Trial Outcomes & Findings for A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) (NCT NCT01242111)
NCT ID: NCT01242111
Last Updated: 2015-09-30
Results Overview
The primary objective of the study is to evaluate the safety of weekly infusions of BMN 110; the safety variables included Adverse Events (AEs). The primary outcome measure data is presented in more detail under the Adverse Events section.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
20 participants
Primary outcome timeframe
Entire Study Period, up to 240 weeks
Results posted on
2015-09-30
Participant Flow
Participant milestones
| Measure |
BMN 110
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
17
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
BMN 110
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Did not consent to MOR-100 follow-up
|
1
|
Baseline Characteristics
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Baseline characteristics by cohort
| Measure |
BMN 110
n=20 Participants
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
|
|---|---|
|
Age, Continuous
|
8.4 years
STANDARD_DEVIATION 2.90 • n=93 Participants
|
|
Age, Customized
>=4 to < 8 years
|
10 participants
n=93 Participants
|
|
Age, Customized
>=8 to <10 years
|
5 participants
n=93 Participants
|
|
Age, Customized
>=10 to <=18 years
|
5 participants
n=93 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
20 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Asian
|
9 participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 participants
n=93 Participants
|
|
Race/Ethnicity, Customized
White
|
9 participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Other
|
2 participants
n=93 Participants
|
|
Region of Enrollment
United Kingdom
|
20 participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Entire Study Period, up to 240 weeksPopulation: The primary outcome measure data is presented in more detail under the Adverse Events section.
The primary objective of the study is to evaluate the safety of weekly infusions of BMN 110; the safety variables included Adverse Events (AEs). The primary outcome measure data is presented in more detail under the Adverse Events section.
Outcome measures
| Measure |
BMN 110
n=20 Participants
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
|
|---|---|
|
Safety Evaluation
Serious Adverse Events
|
19 participants
|
|
Safety Evaluation
Other (Not Including Serious) Adverse Events
|
20 participants
|
SECONDARY outcome
Timeframe: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trialPopulation: Intent-to-Treat population (all subjects who enrolled in the study). Two patients were either physically (score was designated as 0 m) or developmentally (score was set to missing) unable to perform the 6MWT. The analysis was based on observed cases.
Change from baseline in meters in 6-minute Walk Test. As a measure of endurance, a 6-minute walk test (6MWT) was performed according to the American Thoracic Society Guidelines. Patients were instructed to walk as far as possible in 6 minutes.
Outcome measures
| Measure |
BMN 110
n=20 Participants
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
|
|---|---|
|
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Week 72 Change from Baseline (n=17)
|
4.0 meters
Standard Deviation 87.24
|
|
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 0 Change from Baseline (n=16)
|
15.7 meters
Standard Deviation 89.00
|
|
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 24 Change from Baseline (n=16)
|
24.5 meters
Standard Deviation 101.23
|
|
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 48 Change from Baseline (n=16)
|
6.8 meters
Standard Deviation 98.66
|
|
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 96 Change from Baseline (n=16)
|
11.2 meters
Standard Deviation 85.24
|
|
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 120 Change from Baseline (n=16)
|
4.2 meters
Standard Deviation 94.09
|
|
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 144 Change from Baseline (n=13)
|
3.1 meters
Standard Deviation 106.82
|
|
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 192 Change from Baseline (n=9)
|
-37.1 meters
Standard Deviation 103.96
|
|
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Week 12 Change from Baseline (n=19)
|
-20.7 meters
Standard Deviation 85.95
|
|
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Week 24 Change from Baseline (n=17)
|
16.3 meters
Standard Deviation 71.74
|
|
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Week 36 Change from Baseline (n=17)
|
13.8 meters
Standard Deviation 63.25
|
|
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Week 48 Change from Baseline (n=17)
|
-4.8 meters
Standard Deviation 64.70
|
|
Change From Baseline in Endurance as Measured by the 6-minute Walk Test During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 72 Change from Baseline (n=17)
|
-49.8 meters
Standard Deviation 132.63
|
SECONDARY outcome
Timeframe: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trialPopulation: Intent-to-Treat population (all subjects who enrolled in the study). One patient was developmentally unable to perform the 3MSCT and the test scores were set to missing. The analysis was based on observed cases.
