Trial Outcomes & Findings for Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP) (NCT NCT01176266)
NCT ID: NCT01176266
Last Updated: 2019-03-13
Results Overview
The effect of asfotase alfa treatment on skeletal manifestations of HPP (i.e., change in rickets severity) was measured by radiographs using a qualitative Radiographic Global Impression of Change (RGI-C) scale. Skeletal radiographs obtained at Week 24 were compared with skeletal radiographs obtained before initiation of treatment. The RGI-C is a 7-point rating scale that ranges from -3 (indicative of severe worsening of HPP-associated rickets) to +3 (indicative of complete or near complete healing of HPP-associated rickets).
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
69 participants
Primary outcome timeframe
From Baseline to Week 24
Results posted on
2019-03-13
Participant Flow
The main criteria for inclusion in the study were male and female patients less than or equal to 5 years of age with a documented diagnosis of infantile onset hypophosphatasia (HPP) who were otherwise medically stable. Patients must have had onset of HPP signs/symptoms prior to 6 months of age.
Participant milestones
| Measure |
Asfotase Alfa
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
|---|---|
|
Overall Study
STARTED
|
69
|
|
Overall Study
COMPLETED
|
60
|
|
Overall Study
NOT COMPLETED
|
9
|
Reasons for withdrawal
| Measure |
Asfotase Alfa
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
|---|---|
|
Overall Study
Death
|
6
|
|
Overall Study
Withdrawal by Subject
|
3
|
Baseline Characteristics
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
Baseline characteristics by cohort
| Measure |
Asfotase Alfa
n=69 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
|---|---|
|
Age, Continuous
|
113.42 weeks
STANDARD_DEVIATION 108.912 • n=5 Participants
|
|
Sex: Female, Male
Female
|
36 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
33 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
63 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
5 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaskan Native
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
7 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
54 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
3 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Unknown
|
5 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From Baseline to Week 24Population: Full Analysis Set
The effect of asfotase alfa treatment on skeletal manifestations of HPP (i.e., change in rickets severity) was measured by radiographs using a qualitative Radiographic Global Impression of Change (RGI-C) scale. Skeletal radiographs obtained at Week 24 were compared with skeletal radiographs obtained before initiation of treatment. The RGI-C is a 7-point rating scale that ranges from -3 (indicative of severe worsening of HPP-associated rickets) to +3 (indicative of complete or near complete healing of HPP-associated rickets).
Outcome measures
| Measure |
Asfotase Alfa
n=69 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Effect of Asfotase Alfa Treatment on Skeletal Manifestations of Hypophosphatasia (HPP)
|
2.00 scores on a scale
Interval -1.67 to 3.0
|
—
|
—
|
PRIMARY outcome
Timeframe: Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years).Population: Full Analysis Set
Safety and tolerability of repeated subcutaneous (SC) injections of asfotase alfa for all treated patients was assessed by the number of patients with 1 or more treatment-emergent adverse event.
Outcome measures
| Measure |
Asfotase Alfa
n=69 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Safety and Tolerability of Repeated Subcutaneous (SC) Injections of Asfotase Alfa
|
69 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 72 Months or regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years).Population: Full Analysis Set
The effect of asfotase alfa treatment on skeletal manifestations of HPP (i.e., change in rickets severity) was measured by radiographs using a qualitative Radiographic Global Impression of Change (RGI-C) scale. Skeletal radiographs obtained at the patient's last assessment were compared with skeletal radiographs obtained before initiation of treatment. The RGI-C is a 7-point rating scale that ranges from -3 (indicative of severe worsening of HPP-associated rickets) to +3 (indicative of complete or near complete healing of HPP-associated rickets).
Outcome measures
| Measure |
Asfotase Alfa
n=67 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Effect of Asfotase Alfa Treatment on Skeletal Manifestations of Hypophosphatasia (HPP)
|
2.33 scores on a scale
Interval -2.67 to 3.0
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years).Population: Patients on respiratory support at Baseline were excluded from the analysis.
