Trial Outcomes & Findings for Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome (NCT NCT01609062)
NCT ID: NCT01609062
Last Updated: 2016-02-01
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
PHASE2
Target enrollment
25 participants
Primary outcome timeframe
Entire Study Period, up to 192 weeks or ETV (early termination visit)
Results posted on
2016-02-01
Participant Flow
Participant milestones
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
|---|---|---|
|
Primary Phase
STARTED
|
15
|
10
|
|
Primary Phase
COMPLETED
|
15
|
10
|
|
Primary Phase
NOT COMPLETED
|
0
|
0
|
|
Extension Phase
STARTED
|
15
|
10
|
|
Extension Phase
COMPLETED
|
0
|
0
|
|
Extension Phase
NOT COMPLETED
|
15
|
10
|
Reasons for withdrawal
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
|---|---|---|
|
Extension Phase
Terminated by Sponsor
|
14
|
9
|
|
Extension Phase
Discontinued drug and then was pregnant
|
1
|
0
|
|
Extension Phase
Anticipated surgery & transition to EAP
|
0
|
1
|
Baseline Characteristics
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
Baseline characteristics by cohort
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=15 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
Total
n=25 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
14.9 years
STANDARD_DEVIATION 9.32 • n=5 Participants
|
12.0 years
STANDARD_DEVIATION 3.16 • n=7 Participants
|
13.7 years
STANDARD_DEVIATION 7.52 • n=5 Participants
|
|
Age, Customized
7 to 11 years
|
9 participants
n=5 Participants
|
5 participants
n=7 Participants
|
14 participants
n=5 Participants
|
|
Age, Customized
12 to 18 years
|
3 participants
n=5 Participants
|
5 participants
n=7 Participants
|
8 participants
n=5 Participants
|
|
Age, Customized
>=19 years
|
3 participants
n=5 Participants
|
0 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
14 participants
n=5 Participants
|
9 participants
n=7 Participants
|
23 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
5 participants
n=5 Participants
|
1 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
0 participants
n=5 Participants
|
3 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=5 Participants
|
5 participants
n=7 Participants
|
14 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Entire Study Period, up to 192 weeks or ETV (early termination visit)Population: 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 Weekly at 2.0 mg/kg/Week
n=15 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
All Subjects
n=25 Participants
Both groups combined
|
|---|---|---|---|
|
Safety Evaluation
Serious Adverse Events
|
3 participants
|
2 participants
|
5 participants
|
|
Safety Evaluation
Other (Not Including Serious) Adverse Events
|
15 participants
|
10 participants
|
25 participants
|
SECONDARY outcome
Timeframe: Baseline, Week 12, 24, and 52Population: Modified Intent-to-Treat Analysis Set
Change from baseline to Week 12, 24, and 52 as measured in distance walked (meters) in 6MWT.
Outcome measures
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=15 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
All Subjects
n=25 Participants
Both groups combined
|
|---|---|---|---|
|
6-minute Walk Test (6MWT)
Week 12
|
14.7 meters
Standard Deviation 30.90
|
-8.9 meters
Standard Deviation 83.52
|
5.3 meters
Standard Deviation 57.55
|
|
6-minute Walk Test (6MWT)
Week 24
|
-13.0 meters
Standard Deviation 54.12
|
0.1 meters
Standard Deviation 34.70
|
-7.8 meters
Standard Deviation 46.94
|
|
6-minute Walk Test (6MWT)
Week 52
|
9.0 meters
Standard Deviation 48.41
|
31.8 meters
Standard Deviation 54.56
|
18.5 meters
Standard Deviation 51.19
|
SECONDARY outcome
Timeframe: Baseline, Week 12, 24, and 52Population: Modified Intent-to-Treat Analysis Set
Change from baseline to Week 12, 24, and 52 as measured in speed (stairs/min) in 3MSCT.
