Trial Outcomes & Findings for Study of Octanorm Subcutaneous IG in Patients With PID (NCT NCT01888484)
NCT ID: NCT01888484
Last Updated: 2021-08-17
Results Overview
The primary efficacy outcome is the rate of SBI (Serious bacterial infections - defined as bacteremia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess) per person-year on treatment.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
75 participants
Primary outcome timeframe
Every 4 weeks until the final evaluation at week 65.
Results posted on
2021-08-17
Participant Flow
Participant milestones
| Measure |
Children ≥2 Years <6 Years
Children ≥2 Years \<6 Years
|
Children Aged ≥6 to <12 Years
Children Aged ≥6 to \<12 years
|
Adolescents ≥12 Years <17 Years
Adolescents ≥12 Years \<17 Years
|
Adults ≥17 Years ≤75 Years
Adults ≥17 Years ≤75 Years
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
12
|
14
|
12
|
37
|
|
Overall Study
COMPLETED
|
12
|
14
|
8
|
34
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
4
|
3
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Octanorm Subcutaneous IG in Patients With PID
Baseline characteristics by cohort
| Measure |
All Patients
n=75 Participants
All patients.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|---|---|
|
Age, Continuous
|
27.81 years
STANDARD_DEVIATION 21.911 • n=5 Participants
|
|
Sex: Female, Male
Female
|
36 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
39 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
75 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
74 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Every 4 weeks until the final evaluation at week 65.Population: The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
The primary efficacy outcome is the rate of SBI (Serious bacterial infections - defined as bacteremia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess) per person-year on treatment.
Outcome measures
| Measure |
All Patients
n=75 Participants
All patients
|
|---|---|
|
Rate of SBI Per Person-year
|
0 SBIs per person year
|
PRIMARY outcome
Timeframe: Measured at Week 28 before the start of the SC infusion, 10 minutes before the end of the infusion, and at 2 h, 1, 2, 3, 4 and 7 days after the end of the infusion. Calculated and averaged.Population: In several cases, AUCτ could not be calculated due to very flat time-concentration profiles.
The primary endpoint with respect to the PK investigations is the area under the curve AUC(t) (i.e., AUC from time 0 (start of the infusion) to the end of the nominal dosing period, standardised to 1 week) at PKSC2 at steady-state conditions.
Outcome measures
| Measure |
All Patients
n=24 Participants
All patients
|
|---|---|
|
AUC(t) at Steady-State Conditions
|
2232.61 h*g/L
Standard Deviation 585.842
|
SECONDARY outcome
Timeframe: Up to 65 weeksPopulation: The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
The annual rate of all infections of any kind or seriousness.
Outcome measures
| Measure |
All Patients
n=75 Participants
All patients
|
|---|---|
|
The Annual Rate of All Infections of Any Kind or Seriousness.
|
3.414 infections per person-year
|
SECONDARY outcome
Timeframe: Up to 65 weeksPopulation: The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
Non-serious infections (total and by category).
Outcome measures
| Measure |
All Patients
n=75 Participants
All patients
|
|---|---|
|
Non-serious Infections
Ear infections
|
8 infections
|
|
Non-serious Infections
Eye infections
|
3 infections
|
|
Non-serious Infections
Infections of the GI tract
|
25 infections
|
|
Non-serious Infections
Infections of the genitourinary tract
|
24 infections
|
|
Non-serious Infections
Upper respiratory tract infections
|
179 infections
|
|
Non-serious Infections
Lower respiratory tract infections
|
32 infections
|
|
Non-serious Infections
Infections of the skin
|
6 infections
|
|
Non-serious Infections
Infections not (elsewhere) classified
|
16 infections
|
SECONDARY outcome
Timeframe: Measured at week 28Population: The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group. Data not available for all patients.