Change from baseline in the 3-minute Stair Climb Test (3MSCT). Patients walked up stairs that have a railing, which could be used for support, for 3 minutes, with the number of stairs climbed recorded. The test result was the number of steps climbed per minute.
Outcome measures
| Measure |
BMN 110
n=20 Participants
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
|
|---|---|
|
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Week 12 Change from Baseline (n=19)
|
0.3 steps/min
Standard Deviation 14.07
|
|
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Week 24 Change from Baseline (n=17)
|
6.1 steps/min
Standard Deviation 8.66
|
|
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Week 36 Change from Baseline (n=17)
|
7.8 steps/min
Standard Deviation 13.69
|
|
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Week 48 Change from Baseline (n=17)
|
9.7 steps/min
Standard Deviation 14.42
|
|
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Week 72 Change from Baseline (n=17)
|
9.7 steps/min
Standard Deviation 13.91
|
|
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 0 Change from Baseline (n=16)
|
12.7 steps/min
Standard Deviation 13.96
|
|
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 24 Change from Baseline (n=16)
|
13.4 steps/min
Standard Deviation 17.07
|
|
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 96 Change from Baseline (n=16)
|
9.9 steps/min
Standard Deviation 18.84
|
|
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 120 Change from Baseline (n=16)
|
6.2 steps/min
Standard Deviation 14.41
|
|
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 144 Change from Baseline (n=12)
|
5.4 steps/min
Standard Deviation 11.93
|
|
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 192 Change from Baseline (n=7)
|
-0.2 steps/min
Standard Deviation 10.34
|
|
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 48 Change from Baseline (n=16)
|
6.6 steps/min
Standard Deviation 16.87
|
|
Change in Baseline in Endurance as Measured by the 3 Minute Stair Climb During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Week 72 Change from Baseline (n=16)
|
-1.4 steps/min
Standard Deviation 21.11
|
SECONDARY outcome
Timeframe: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 168 weeks during the MOR-100 extension trialPopulation: Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases.
Percent Change from baseline in Normalized Urine KS. The percent change was calculated (Week X value - baseline value)/baseline value \*100%
Outcome measures
| Measure |
BMN 110
n=20 Participants
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
|
|---|---|
|
Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Wk 24 Percent Change from Baseline (n=18)
|
-27.9 percentage change
Standard Deviation 17.92
|
|
Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Wk 36 Percent Change from Baseline (n=18)
|
-40.6 percentage change
Standard Deviation 20.16
|
|
Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Wk 72 Percent Change from Baseline (n=17)
|
-32.2 percentage change
Standard Deviation 17.10
|
|
Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 0 Percent Change from Baseline (n=17)
|
-30.0 percentage change
Standard Deviation 19.23
|
|
Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 24 Percent Change from Baseline (n=17)
|
-43.6 percentage change
Standard Deviation 19.56
|
|
Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 48 Percent Change from Baseline (n=16)
|
-41.9 percentage change
Standard Deviation 19.29
|
|
Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 72 Percent Change from Baseline (n=12)
|
-36.4 percentage change
Standard Deviation 36.70
|
|
Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 96 Percent Change from Baseline (n=17)
|
-49.7 percentage change
Standard Deviation 19.93
|
|
Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Wk 12 Percent Change from Baseline (n=19)
|
-23.2 percentage change
Standard Deviation 19.04
|
|
Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 120 Percent Change from Baseline (n=16)
|
-49.3 percentage change
Standard Deviation 22.28
|
|
Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 144 Percent Change from Baseline (n=16)
|
-56.6 percentage change
Standard Deviation 19.54
|
|
Percent Change From Baseline in Urine Keratan Sulfate (uKS) Levels During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 168 Percent Change from Baseline (n=15)
|
-58.9 percentage change
Standard Deviation 16.02
|
SECONDARY outcome
Timeframe: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trialPopulation: Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases.
Percent Change from baseline in Maximum Voluntary Ventilation.