Outcome measures
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=15 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
All Subjects
n=25 Participants
Both groups combined
|
|---|---|---|---|
|
3-minute Stair Climb Test (3MSCT)
Week 12
|
-3.7 stairs/min
Standard Deviation 13.16
|
1.7 stairs/min
Standard Deviation 11.28
|
-1.5 stairs/min
Standard Deviation 12.49
|
|
3-minute Stair Climb Test (3MSCT)
Week 24
|
-2.2 stairs/min
Standard Deviation 11.01
|
12.5 stairs/min
Standard Deviation 15.26
|
3.9 stairs/min
Standard Deviation 14.65
|
|
3-minute Stair Climb Test (3MSCT)
Week 52
|
-4.4 stairs/min
Standard Deviation 9.82
|
10.0 stairs/min
Standard Deviation 16.88
|
1.3 stairs/min
Standard Deviation 14.66
|
SECONDARY outcome
Timeframe: Baseline, Week 12, 24, and 52Population: Modified Intent-to-treat Analysis Set
Respiratory Function was assessed by spirometry in accordance with American Thoracic Society standards. Percent change from baseline to Week 12, 24, and 52 as measured by Maximum Voluntary Ventilation (MVV, L/min) Percent change from baseline to Week 12, 24, and 52 as measured by Forced Vital Capacity (FVC, L)
Outcome measures
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=15 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
All Subjects
n=25 Participants
Both groups combined
|
|---|---|---|---|
|
Respiratory Function Test (MVV and FVC)
MVV Week 12
|
12.8 Percent Change
Standard Deviation 21.77
|
32.4 Percent Change
Standard Deviation 140.73
|
21.7 Percent Change
Standard Deviation 94.00
|
|
Respiratory Function Test (MVV and FVC)
MVV Week 24
|
8.8 Percent Change
Standard Deviation 17.20
|
-0.6 Percent Change
Standard Deviation 27.00
|
4.3 Percent Change
Standard Deviation 22.34
|
|
Respiratory Function Test (MVV and FVC)
MVV Week 52
|
2.2 Percent Change
Standard Deviation 41.24
|
-17.8 Percent Change
Standard Deviation 38.61
|
-6.9 Percent Change
Standard Deviation 40.43
|
|
Respiratory Function Test (MVV and FVC)
FVC Week 12
|
1.7 Percent Change
Standard Deviation 8.77
|
3.2 Percent Change
Standard Deviation 10.63
|
2.3 Percent Change
Standard Deviation 9.39
|
|
Respiratory Function Test (MVV and FVC)
FVC Week 24
|
3.4 Percent Change
Standard Deviation 9.01
|
13.0 Percent Change
Standard Deviation 16.30
|
7.4 Percent Change
Standard Deviation 13.16
|
|
Respiratory Function Test (MVV and FVC)
FVC Week 52
|
4.8 Percent Change
Standard Deviation 12.68
|
15.1 Percent Change
Standard Deviation 15.94
|
8.9 Percent Change
Standard Deviation 14.69
|
SECONDARY outcome
Timeframe: Baseline, Week 12, 24, and 52Population: Modified Intent-to-treat Analysis Set
Urinary KS was measured by a quantitative method and normalized using the sample urinary creatinine measurement. Percent change from baseline to Week 12, 24, and 52 in normalized urine keratan sulfate (ug/mg).
Outcome measures
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=15 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
All Subjects
n=25 Participants
Both groups combined
|
|---|---|---|---|
|
Normalized Urine Keratan Sulfate (uKS)
Week 12 (%)
|
-27.6 Percent Change
Standard Deviation 25.10
|
-50.9 Percent Change
Standard Deviation 14.00
|
-36.7 Percent Change
Standard Deviation 24.07
|
|
Normalized Urine Keratan Sulfate (uKS)
Week 24 (%)
|
-37.4 Percent Change
Standard Deviation 23.21
|
-55.5 Percent Change
Standard Deviation 12.62
|
-44.5 Percent Change
Standard Deviation 21.39
|
|
Normalized Urine Keratan Sulfate (uKS)
Week 52 (%)
|
-20.5 Percent Change
Standard Deviation 36.95
|
-52.8 Percent Change
Standard Deviation 5.74
|
-31.3 Percent Change
Standard Deviation 33.69
|
SECONDARY outcome
Timeframe: Baseline, Week 25 and 52Population: A subset of mITT subjects who were scheduled to perform CPET
Subjects performed maximal incremental exercise testing using an electronically braked upright cycle ergometer. Cycle ergometry is a method of CPET that may be feasible in subjects who have orthopedic, peripheral vascular, or neurological limitations that restrict weight bearing. Change from baseline to Week 25 and 52 as measured by the CPET Duration of Exercise (min)
Outcome measures
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=5 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