For patients who were not on respiratory support at the time of enrollment, the Kaplan-Meier estimate of ventilator-free survival at the end of the study
Outcome measures
| Measure |
Asfotase Alfa
n=45 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Effect of Asfotase Alfa Treatment on Ventilator-free Survival (Week 312)
|
0.81 Probability
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years).Population: Full Analysis Set
Effect of asfotase alfa treatment on respiratory function as measured by the shift in proportion of patients requiring respiratory support at their last assessment compared with Baseline.
Outcome measures
| Measure |
Asfotase Alfa
n=69 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
n=45 Participants
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
n=24 Participants
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Effect of Asfotase Alfa Treatment on Respiratory Function
No respiratory support
|
45 Participants
|
43 Participants
|
11 Participants
|
|
Effect of Asfotase Alfa Treatment on Respiratory Function
Supplemental oxygen
|
6 Participants
|
1 Participants
|
2 Participants
|
|
Effect of Asfotase Alfa Treatment on Respiratory Function
Biphasic positive airway pressure
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Effect of Asfotase Alfa Treatment on Respiratory Function
Continuous positive airway pressure
|
4 Participants
|
1 Participants
|
2 Participants
|
|
Effect of Asfotase Alfa Treatment on Respiratory Function
Mechanical ventilation
|
13 Participants
|
0 Participants
|
9 Participants
|
|
Effect of Asfotase Alfa Treatment on Respiratory Function
Other
|
1 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years).Population: Full Analysis Set
Effect of asfotase alfa treatment on physical growth as measured by change from Baseline to last assessment for each patient in length/height Z-scores
Outcome measures
| Measure |
Asfotase Alfa
n=66 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Effect of Asfotase Alfa Treatment on Physical Growth - Length/Height Z-scores Change From Baseline to Last Obtained Value
|
0.5 Z-score
Interval -4.0 to 4.0
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years).Population: Full Analysis Set
Effect of asfotase alfa treatment on physical growth as measured by change from Baseline to last assessment for each patient in weight Z-scores
Outcome measures
| Measure |
Asfotase Alfa
n=67 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Effect of Asfotase Alfa Treatment on Physical Growth - Weight Z-scores Change From Baseline to Last Obtained Value
|
1.0 Z-score
Interval -5.0 to 6.0
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years).Population: Full Analysis Set
Effect of asfotase alfa on PPi as measured by change from Baseline to last assessment for each patient
Outcome measures
| Measure |
Asfotase Alfa
n=63 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Effect of Asfotase Alfa on Biomarkers - Plasma Inorganic Pyrophosphate (PPi) Change From Baseline to Last Obtained Value
|
-2.460 uM
Interval -12.18 to 22.47
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years).Population: Full Analysis Set
Effect of asfotase alfa on PLP as measured by change from Baseline to last assessment for each patient
Outcome measures
| Measure |
Asfotase Alfa
n=56 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Effect of Asfotase Alfa on Biomarkers - Plasma Pyridoxal-5' Phosphate (PLP) Change From Baseline to Last Obtained Value
|
-395.40 ng/mL
Interval -23836.0 to 2400.0
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years).Population: Full Analysis Set
Effect of asfotase alfa on serum PTH as measured by change from Baseline to last assessment for each patient
Outcome measures
| Measure |
Asfotase Alfa
n=47 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Effect of Asfotase Alfa on Serum Parathyroid Hormone (PTH) - Change From Baseline to Last Obtained Value
|
0.70 pmol/L
Interval -4.7 to 5.7
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 72 months or until regulatory approval in the country of residence. Patients received study drug for a median duration of 829.0 days, with a range from 6 to 2116 days (ie, from 0.9 week to 5.8 years).Population: Full Analysis Set
Effect of asfotase alfa treatment on tooth loss assessed by the proportion of patients who experienced tooth loss during the study
Outcome measures
| Measure |
Asfotase Alfa
n=69 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Effect of Asfotase Alfa Treatment on Tooth Loss
|
42 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: PK parameters were calculated using Week 6 study visit data. Week 6 study visit blood samples for PK testing were drawn pre-dose and 6, 12, 24, 32, and 48 hours post dosePopulation: \[14 out of the total 69 subjects had provided sufficient concentration-time data for PK analysis and estimating the PK parameters\]
The PK properties (tlast) of asfotase alfa
Outcome measures
| Measure |
Asfotase Alfa
n=14 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Pharmacokinetic (PK) Properties of Asfotase Alfa (Tlast)
|
48.1 hours
Standard Deviation 0.1
|
—
|
—
|
SECONDARY outcome
Timeframe: PK parameters were calculated using Week 6 study visit data. Week 6 study visit blood samples for PK testing were drawn pre-dose and 6, 12, 24, 32, and 48 hours post dosePopulation: 14 out of the total 69 subjects had provided sufficient concentration-time data for PK analysis and estimating the PK parameters
The PK properties (tmax) of asfotase alfa
Outcome measures
| Measure |
Asfotase Alfa
n=14 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Pharmacokinetic (PK) Properties of Asfotase Alfa (Tmax)
|
14.9 hours
Standard Deviation 10.4
|
—
|
—
|
SECONDARY outcome