Cmax (Maximum Plasma Concentration) of total IgG and IgG Subclasses, where the mean value was calculated and reported
Outcome measures
| Measure |
All Patients
n=37 Participants
All patients
|
|---|---|
|
Cmax of Total IgG and IgG Subclasses
IgG4 Cmax
|
0.40 g/L
Standard Deviation 0.567
|
|
Cmax of Total IgG and IgG Subclasses
IgG2 Cmax
|
3.88 g/L
Standard Deviation 1.532
|
|
Cmax of Total IgG and IgG Subclasses
IgG Total Cmax
|
13.47 g/L
Standard Deviation 3.655
|
|
Cmax of Total IgG and IgG Subclasses
IgG1 Cmax
|
8.56 g/L
Standard Deviation 1.858
|
|
Cmax of Total IgG and IgG Subclasses
IgG3 Cmax
|
0.32 g/L
Standard Deviation 0.127
|
SECONDARY outcome
Timeframe: Measured at Week 28 for all patients, median value was calculatedPopulation: The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group. Data not available for all patients.
Tmax (Time to Maximum Plasma Concentration) of total IgG and IgG Subclasses
Outcome measures
| Measure |
All Patients
n=37 Participants
All patients
|
|---|---|
|
Tmax of Total IgG and IgG Subclasses
Tmax Total IgG
|
49.62 hours
Interval 0.8 to 98.3
|
|
Tmax of Total IgG and IgG Subclasses
Tmax IgG1
|
50.50 hours
Interval 1.5 to 167.7
|
|
Tmax of Total IgG and IgG Subclasses
Tmax IgG2
|
72.20 hours
Interval 0.5 to 167.7
|
|
Tmax of Total IgG and IgG Subclasses
Tmax IgG3
|
49.62 hours
Interval 1.8 to 98.1
|
|
Tmax of Total IgG and IgG Subclasses
Tmax IgG4
|
47.50 hours
Interval 0.6 to 167.7
|
SECONDARY outcome
Timeframe: Measured at Week 28Population: The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group. Data not available for all patients.
AUC (Area Under the Concentration-Time Curve) of total IgG and IgG Subclasses calculated for all patients and mean value was calculated and reported
Outcome measures
| Measure |
All Patients
n=25 Participants
All patients
|
|---|---|
|
AUC of Total IgG and IgG Subclasses
IgG Total AUC
|
2232.61 hr*g/L
Standard Deviation 585.842
|
|
AUC of Total IgG and IgG Subclasses
IgG1 AUC
|
1424.69 hr*g/L
Standard Deviation 298.776
|
|
AUC of Total IgG and IgG Subclasses
IgG2 AUC
|
659.57 hr*g/L
Standard Deviation 262.295
|
|
AUC of Total IgG and IgG Subclasses
IgG3 AUC
|
48.65 hr*g/L
Standard Deviation 18.455
|
|
AUC of Total IgG and IgG Subclasses
IgG4 AUC
|
71.40 hr*g/L
Standard Deviation 102.578
|
SECONDARY outcome
Timeframe: Measured once at Week 28, seven days after 28th infusion of octanormPopulation: The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group. Data not available for all patients.
Trough levels of serum IgG, IgG1, IgG2, IgG3, IgG4 at PK 7 days after 28th infusion of octanorm measured for all patients and median value was calculated and reported
Outcome measures
| Measure |
All Patients
n=36 Participants
All patients
|
|---|---|
|
Trough Levels of Serum IgG
Total IgG
|
11.85 g/L
Interval 10.0 to 13.7
|
|
Trough Levels of Serum IgG
IgG1
|
7.51 g/L
Interval 6.58 to 9.0
|
|
Trough Levels of Serum IgG
IgG2
|
3.12 g/L
Interval 2.69 to 3.85
|
|
Trough Levels of Serum IgG
IgG3
|
0.27 g/L
Interval 0.2 to 0.34
|
|
Trough Levels of Serum IgG
IgG4
|
0.15 g/L
Interval 0.11 to 0.32
|
SECONDARY outcome
Timeframe: AUC Measured at Week 28Population: The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group. Data not available for all patients.
IVIG to Octanorm Dose Conversion Factor (based on the area under the concentration-time curve \[AUCτ\]) -- determined by least-squares regression Model which was without restriction of the intercept.