All Subjects
n=15 Participants
Both groups combined
|
|---|---|---|---|
|
Cardiopulmonary Exercise Testing (CPET) - Duration of Exercise
CPET Duration of Exercise Week 25 (min)
|
0.8 Percent Change
Standard Deviation 0.88
|
1.1 Percent Change
Standard Deviation 1.43
|
0.9 Percent Change
Standard Deviation 1.05
|
|
Cardiopulmonary Exercise Testing (CPET) - Duration of Exercise
CPET Duration of Exercise Week 52 (min)
|
0.6 Percent Change
Standard Deviation 1.34
|
0.7 Percent Change
Standard Deviation 1.46
|
0.6 Percent Change
Standard Deviation 1.33
|
SECONDARY outcome
Timeframe: Baseline, Week 25 and 52Population: A subset of mITT subjects who were scheduled to perform CPET
Subjects performed maximal incremental exercise testing using an electronically braked upright cycle ergometer. Cycle ergometry is a method of CPET that may be feasible in subjects who have orthopedic, peripheral vascular, or neurological limitations that restrict weight bearing. Percent change from baseline to Week 25 and 52 as measured by the CPET Peak workload (watt)
Outcome measures
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=5 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
All Subjects
n=15 Participants
Both groups combined
|
|---|---|---|---|
|
Cardiopulmonary Exercise Testing (CPET) - Peak Workload
Week 25
|
23.6 Percent Change
Standard Deviation 20.87
|
38.4 Percent Change
Standard Deviation 44.66
|
28.5 Percent Change
Standard Deviation 30.04
|
|
Cardiopulmonary Exercise Testing (CPET) - Peak Workload
Week 52
|
24.7 Percent Change
Standard Deviation 26.17
|
13.0 Percent Change
Standard Deviation 22.69
|
20.8 Percent Change
Standard Deviation 24.90
|
SECONDARY outcome
Timeframe: Baseline, Week 25 and 52Population: A subset of mITT subjects who were scheduled to perform CPET
Subjects performed maximal incremental exercise testing using an electronically braked upright cycle ergometer. Cycle ergometry is a method of CPET that may be feasible in subjects who have orthopedic, peripheral vascular, or neurological limitations that restrict weight bearing. Percent change from baseline to Week 25 and 52 as measured by the CPET O2 pulse (ml/beat)
Outcome measures
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=5 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
All Subjects
n=15 Participants
Both groups combined
|
|---|---|---|---|
|
Cardiopulmonary Exercise Testing (CPET) - O2 Pulse
Week 25
|
6.8 Percent Change
Standard Deviation 14.17
|
13.5 Percent Change
Standard Deviation 17.65
|
9.0 Percent Change
Standard Deviation 15.13
|
|
Cardiopulmonary Exercise Testing (CPET) - O2 Pulse
Week 52
|
7.6 Percent Change
Standard Deviation 27.89
|
2.0 Percent Change
Standard Deviation 31.74
|
5.6 Percent Change
Standard Deviation 28.22
|
SECONDARY outcome
Timeframe: Baseline, Week 25 and 52Population: A subset of mITT subjects who were scheduled to perform CPET
Subjects performed maximal incremental exercise testing using an electronically braked upright cycle ergometer. Cycle ergometry is a method of CPET that may be feasible in subjects who have orthopedic, peripheral vascular, or neurological limitations that restrict weight bearing. Percent change from baseline to Week 25 and 52 as measured by the CPET Aerobic Efficiency (ml/watt). Note that decline in Aerobic Efficiency translate into an improvement
Outcome measures
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=5 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
All Subjects
n=15 Participants
Both groups combined
|
|---|---|---|---|
|
Cardiopulmonary Exercise Testing (CPET) - Aerobic Efficiency
Week 52
|
-7.5 Percent Change
Standard Deviation 28.39
|
-9.8 Percent Change
Standard Deviation 33.14
|
-8.2 Percent Change
Standard Deviation 28.52
|
|
Cardiopulmonary Exercise Testing (CPET) - Aerobic Efficiency
Week 25
|
-8.9 Percent Change
Standard Deviation 15.41
|
-6.0 Percent Change
Standard Deviation 8.29
|
-7.9 Percent Change
Standard Deviation 13.02
|
SECONDARY outcome
Timeframe: Baseline, Week 25 and 52Population: Modified Intent-to-treat Analysis Set
Change from baseline to Week 25 and 52 as measured by the peak force in MST knee extension test (newton meters).