Outcome measures
| Measure |
BMN 110
n=20 Participants
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
|
|---|---|
|
Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Wk 12 Percent Change from Baseline (n=14)
|
9.9 percentage change
Standard Deviation 21.29
|
|
Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Wk 24 Percent Change from Baseline (n=13)
|
11.0 percentage change
Standard Deviation 21.48
|
|
Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Wk 36 Percent Change from Baseline (n=14)
|
10.5 percentage change
Standard Deviation 17.43
|
|
Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Wk 72 Percent Change from Baseline (n=14)
|
18.4 percentage change
Standard Deviation 20.77
|
|
Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 0 Percent Change from Baseline (n=13)
|
11.1 percentage change
Standard Deviation 16.44
|
|
Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 24 Percent Change from Baseline (n=13)
|
9.8 percentage change
Standard Deviation 22.25
|
|
Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 48 Percent Change from Baseline (n=12)
|
3.5 percentage change
Standard Deviation 17.78
|
|
Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 72 Percent Change from Baseline (n=13)
|
10.1 percentage change
Standard Deviation 27.83
|
|
Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 96 Percent Change from Baseline (n=12)
|
-4.9 percentage change
Standard Deviation 35.53
|
|
Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 120 Percent Change from Baseline (n=13)
|
-3.3 percentage change
Standard Deviation 28.35
|
|
Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 144 Percent Change from Baseline (n=11)
|
-3.5 percentage change
Standard Deviation 34.61
|
|
Percent Change From Baseline in Respiratory Function Test MVV During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 192 Percent Change from Baseline (n=6)
|
28.2 percentage change
Standard Deviation 38.78
|
SECONDARY outcome
Timeframe: Baseline and every 12 weeks for up to 72 weeks during the MOR-002 pilot trial and every 24 weeks for up to 192 weeks during the MOR-100 extension trialPopulation: Intent-to-Treat population (all subjects who enrolled in the study). The analysis was based on observed cases.
Percent Change from baseline in Forced Vital Capacity.
Outcome measures
| Measure |
BMN 110
n=20 Participants
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
|
|---|---|
|
Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 120 Percent Change from Baseline (n=16)
|
22.8 percentage change
Standard Deviation 21.14
|
|
Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 144 Percent Change from Baseline (n=12)
|
17.5 percentage change
Standard Deviation 24.32
|
|
Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 192 Percent Change from Baseline (n=7)
|
18.6 percentage change
Standard Deviation 30.98
|
|
Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Wk 12 Percent Change from Baseline (n=18)
|
3.4 percentage change
Standard Deviation 10.85
|
|
Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Wk 24 Percent Change from Baseline (n=16)
|
0.2 percentage change
Standard Deviation 16.60
|
|
Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Wk 36 Percent Change from Baseline (n=16)
|
10.7 percentage change
Standard Deviation 20.81
|
|
Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-002 Wk 72 Percent Change from Baseline (n=16)
|
12.5 percentage change
Standard Deviation 14.88
|
|
Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 0 Percent Change from Baseline (n=15)
|
11.8 percentage change
Standard Deviation 14.97
|
|
Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 24 Percent Change from Baseline (n=14)
|
15.3 percentage change
Standard Deviation 16.31
|
|
Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 48 Percent Change from Baseline (n=13)
|
15.8 percentage change
Standard Deviation 16.56
|
|
Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 72 Percent Change from Baseline (n=15)
|
16.1 percentage change
Standard Deviation 21.96
|
|
Percent Change From Baseline in Respiratory Function Test FVC During the Pilot Trial (MOR-002) and Current Extension Trial (MOR-100).
