures
| Measure |
Octanorm
n=24 Participants
Octanorm: Octanorm
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|---|---|
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Patients With Days Missed From Work/Study Due to Infections and Treatment
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3 Participants
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SECONDARY outcome
Timeframe: Baseline to the end of study (up to 36 weeks)The SF-36-HS consists of 36 items organized into 8 subscales. The 8 subscales could be combined into 2 summary scores, physical and mental. The calculated summary scores were transformed to a range of 0-100, where a higher score indicates better health. A positive change score indicates improvement.
Outcome measures
| Measure |
Octanorm
n=25 Participants
Octanorm: Octanorm
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|---|---|
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Changes in the Subscales of the Form-36 Health Survey Scores From Baseline to the End of the Study
Physical Functioning
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2.5 score on a scale
Standard Deviation 12.25
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Changes in the Subscales of the Form-36 Health Survey Scores From Baseline to the End of the Study
Role Physical
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8.07 score on a scale
Standard Deviation 25.83
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Changes in the Subscales of the Form-36 Health Survey Scores From Baseline to the End of the Study
Bodily Pain
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21.38 score on a scale
Standard Deviation 28.12
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Changes in the Subscales of the Form-36 Health Survey Scores From Baseline to the End of the Study
General Health
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-1.08 score on a scale
Standard Deviation 15.16
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Changes in the Subscales of the Form-36 Health Survey Scores From Baseline to the End of the Study
Vitality
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5.73 score on a scale
Standard Deviation 17.67
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Changes in the Subscales of the Form-36 Health Survey Scores From Baseline to the End of the Study
Social Functioning
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7.81 score on a scale
Standard Deviation 23.26
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Changes in the Subscales of the Form-36 Health Survey Scores From Baseline to the End of the Study
Role Emotional
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10.76 score on a scale
Standard Deviation 22.18
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Changes in the Subscales of the Form-36 Health Survey Scores From Baseline to the End of the Study
Mental Health
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5.83 score on a scale
Standard Deviation 20.62
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SECONDARY outcome
Timeframe: At baseline and at last infusion (week 33)Total IgG trough concentrations were measured in serum samples taken before each infusion given at the study site.
Outcome measures
| Measure |
Octanorm
n=25 Participants
Octanorm: Octanorm
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|---|---|
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Trough Levels of Serum Total IgG
Screening Draw 1
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8.71 g/L
Standard Deviation 2.68
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Trough Levels of Serum Total IgG
Week 33
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9.99 g/L
Standard Deviation 1.79
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SECONDARY outcome
Timeframe: Up to 36 weeksTEAEs were classified as temporally associated if the onset was during the infusion or within 72 hours after the end of the infusion.
Outcome measures
| Measure |
Octanorm
n=25 Participants
Octanorm: Octanorm
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|---|---|
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Number of Participants Experiencing Treatment-Emergent AEs
Without Infusion Site Reactions and With Infection
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18 patients
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Number of Participants Experiencing Treatment-Emergent AEs
Without Infusion Site Reactions/Without Infection
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11 patients
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Number of Participants Experiencing Treatment-Emergent AEs
Infections (only)
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16 patients
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SECONDARY outcome
Timeframe: Up to 36 weeksThe proportion of infusions with at least 1 temporally associated AE (TAAE) was calculated by dividing the total number of TAAE by the total number of infusions.
Outcome measures
| Measure |
Octanorm
n=25 Participants
Octanorm: Octanorm
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|---|---|
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Proportion of Infusions With at Least 1 Temporally Associated AE
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.013 proportion of infusions
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SECONDARY outcome
Timeframe: Up to 36 weeksAn AE is any untoward medical occurrence in a study patient receiving an IMP and which does not necessarily have a causal relationship with this treatment.
Outcome measures
| Measure |
Octanorm
n=25 Participants
Octanorm: Octanorm
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|---|---|
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Total Number of Adverse Events Regardless of Causality
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59 adverse events
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SECONDARY outcome
Timeframe: Up to 36 weeksA related adverse event is an AE for which a causal relationship between the IMP and the AE cannot be ruled out.
Outcome measures
| Measure |
Octanorm
n=25 Participants
Octanorm: Octanorm
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|---|---|
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Number of Related Adverse Events
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3 related adverse events
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SECONDARY outcome
Timeframe: Up to 36 weeksTotal number of infusions that triggered an infusion site reaction and number of infusions that triggered mild, moderate, severe or no infusion site reactions.
Outcome measures
| Measure |
Octanorm
n=25 Participants
Octanorm: Octanorm
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|---|---|
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Number of Infusions With Infusion Site Reaction
Infusions with Mild Local Site Reactions
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102 infusions
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Number of Infusions With Infusion Site Reaction
Infusions with Moderate Local Site Reactions
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17 infusions
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Number of Infusions With Infusion Site Reaction
Infusions with Severe Local Site Reactions
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0 infusions
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Number of Infusions With Infusion Site Reaction
Infusions with No Infusion Site Reaction
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659 infusions
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SECONDARY outcome
Timeframe: Up to 36 weeksThe annual rate of all infections of any kind of seriousness
Outcome measures
| Measure |
Octanorm
n=24 Participants
Octanorm: Octanorm
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|---|---|
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Annual Rate of Infections
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2.37 infections/person-year
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Adverse Events
Octanorm
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Octanorm
n=25 participants at risk
Octanorm: Octanorm
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|---|---|
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Nervous system disorders
Dizziness
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8.0%
2/25 • Number of events 2 • Up to 36 weeks
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Gastrointestinal disorders
Food Poisoning
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8.0%
2/25 • Number of events 2 • Up to 36 weeks
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General disorders
Condition Aggravated
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20.0%
5/25 • Number of events 6 • Up to 36 weeks
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Infections and infestations
Respiratory Tract Infection
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24.0%
6/25 • Number of events 8 • Up to 36 weeks
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Infections and infestations
Bronchitis
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16.0%
4/25 • Number of events 5 • Up to 36 weeks
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Infections and infestations
Rhinitis
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8.0%
2/25 • Number of events 2 • Up to 36 weeks
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Infections and infestations
Pharyngitis
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8.0%
2/25 • Number of events 2 • Up to 36 weeks
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Blood and lymphatic system disorders
Blood and Lymphatic Infection
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8.0%
2/25 • Number of events 2 • Up to 36 weeks
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Investigations
Investigations
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8.0%
2/25 • Number of events 2 • Up to 36 weeks
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place