Outcome measures
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=15 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
All Subjects
n=25 Participants
Both groups combined
|
|---|---|---|---|
|
Muscle Strength Testing (MST) - Knee Extension Test
Week 25
|
-0.6 Percent Change
Standard Deviation 9.76
|
5.2 Percent Change
Standard Deviation 7.63
|
1.7 Percent Change
Standard Deviation 9.26
|
|
Muscle Strength Testing (MST) - Knee Extension Test
Week 52
|
3.3 Percent Change
Standard Deviation 6.88
|
6.7 Percent Change
Standard Deviation 8.87
|
4.7 Percent Change
Standard Deviation 7.74
|
SECONDARY outcome
Timeframe: Baseline, Week 25 and 52Population: Modified Intent-to-treat Analysis Set
Percent change from baseline to Week 25 and 52 as measured by the peak force in MST knee flexion test (newton meters).
Outcome measures
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=15 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
All Subjects
n=25 Participants
Both groups combined
|
|---|---|---|---|
|
Muscle Strength Testing (MST) - Knee Flexion Test
Week 25
|
-4.2 Percent Change
Standard Deviation 24.83
|
9.4 Percent Change
Standard Deviation 44.87
|
1.5 Percent Change
Standard Deviation 34.39
|
|
Muscle Strength Testing (MST) - Knee Flexion Test
Week 52
|
-10.8 Percent Change
Standard Deviation 22.72
|
8.2 Percent Change
Standard Deviation 41.57
|
-2.7 Percent Change
Standard Deviation 32.69
|
SECONDARY outcome
Timeframe: Baseline, Week 25 and 52Population: Modified Intent-to-treat Analysis Set
Percent change from baseline to Week 25 and 52 as measured by the peak force in MST elbow flexion test (newton meters).
Outcome measures
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=15 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
All Subjects
n=25 Participants
Both groups combined
|
|---|---|---|---|
|
Muscle Strength Testing (MST) - Elbow Flexion Test
Week 25
|
12.0 Percent Change
Standard Deviation 55.42
|
17.7 Percent Change
Standard Deviation 41.82
|
14.1 Percent Change
Standard Deviation 49.67
|
|
Muscle Strength Testing (MST) - Elbow Flexion Test
Week 52
|
49.7 Percent Change
Standard Deviation 86.83
|
50.7 Percent Change
Standard Deviation 59.81
|
50.1 Percent Change
Standard Deviation 75.60
|
SECONDARY outcome
Timeframe: Baseline, Week 12, 24, and 52Population: Modified Intent-to-treat
The APPT is a validated, multidimensional tool to evaluate pain in children, adolescents, and young adults. The complete APPT is measured in three parts - Part 1 of the APPT scale determines the subject's pain locations using a body template. Part 2 of the APPT scale determines the intensity of the pain using a 10 cm visual analog scale (VAS) with the lowest point of the scale (0) labeled No Pain and the highest point on the scale (10) labeled Worst Possible Pain. Intermediate regions of the sale were labeled with 3 intermediate descriptors (Little Pain, Medium Pain, and Large Pain). Part 3 of the APPT scale characterizes the pain by tracking the number and percentage of words selected by subjects to describe their pain from a total of 57 choices. Part 2 corresponds most closely to other typically used pain scales (based on VAS) and for this reason the results from Part 2 are presented here. Change from baseline to Week 12, 24, and 52 in pain intensity.