Timeframe: PK parameters were calculated using Week 6 study visit data. Week 6 study visit blood samples for PK testing were drawn pre-dose and 6, 12, 24, 32, and 48 hours post dosePopulation: 14 out of the total 69 subjects had provided sufficient concentration-time data for PK analysis and estimating the PK parameters
The PK properties (Cmax) of asfotase alfa
Outcome measures
| Measure |
Asfotase Alfa
n=14 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Pharmacokinetic (PK) Properties of Asfotase Alfa (Cmax)
|
1794 ng/mL
Standard Deviation 690
|
—
|
—
|
SECONDARY outcome
Timeframe: PK parameters were calculated using Week 6 study visit data. Week 6 study visit blood samples for PK testing were drawn pre-dose and 6, 12, 24, 32, and 48 hours post dosePopulation: 14 out of the total 69 subjects had provided sufficient concentration-time data for PK analysis and estimating the PK parameters.
The PK properties (AUCt) of asfotase alfa
Outcome measures
| Measure |
Asfotase Alfa
n=14 Participants
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
Asfotase Alfa - Without Respiratory Support at Baseline
Results at End of Study for Those Without Respiratory Support at Baseline (Last Assessment for Each Patient)
|
Asfotase Alfa - With Respiratory Support at Baseline
Results at End of Study for Those With Respiratory Support at Baseline (Last Assessment for Each Patient)
|
|---|---|---|---|
|
Pharmacokinetic (PK) Properties of Asfotase Alfa (AUCt)
|
66042 h*ng/mL
Standard Deviation 25758
|
—
|
—
|
Adverse Events
Asfotase Alfa
Serious events: 50 serious events
Other events: 68 other events
Deaths: 9 deaths
Serious adverse events
| Measure |
Asfotase Alfa
n=69 participants at risk
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
|---|---|
|
Infections and infestations
Pneumonia
|
11.6%
8/69 • Number of events 9 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Pneumonia viral
|
4.3%
3/69 • Number of events 3 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Device related infection
|
2.9%
2/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Gastroenteritis
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Gastroenteritis rotavirus
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Respiratory syncytial virus infection
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Rhinovirus infection
|
2.9%
2/69 • Number of events 3 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Sepsis
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Upper respiratory tract infection
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Viral infection
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Viral upper respiratory tract infection
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Adenoviral upper respiratory infection
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Bacteraemia
|
1.4%
1/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Beta haemolytic streptococcal infection
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Bronchiolitis
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Bronchitis
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Clostridium difficile colitis
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Corona virus infection
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Enterobacter infection
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Gastroenteritis viral
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Influenza
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Lower respiratory tract infection
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Meningitis staphylococcal
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Otitis media
|
1.4%
1/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Parainfluenzae virus infection
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Postoperative wound infection
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Pseudomonas infection
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Respiratory tract infection
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Respiratory tract infection viral
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Serratia infection
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Serratia sepsis
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Staphylococcal infection
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Staphylococcal sepsis
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Stenotrophomonas infection
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
5.8%
4/69 • Number of events 6 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Apnoea
|
4.3%
3/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
4.3%
3/69 • Number of events 4 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
4.3%
3/69 • Number of events 3 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
2.9%
2/69 • Number of events 3 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
2.9%
2/69 • Number of events 8 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Adenoidal hypertrophy
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Collapse of lung
|
1.4%
1/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
1.4%
1/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Obstructive airways disorder
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory arrest
|
1.4%
1/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract inflammation
|
1.4%
1/69 • Number of events 3 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Congenital, familial and genetic disorders
Craniosynostosis
|
18.8%
13/69 • Number of events 13 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Congenital, familial and genetic disorders
Arnold-Chiari malformation
|
4.3%
3/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Congenital, familial and genetic disorders
Hypophosphatasia
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Hydrocephalus
|
4.3%
3/69 • Number of events 4 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Intracranial pressure increased