MOR-100 Wk 96 Percent Change from Baseline (n=14)
|
14.8 percentage change
Standard Deviation 17.36
|
Adverse Events
BMN 110
Serious events: 19 serious events
Other events: 20 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
BMN 110
n=20 participants at risk
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
|
|---|---|
|
Cardiac disorders
Supraventricular tachycardia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Cardiac disorders
Tachycardia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Gait disturbance
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Infusion site inflammation
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Infusion site reaction
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
General disorders
Malaise
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Pyrexia
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Immune system disorders
Type I hypersensitivity
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Abdominal abscess
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Abscess limb
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Catheter site infection
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Device related infection
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Infections and infestations
Infusion site infection
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Lobar pneumonia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Lower respiratory tract infection
|
10.0%
2/20 • Number of events 5 • Study Period, up to 240 weeks
|
|
Infections and infestations
Otitis media
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Infections and infestations
Pneumonia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Pneumonia mycoplasmal
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Respiratory syncytial virus infection
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Viral infection
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
5.0%
1/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Procedural pain
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Skull fracture
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Knee deformity
|
30.0%
6/20 • Number of events 7 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Nervous system disorders
Cervical cord compression
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Psychiatric disorders
Panic attack
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnoea syndrome
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.0%
1/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Surgical and medical procedures
Abscess drainage
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Surgical and medical procedures
Bone operation
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Surgical and medical procedures
Central venous catheterisation
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Surgical and medical procedures
Knee operation
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Surgical and medical procedures
Medical device implantation
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Surgical and medical procedures
Medical device removal
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Surgical and medical procedures
Orthopaedic procedure
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Vascular disorders
Flushing
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Vascular disorders
Hypertension
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Vascular disorders
Jugular vein distension
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Vascular disorders
Poor venous access
|
40.0%
8/20 • Number of events 9 • Study Period, up to 240 weeks
|
Other adverse events
| Measure |
BMN 110
n=20 participants at risk
BMN 110: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.
|
|---|---|
|
Blood and lymphatic system disorders
Eosinophilia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
20.0%
4/20 • Number of events 5 • Study Period, up to 240 weeks
|
|
Cardiac disorders
Aortic valve incompetence
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Cardiac disorders
Diastolic dysfunction
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Cardiac disorders
Mitral valve disease
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Cardiac disorders
Mitral valve incompetence
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Cardiac disorders
Pulmonary valve incompetence
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Cardiac disorders
Tachycardia
|
15.0%
3/20 • Number of events 6 • Study Period, up to 240 weeks
|
|
Cardiac disorders
Tricuspid valve incompetence
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Congenital, familial and genetic disorders
Congenital heart valve disorder
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Cerumen impaction
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Deafness
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Deafness neurosensory
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Ear canal erythema
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Ear discomfort
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Ear disorder
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Ear pain
|
60.0%
12/20 • Number of events 41 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Ear pruritus
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Hyperacusis
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Hypoacusis
|
10.0%
2/20 • Number of events 4 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Middle ear disorder
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Motion sickness
|
20.0%
4/20 • Number of events 9 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Otorrhoea
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Tinnitus
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Tympanic membrane disorder
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Tympanic membrane hyperaemia
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Ear and labyrinth disorders
Tympanic membrane perforation
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Endocrine disorders
Autoimmune thyroiditis
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Eye disorders
Conjunctivitis
|
15.0%
3/20 • Number of events 4 • Study Period, up to 240 weeks
|
|
Eye disorders
Dry eye
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Eye disorders
Eye discharge
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Eye disorders
Eye pain
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Eye disorders
Eye pruritus
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Eye disorders
Eye swelling
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Eye disorders
Eyelid cyst
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Eye disorders
Ocular hyperaemia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Abdominal discomfort
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Abdominal pain
|
60.0%
12/20 • Number of events 27 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Abdominal pain upper
|
55.0%
11/20 • Number of events 33 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Constipation
|
25.0%
5/20 • Number of events 5 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Dental caries