Outcome measures
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=15 Participants
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=10 Participants
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
All Subjects
n=25 Participants
Both groups combined
|
|---|---|---|---|
|
Adolescent Pediatric Pain Tool (APPT) - Pain Intensity
APPT Word Graphic Rating Scale Week 12
|
-2.3 units on a scale
Standard Deviation 3.37
|
-1.2 units on a scale
Standard Deviation 2.23
|
-1.8 units on a scale
Standard Deviation 2.93
|
|
Adolescent Pediatric Pain Tool (APPT) - Pain Intensity
APPT Word Graphic Rating Scale Week 24
|
-2.2 units on a scale
Standard Deviation 3.68
|
-1.2 units on a scale
Standard Deviation 2.87
|
-1.8 units on a scale
Standard Deviation 3.33
|
|
Adolescent Pediatric Pain Tool (APPT) - Pain Intensity
APPT Word Graphic Rating Scale Week 52
|
-0.8 units on a scale
Standard Deviation 3.41
|
-0.8 units on a scale
Standard Deviation 3.04
|
-0.8 units on a scale
Standard Deviation 3.17
|
Adverse Events
BMN 110 Weekly at 2.0 mg/kg/Week
Serious events: 3 serious events
Other events: 15 other events
Deaths: 0 deaths
BMN 110 Weekly at 4.0 mg/kg/Week
Serious events: 2 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=15 participants at risk
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=10 participants at risk
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
|---|---|---|
|
Gastrointestinal disorders
Inflammatory bowel disease
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Umbilical hernia
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Knee deformity
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Kyphoscoliosis
|
6.7%
1/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Renal and urinary disorders
Enuresis
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Surgical and medical procedures
Medical device removal
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
Other adverse events
| Measure |
BMN 110 Weekly at 2.0 mg/kg/Week
n=15 participants at risk
BMN 110: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
BMN 110 Weekly at 4.0 mg/kg/Week
n=10 participants at risk
BMN 110: Weekly IV infusions of BMN 110 at 4.0 mg/kg/week over a period of approximately 4 hours per infusion in the primary phase.
|
|---|---|---|
|
Blood and lymphatic system disorders
Lymph node pain
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Cardiac disorders
Bradycardia
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Cardiac disorders
Sinus bradycardia
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Cardiac disorders
Tachycardia
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Congenital, familial and genetic disorders
Pectus carinatum
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Ear and labyrinth disorders
Ear discomfort
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Ear and labyrinth disorders
Ear pain
|
13.3%
2/15 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Ear and labyrinth disorders
Ear pruritus
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Ear and labyrinth disorders
Motion sickness
|
13.3%
2/15 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Ear and labyrinth disorders
Otorrhoea
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Ear and labyrinth disorders
Tinnitus
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Eye disorders
Eye pruritus
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Eye disorders
Photophobia
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Eye disorders
Vision blurred
|
6.7%
1/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Eye disorders
Vitreous floaters
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Abdominal discomfort
|
6.7%
1/15 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Abdominal pain
|
40.0%
6/15 • Number of events 18 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
30.0%
3/10 • Number of events 23 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Abdominal pain lower
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Abdominal pain upper
|
26.7%
4/15 • Number of events 8 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
40.0%
4/10 • Number of events 11 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Aerophagia
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Anal pruritus
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Aphthous stomatitis
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Constipation
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Diarrhoea
|
40.0%
6/15 • Number of events 21 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
40.0%
4/10 • Number of events 10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Dyspepsia
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 6 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Haematochezia
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Lip oedema
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Nausea
|
66.7%
10/15 • Number of events 33 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
50.0%
5/10 • Number of events 6 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Oral pain
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Retching
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Swollen tongue
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Toothache
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Gastrointestinal disorders
Vomiting
|
73.3%
11/15 • Number of events 34 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
50.0%
5/10 • Number of events 10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Catheter site pain
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Chest pain
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Chills
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Cyst
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Device malfunction
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Extravasation
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Face oedema
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Fatigue
|
53.3%
8/15 • Number of events 12 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 5 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Influenza like illness
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Infusion site bruising
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Infusion site erythema
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Infusion site extravasation
|
20.0%
3/15 • Number of events 6 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 4 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Infusion site swelling
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Injection site pain
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Injection site pruritus
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Injection site reaction
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Irritability
|
13.3%
2/15 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Local swelling
|
13.3%
2/15 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Malaise
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Medical device pain
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Oedema peripheral
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Pain
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
General disorders
Pyrexia
|
60.0%
9/15 • Number of events 24 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
30.0%
3/10 • Number of events 9 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Immune system disorders
Seasonal allergy
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Ear infection
|
13.3%
2/15 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Enterobiasis
|
6.7%
1/15 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Fungal infection
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Gastroenteritis
|
13.3%
2/15 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
30.0%
3/10 • Number of events 5 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Gastroenteritis viral
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Gastrointestinal infection
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Influenza
|
26.7%
4/15 • Number of events 4 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Nasopharyngitis
|
40.0%
6/15 • Number of events 10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
70.0%