|
4.3%
3/69 • Number of events 3 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Syringomyelia
|
4.3%
3/69 • Number of events 3 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Convulsion
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Headache
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Brain oedema
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Encephalitis
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Febrile convulsion
|
1.4%
1/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Haemorrhagic stroke
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Intracranial hypotension
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Motor developmental delay
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Motor dysfunction
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Neurological symptom
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Petit mal epilepsy
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Oxygen saturation decreased
|
5.8%
4/69 • Number of events 14 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Aspiration tracheal
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
CSF pressure
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Enterovirus test positive
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Hepatic enzyme increased
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Human rhinovirus test positive
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Neurological examination abnormal
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Nuclear magnetic resonance imaging
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Respiratory rate increased
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Respiratory syncytial virus test positive
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Weight decreased
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Pyrexia
|
8.7%
6/69 • Number of events 14 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Irritability
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Chills
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Device dislocation
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Injection site erythema
|
1.4%
1/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Oedema
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Femur fracture
|
4.3%
3/69 • Number of events 3 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Feeding tube complication
|
2.9%
2/69 • Number of events 6 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Abdominal wound dehiscence
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Brain herniation
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Endotracheal intubation complication
|
1.4%
1/69 • Number of events 3 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Eye injury
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Humerus fracture
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Incorrect dose administered
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Rib fracture
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Food intolerance
|
5.8%
4/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Failure to thrive
|
2.9%
2/69 • Number of events 3 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Feeding disorder
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Dehydration
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Dystrophic calcification
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Fluid overload
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Hypernatraemia
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Hyperphosphataemia
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Hypophagia
|
1.4%
1/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Hypervolaemia
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Cardiac disorders
Bradycardia
|
4.3%
3/69 • Number of events 10 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Cardiac disorders
Cardio-respiratory arrest
|
2.9%
2/69 • Number of events 4 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Cardiac disorders
Cardiac arrest
|
4.3%
3/69 • Number of events 4 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Cardiac disorders
Cyanosis
|
2.9%
2/69 • Number of events 3 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Cardiac disorders
Arrhythmia
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Cardiac disorders
Cardiopulmonary failure
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Cardiac disorders
Wolff-Parkinson-White syndrome
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Vomiting
|
4.3%
3/69 • Number of events 4 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Inguinal hernia
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Pancreatitis acute
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Pneumatosis intestinalis
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Rectal prolapse
|
1.4%
1/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Musculoskeletal and connective tissue disorders
Osteopenia
|
2.9%
2/69 • Number of events 3 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Musculoskeletal and connective tissue disorders
Joint contracture
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Musculoskeletal and connective tissue disorders
Pathological fracture
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Surgical and medical procedures
Cranial operation
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Surgical and medical procedures
Medical device removal
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Surgical and medical procedures
Tracheal operation
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Surgical and medical procedures
Tracheostomy
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Surgical and medical procedures
Wean from ventilator