|
15.0%
3/20 • Number of events 5 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Dental plaque
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Diarrhoea
|
60.0%
12/20 • Number of events 28 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Dyspepsia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Faecal incontinence
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Gingival hyperpigmentation
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Glossodynia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Haematemesis
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Lip disorder
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Lip pain
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Mouth ulceration
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Nausea
|
25.0%
5/20 • Number of events 8 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Retching
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Tongue discolouration
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Tooth deposit
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Tooth disorder
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Toothache
|
25.0%
5/20 • Number of events 8 • Study Period, up to 240 weeks
|
|
Gastrointestinal disorders
Vomiting
|
80.0%
16/20 • Number of events 92 • Study Period, up to 240 weeks
|
|
General disorders
Application site reaction
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Application site vesicles
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Catheter site erythema
|
15.0%
3/20 • Number of events 9 • Study Period, up to 240 weeks
|
|
General disorders
Catheter site extravasation
|
35.0%
7/20 • Number of events 12 • Study Period, up to 240 weeks
|
|
General disorders
Catheter site inflammation
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Catheter site oedema
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Catheter site pain
|
20.0%
4/20 • Number of events 5 • Study Period, up to 240 weeks
|
|
General disorders
Catheter site rash
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Catheter site related reaction
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
General disorders
Catheter site scab
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Catheter site swelling
|
10.0%
2/20 • Number of events 6 • Study Period, up to 240 weeks
|
|
General disorders
Chest pain
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
General disorders
Chills
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Device connection issue
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Device dislocation
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
General disorders
Device expulsion
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
General disorders
Device leakage
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Device malfunction
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Device occlusion
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
General disorders
Extravasation
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
General disorders
Fatigue
|
35.0%
7/20 • Number of events 11 • Study Period, up to 240 weeks
|
|
General disorders
Feeling hot
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
General disorders
Gait disturbance
|
15.0%
3/20 • Number of events 4 • Study Period, up to 240 weeks
|
|
General disorders
Influenza like illness
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
General disorders
Infusion site erythema
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Infusion site extravasation
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
General disorders
Infusion site pain
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Infusion site reaction
|
5.0%
1/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
General disorders
Injection site reaction
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Local swelling
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
General disorders
Malaise
|
20.0%
4/20 • Number of events 8 • Study Period, up to 240 weeks
|
|
General disorders
Medical device complication
|
10.0%
2/20 • Number of events 4 • Study Period, up to 240 weeks
|
|
General disorders
Medical device site reaction
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
General disorders
Pain
|
30.0%
6/20 • Number of events 12 • Study Period, up to 240 weeks
|
|
General disorders
Pyrexia
|
90.0%
18/20 • Number of events 103 • Study Period, up to 240 weeks
|
|
General disorders
Vaccination site pain
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Hepatobiliary disorders
Hepatomegaly
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Immune system disorders
Drug hypersensitivity
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Immune system disorders
Seasonal allergy
|
25.0%
5/20 • Number of events 8 • Study Period, up to 240 weeks
|
|
Infections and infestations
Abdominal abscess
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Catheter site infection
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Ear infection
|
30.0%
6/20 • Number of events 15 • Study Period, up to 240 weeks
|
|
Infections and infestations
Eye infection
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Infections and infestations
Fungal infection
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Fungal skin infection
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Infections and infestations
Furuncle
|
15.0%
3/20 • Number of events 4 • Study Period, up to 240 weeks
|
|
Infections and infestations
Gastroenteritis viral
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Gingivitis
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Infections and infestations
Helminthic infection
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Herpes simplex
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Infections and infestations
Herpes zoster
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Infections and infestations
Impetigo
|
5.0%
1/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Infections and infestations
Infection
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Infections and infestations
Influenza
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Lice infestation
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Infections and infestations
Localised infection
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Infections and infestations
Lower respiratory tract infection
|
25.0%
5/20 • Number of events 13 • Study Period, up to 240 weeks
|
|
Infections and infestations
Molluscum contagiosum
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Infections and infestations
Nail infection
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Nasopharyngitis
|
85.0%
17/20 • Number of events 53 • Study Period, up to 240 weeks
|
|
Infections and infestations
Oral candidiasis
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Otitis externa
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Otitis media
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Infections and infestations
Paronychia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Pharyngitis
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Postoperative wound infection