Outcome measures
| Measure |
All Patients
n=37 Participants
All patients
|
|---|---|
|
IVIG to Octanorm DCF (Based on the Area Under the Concentration-time Curve [AUCτ])
|
1.278 unitless
Standard Deviation 0.9401
|
Adverse Events
All Patients
Serious events: 9 serious events
Other events: 61 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Patients
n=75 participants at risk
All patients
|
|---|---|
|
Infections and infestations
Abscess limb
|
1.3%
1/75 • Number of events 1 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Appendicitis
|
1.3%
1/75 • Number of events 1 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Acute odontogenic jaw osteomyelitis
|
1.3%
1/75 • Number of events 1 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Pneumocystitis jiroveci infection
|
1.3%
1/75 • Number of events 1 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Respiratory syncytial virus bronchiolitis
|
1.3%
1/75 • Number of events 1 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Tracheitis
|
1.3%
1/75 • Number of events 1 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Thyroid neoplasm
|
1.3%
1/75 • Number of events 1 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Psychiatric disorders
Asperger's syndrome
|
1.3%
1/75 • Number of events 1 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Nervous system disorders
Grand mal convulsion
|
1.3%
1/75 • Number of events 1 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
1.3%
1/75 • Number of events 1 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
1.3%
1/75 • Number of events 1 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Injury, poisoning and procedural complications
Spinal compression fracture
|
1.3%
1/75 • Number of events 1 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
Other adverse events
| Measure |
All Patients
n=75 participants at risk
All patients
|
|---|---|
|
Blood and lymphatic system disorders
Leukopenia
|
6.7%
5/75 • Number of events 5 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Blood and lymphatic system disorders
Neutropenia
|
5.3%
4/75 • Number of events 5 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Nervous system disorders
Headache
|
8.0%
6/75 • Number of events 14 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
8.0%
6/75 • Number of events 9 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Gastrointestinal disorders
Diarrhoea
|
14.7%
11/75 • Number of events 18 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Gastrointestinal disorders
Vomiting
|
8.0%
6/75 • Number of events 8 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
6.7%
5/75 • Number of events 5 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
5.3%
4/75 • Number of events 6 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.3%
4/75 • Number of events 4 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
5.3%
4/75 • Number of events 4 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
General disorders
Pyrexia
|
16.0%
12/75 • Number of events 14 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
10.7%
8/75 • Number of events 9 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Injury, poisoning and procedural complications
Excoriation
|
6.7%
5/75 • Number of events 6 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Injury, poisoning and procedural complications
Fall
|
5.3%
4/75 • Number of events 4 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Acute sinusitis
|
6.7%
5/75 • Number of events 13 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Bronchitis
|
9.3%
7/75 • Number of events 14 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Gastroenteritis viral
|
5.3%
4/75 • Number of events 6 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Influenza
|
6.7%
5/75 • Number of events 5 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Laryngitis
|
5.3%
4/75 • Number of events 5 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Nasopharyngitis
|
22.7%
17/75 • Number of events 37 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Otitis media
|
5.3%
4/75 • Number of events 5 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Rhinitis
|
14.7%
11/75 • Number of events 17 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Rhinovirus infection
|
6.7%
5/75 • Number of events 7 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Sinusitis
|
20.0%
15/75 • Number of events 25 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Upper respiratory tract infection
|
28.0%
21/75 • Number of events 33 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Infections and infestations
Urinary tract infection
|
8.0%
6/75 • Number of events 11 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.0%
15/75 • Number of events 22 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.3%
4/75 • Number of events 4 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Renal and urinary disorders
Dysuria
|
5.3%
4/75 • Number of events 4 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
|
Skin and subcutaneous tissue disorders
Any infusion site reaction
|
73.3%
55/75 • Number of events 994 • For the duration of the 65 week treatment period starting with the first infusion of octanorm.
The data are reported as the overall population as it was not the intent of the protocol to report analysis by age group
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place