7/10 • Number of events 16 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Oral herpes
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Otitis media
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Pharyngitis
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Pharyngitis streptococcal
|
13.3%
2/15 • Number of events 5 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Pneumonia
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Rhinitis
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Sinusitis
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Upper respiratory tract infection
|
33.3%
5/15 • Number of events 13 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
50.0%
5/10 • Number of events 7 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Infections and infestations
Urinary tract infection
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Contusion
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Excoriation
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Fall
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Hand fracture
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Head injury
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Inadequate analgesia
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Injection related reaction
|
6.7%
1/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Joint injury
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Laceration
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Limb crushing injury
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Limb injury
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Muscle strain
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Procedural pain
|
33.3%
5/15 • Number of events 8 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Injury, poisoning and procedural complications
Wound complication
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Investigations
Blood glucose decreased
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Investigations
Haemoglobin increased
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Investigations
Urine leukocyte esterase positive
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Investigations
Urine output decreased
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Investigations
Weight decreased
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Investigations
White blood cells urine positive
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Metabolism and nutrition disorders
Decreased appetite
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Metabolism and nutrition disorders
Increased appetite
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
40.0%
6/15 • Number of events 28 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
50.0%
5/10 • Number of events 11 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
40.0%
6/15 • Number of events 13 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Elbow deformity
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Knee deformity
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Kyphoscoliosis
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
20.0%
3/15 • Number of events 4 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
13.3%
2/15 • Number of events 5 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Osteochondrosis
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Osteopenia
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
53.3%
8/15 • Number of events 22 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
40.0%
4/10 • Number of events 5 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Nervous system disorders
Coordination abnormal
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Nervous system disorders
Dizziness
|
40.0%
6/15 • Number of events 8 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Nervous system disorders
Dysgeusia
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Nervous system disorders
Headache
|
86.7%
13/15 • Number of events 89 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
70.0%
7/10 • Number of events 44 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Nervous system disorders
Lethargy
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Nervous system disorders
Neuralgia
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Nervous system disorders
Poor quality sleep
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Nervous system disorders
Somnolence
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Nervous system disorders
Syncope
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Psychiatric disorders
Anxiety
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Psychiatric disorders
Depression
|
6.7%
1/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Psychiatric disorders
Insomnia
|
20.0%
3/15 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Psychiatric disorders
Phobia
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Renal and urinary disorders
Dysuria
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Renal and urinary disorders
Enuresis
|
6.7%
1/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Renal and urinary disorders
Pollakiuria
|
6.7%
1/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
6.7%
1/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
40.0%
6/15 • Number of events 9 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
40.0%
4/10 • Number of events 8 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
26.7%
4/15 • Number of events 4 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
20.0%
3/15 • Number of events 5 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Hypoventilation
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
46.7%
7/15 • Number of events 9 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
46.7%
7/15 • Number of events 11 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
40.0%
4/10 • Number of events 6 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
6.7%
1/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Acne
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 4 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Decubitus ulcer
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Ecchymosis
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Eczema
|
13.3%
2/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Erythema
|
26.7%
4/15 • Number of events 9 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Nail disorder
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
20.0%
3/15 • Number of events 6 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Rash
|
40.0%
6/15 • Number of events 7 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Rash erythematous
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Rash generalised
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Rash pruritic
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 3 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Swelling face
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
20.0%
3/15 • Number of events 11 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Surgical and medical procedures
Catheterisation venous
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Surgical and medical procedures
Central venous catheterisation
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
10.0%
1/10 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Surgical and medical procedures
Medical device removal
|
0.00%
0/15 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
20.0%
2/10 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Surgical and medical procedures
Tooth extraction
|
6.7%
1/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Vascular disorders
Flushing
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Vascular disorders
Hot flush
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Vascular disorders
Hypotension
|
6.7%
1/15 • Number of events 2 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Vascular disorders
Pallor
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
|
Vascular disorders
Peripheral coldness
|
6.7%
1/15 • Number of events 1 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
0.00%
0/10 • Study Period, up to 192 weeks or ETV
All Adverse Events
|
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 up to 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