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Rash pustular
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Rhinitis
|
25.0%
5/20 • Number of events 8 • Study Period, up to 240 weeks
|
|
Infections and infestations
Skin infection
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Infections and infestations
Subcutaneous abscess
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Tinea pedis
|
20.0%
4/20 • Number of events 4 • Study Period, up to 240 weeks
|
|
Infections and infestations
Tonsillitis
|
15.0%
3/20 • Number of events 5 • Study Period, up to 240 weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
25.0%
5/20 • Number of events 5 • Study Period, up to 240 weeks
|
|
Infections and infestations
Urinary tract infection
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Infections and infestations
Varicella
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Viral infection
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Viral upper respiratory tract infection
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Infections and infestations
Wound infection
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
20.0%
4/20 • Number of events 5 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Arthropod sting
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Contusion
|
30.0%
6/20 • Number of events 8 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Ear injury
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Excoriation
|
20.0%
4/20 • Number of events 4 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Eye contusion
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Face injury
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Fall
|
40.0%
8/20 • Number of events 20 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Head injury
|
40.0%
8/20 • Number of events 14 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Injury
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Laceration
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Limb injury
|
15.0%
3/20 • Number of events 4 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Lip injury
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Neck injury
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Occupational exposure to radiation
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Periorbital contusion
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Post procedural complication
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Procedural headache
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Procedural pain
|
35.0%
7/20 • Number of events 7 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Procedural vomiting
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Scar
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Scratch
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Skeletal injury
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Sunburn
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Thermal burn
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Upper limb fracture
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Injury, poisoning and procedural complications
Wound complication
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Alanine aminotransferase increased
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Aspartate aminotransferase increased
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Aspiration joint
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Blood cholesterol increased
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Investigations
Blood immunoglobulin E increased
|
30.0%
6/20 • Number of events 6 • Study Period, up to 240 weeks
|
|
Investigations
Blood immunoglobulin G decreased
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Blood lactate dehydrogenase increased
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Blood pressure systolic increased
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Blood sodium increased
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Body temperature increased
|
20.0%
4/20 • Number of events 6 • Study Period, up to 240 weeks
|
|
Investigations
Breath sounds abnormal
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Cardiac murmur
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Investigations
Computerised tomogram
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Electrocardiogram T wave amplitude decreased
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Investigations
Eosinophil count increased
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Investigations
Eosinophil percentage increased
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Liver palpable subcostal
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Nuclear magnetic resonance imaging
|
30.0%
6/20 • Number of events 8 • Study Period, up to 240 weeks
|
|
Investigations
Nuclear magnetic resonance imaging spinal
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Oxygen saturation decreased
|
5.0%
1/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Investigations
Protein total abnormal
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Protein urine
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Investigations
Respiratory rate increased
|
15.0%
3/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Investigations
Ultrasound scan abnormal
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Investigations
Weight increased
|
10.0%
2/20 • Number of events 4 • Study Period, up to 240 weeks
|
|
Metabolism and nutrition disorders
Appetite disorder
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Metabolism and nutrition disorders
Decreased appetite
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Metabolism and nutrition disorders
Dehydration
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Metabolism and nutrition disorders
Hypercholesterolaemia
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
60.0%
12/20 • Number of events 58 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Atlantoaxial instability
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Axillary mass
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
45.0%
9/20 • Number of events 17 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Bursitis
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Joint instability
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Joint stiffness
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Knee deformity
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Mobility decreased
|
25.0%
5/20 • Number of events 6 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
5.0%
1/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
40.0%
8/20 • Number of events 12 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
85.0%
17/20 • Number of events 76 • Study Period, up to 240 weeks
|
|
Musculoskeletal and connective tissue disorders
Pain in jaw
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Nervous system disorders
Ageusia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Nervous system disorders
Areflexia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Nervous system disorders
Cervical cord compression