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Immune system disorders
Drug hypersensitivity
|
2.9%
2/69 • Number of events 2 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Immune system disorders
Anaphylactoid reaction
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Eye disorders
Conjunctivitis
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Eye disorders
Optic neuropathy
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Blood and lymphatic system disorders
Coagulopathy
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Endocrine disorders
Hyperparathyroidism tertiary
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Psychiatric disorders
Breath holding
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Psychiatric disorders
Staring
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Renal and urinary disorders
Renal failure
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Vascular disorders
Deep vein thrombosis
|
1.4%
1/69 • Number of events 1 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
Other adverse events
| Measure |
Asfotase Alfa
n=69 participants at risk
A total of 6 mg/kg/week of asfotase alfa administered by SC injection (either 1 mg/kg asfotase alfa 6 times per week, or 2 mg/kg asfotase alfa 3 times per week)
|
|---|---|
|
Infections and infestations
Nasopharyngitis
|
26.1%
18/69 • Number of events 52 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Upper respiratory tract infection
|
24.6%
17/69 • Number of events 31 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Gastroenteritis
|
23.2%
16/69 • Number of events 20 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Respiratory tract infection
|
23.2%
16/69 • Number of events 62 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Ear infection
|
18.8%
13/69 • Number of events 18 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Rhinitis
|
14.5%
10/69 • Number of events 17 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Bronchitis
|
13.0%
9/69 • Number of events 16 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Urinary tract infection
|
11.6%
8/69 • Number of events 8 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Influenza
|
10.1%
7/69 • Number of events 8 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Pneumonia
|
10.1%
7/69 • Number of events 9 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Device related infection
|
7.2%
5/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Hand-foot-and-mouth disease
|
7.2%
5/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Otitis media
|
7.2%
5/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Tonsillitis
|
7.2%
5/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Gastroenteritis viral
|
5.8%
4/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Gastrointestinal infection
|
5.8%
4/69 • Number of events 9 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Infections and infestations
Viral upper respiratory tract infection
|
5.8%
4/69 • Number of events 4 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Pyrexia
|
68.1%
47/69 • Number of events 131 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Injection site erythema
|
47.8%
33/69 • Number of events 223 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Injection site discolouration
|
17.4%
12/69 • Number of events 43 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Injection site induration
|
15.9%
11/69 • Number of events 44 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Injection site haematoma
|
14.5%
10/69 • Number of events 22 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Injection site pain
|
11.6%
8/69 • Number of events 22 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Injection site reaction
|
11.6%
8/69 • Number of events 13 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Injection site swelling
|
10.1%
7/69 • Number of events 21 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Injection site pruritus
|
8.7%
6/69 • Number of events 14 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Catheter site erythema
|
7.2%
5/69 • Number of events 16 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Injection site papule
|
7.2%
5/69 • Number of events 91 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Injection site rash
|
7.2%
5/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Catheter site inflammation
|
5.8%
4/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Device occlusion
|
5.8%
4/69 • Number of events 8 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Injection site atrophy
|
5.8%
4/69 • Number of events 15 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
General disorders
Irritability
|
5.8%
4/69 • Number of events 6 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Tooth loss
|
59.4%
41/69 • Number of events 109 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Vomiting
|
43.5%
30/69 • Number of events 92 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Diarrhoea
|
29.0%
20/69 • Number of events 35 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Constipation
|
23.2%
16/69 • Number of events 37 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
13.0%
9/69 • Number of events 10 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Abdominal pain
|
10.1%
7/69 • Number of events 7 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Dental caries
|
10.1%
7/69 • Number of events 11 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Teething
|
7.2%
5/69 • Number of events 10 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Gastrointestinal disorders
Toothache
|
7.2%
5/69 • Number of events 8 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Contusion
|
14.5%
10/69 • Number of events 21 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Fall
|
10.1%
7/69 • Number of events 9 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