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Nervous system disorders
Clonus
|
15.0%
3/20 • Number of events 4 • Study Period, up to 240 weeks
|
|
Nervous system disorders
Dizziness
|
20.0%
4/20 • Number of events 7 • Study Period, up to 240 weeks
|
|
Nervous system disorders
Headache
|
80.0%
16/20 • Number of events 100 • Study Period, up to 240 weeks
|
|
Nervous system disorders
Lethargy
|
25.0%
5/20 • Number of events 5 • Study Period, up to 240 weeks
|
|
Nervous system disorders
Migraine
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Nervous system disorders
Neuralgia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Nervous system disorders
Paraesthesia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Nervous system disorders
Reflexes abnormal
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Nervous system disorders
Spinal cord compression
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Psychiatric disorders
Anxiety
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Psychiatric disorders
Insomnia
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Renal and urinary disorders
Dysuria
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Renal and urinary disorders
Enuresis
|
5.0%
1/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Renal and urinary disorders
Incontinence
|
5.0%
1/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Renal and urinary disorders
Polyuria
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Renal and urinary disorders
Urinary incontinence
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Reproductive system and breast disorders
Balanitis
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Reproductive system and breast disorders
Genital ulceration
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Reproductive system and breast disorders
Menorrhagia
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Reproductive system and breast disorders
Penile pain
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Reproductive system and breast disorders
Perineal ulceration
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Reproductive system and breast disorders
Scrotal erythema
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Reproductive system and breast disorders
Vulvovaginal pruritus
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
90.0%
18/20 • Number of events 81 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dry throat
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
20.0%
4/20 • Number of events 5 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea exertional
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
20.0%
4/20 • Number of events 12 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Nasal obstruction
|
25.0%
5/20 • Number of events 6 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
75.0%
15/20 • Number of events 31 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal disorder
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal erythema
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal oedema
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Rales
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
15.0%
3/20 • Number of events 6 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnoea syndrome
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnoea
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
20.0%
4/20 • Number of events 6 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Acne
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
25.0%
5/20 • Number of events 6 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Eczema
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Eczema asteatotic
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Erythema
|
20.0%
4/20 • Number of events 43 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Lichen planus
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Miliaria
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Onychomadesis
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Pain of skin
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Papule
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Pigmentation disorder
|
30.0%
6/20 • Number of events 14 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
25.0%
5/20 • Number of events 11 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Psoriasis
|
5.0%
1/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
55.0%
11/20 • Number of events 34 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Rash erythematous
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Rash generalised
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
25.0%
5/20 • Number of events 35 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Skin depigmentation
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Skin discolouration
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Skin disorder
|
20.0%
4/20 • Number of events 5 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Skin exfoliation
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Skin mass
|
10.0%
2/20 • Number of events 4 • Study Period, up to 240 weeks
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
10.0%
2/20 • Number of events 53 • Study Period, up to 240 weeks
|
|
Surgical and medical procedures
Catheter removal
|
5.0%
1/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Surgical and medical procedures
Cautery to nose
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Surgical and medical procedures
Central venous catheterisation
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Surgical and medical procedures
Ear tube insertion
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Surgical and medical procedures
Medical device removal
|
20.0%
4/20 • Number of events 4 • Study Period, up to 240 weeks
|
|
Surgical and medical procedures
Thrombectomy
|
5.0%
1/20 • Number of events 1 • Study Period, up to 240 weeks
|
|
Vascular disorders
Flushing
|
20.0%
4/20 • Number of events 8 • Study Period, up to 240 weeks
|
|
Vascular disorders
Hot flush
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
|
Vascular disorders
Hypertension
|
15.0%
3/20 • Number of events 6 • Study Period, up to 240 weeks
|
|
Vascular disorders
Hypotension
|
10.0%
2/20 • Number of events 3 • Study Period, up to 240 weeks
|
|
Vascular disorders
Poor venous access
|
15.0%
3/20 • Number of events 6 • Study Period, up to 240 weeks
|
|
Vascular disorders
Thrombosis
|
10.0%
2/20 • Number of events 2 • Study Period, up to 240 weeks
|
Additional Information
BioMarin Medical Information Services
BioMarin Pharmaceutical Inc.
Phone: 800-983-4587
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee PI cannot publish any data prior to multi-centre publication. If publication related to sub-sets of data it shall make reference to the relevant multi-centre publication. PI agrees to submit material to Sponsor for review at least 60 days prior to publication and incorporate all reasonable comments. During review, Sponsor may request that publication be delayed for up to 6 months from date of first submission to Sponsor in order for Sponsor to take steps to protect its proprietary information.
- Publication restrictions are in place
Restriction type: OTHER