7.2%
5/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Procedural pain
|
7.2%
5/69 • Number of events 6 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Rib fracture
|
7.2%
5/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Injury, poisoning and procedural complications
Tooth fracture
|
5.8%
4/69 • Number of events 4 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
24.6%
17/69 • Number of events 34 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
11.6%
8/69 • Number of events 30 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Use of accessory respiratory muscles
|
10.1%
7/69 • Number of events 16 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
8.7%
6/69 • Number of events 12 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
8.7%
6/69 • Number of events 7 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnoea
|
8.7%
6/69 • Number of events 7 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
7.2%
5/69 • Number of events 6 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.8%
4/69 • Number of events 6 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
5.8%
4/69 • Number of events 4 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Respiratory, thoracic and mediastinal disorders
Tracheomalacia
|
5.8%
4/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Skin and subcutaneous tissue disorders
Rash
|
15.9%
11/69 • Number of events 23 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
14.5%
10/69 • Number of events 18 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
11.6%
8/69 • Number of events 10 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Skin and subcutaneous tissue disorders
Eczema
|
10.1%
7/69 • Number of events 14 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Skin and subcutaneous tissue disorders
Erythema
|
8.7%
6/69 • Number of events 8 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Skin and subcutaneous tissue disorders
Excessive granulation tissue
|
7.2%
5/69 • Number of events 6 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Skin and subcutaneous tissue disorders
Granuloma skin
|
5.8%
4/69 • Number of events 7 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Skin and subcutaneous tissue disorders
Heat rash
|
5.8%
4/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Alanine aminotransferase increased
|
8.7%
6/69 • Number of events 8 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Aspartate aminotransferase increased
|
7.2%
5/69 • Number of events 6 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Oxygen saturation decreased
|
7.2%
5/69 • Number of events 54 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Investigations
Vitamin D decreased
|
5.8%
4/69 • Number of events 6 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
8.7%
6/69 • Number of events 8 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
8.7%
6/69 • Number of events 6 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
8.7%
6/69 • Number of events 7 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Metabolism and nutrition disorders
Food intolerance
|
5.8%
4/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
15.9%
11/69 • Number of events 13 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Musculoskeletal and connective tissue disorders
Kyphosis
|
8.7%
6/69 • Number of events 10 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Musculoskeletal and connective tissue disorders
Knee deformity
|
7.2%
5/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Musculoskeletal and connective tissue disorders
Bone disorder
|
5.8%
4/69 • Number of events 23 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Eye disorders
Conjunctivitis
|
11.6%
8/69 • Number of events 16 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Eye disorders
Papilloedema
|
7.2%
5/69 • Number of events 8 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Headache
|
14.5%
10/69 • Number of events 22 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Nervous system disorders
Cerebral ventricle dilatation
|
5.8%
4/69 • Number of events 6 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Congenital, familial and genetic disorders
Craniosynostosis
|
14.5%
10/69 • Number of events 11 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Congenital, familial and genetic disorders
Congenital bowing of long bones
|
7.2%
5/69 • Number of events 6 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Blood and lymphatic system disorders
Anaemia
|
13.0%
9/69 • Number of events 13 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
7.2%
5/69 • Number of events 7 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Blood and lymphatic system disorders
Neutropenia
|
5.8%
4/69 • Number of events 4 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Renal and urinary disorders
Nephrocalcinosis
|
7.2%
5/69 • Number of events 5 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Psychiatric disorders
Agitation
|
5.8%
4/69 • Number of events 11 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Ear and labyrinth disorders
Cerumen impaction
|
7.2%
5/69 • Number of events 11 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Cardiac disorders
Tachycardia
|
8.7%
6/69 • Number of events 16 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Cardiac disorders
Bradycardia
|
5.8%
4/69 • Number of events 7 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Vascular disorders
Hypertension
|
5.8%
4/69 • Number of events 4 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
|
Hepatobiliary disorders
Hepatomegaly
|
5.8%
4/69 • Number of events 4 • Starting after signing of informed consent through 30 days after the EOS visit, approximately 6 years
|
Additional Information
Director of Clinical Trials
Alexion Pharmaceuticals, Inc.
Phone: 475-230-2596
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60