Trial Outcomes & Findings for Study of OnabotulinumtoxinA (BOTOX®) for Urinary Incontinence Due to Neurogenic Detrusor Overactivity in Pediatric Patients (NCT NCT01852045)
NCT ID: NCT01852045
Last Updated: 2019-11-21
Results Overview
Urinary incontinence was defined as involuntary loss of urine as recorded by the participant in a bladder diary during the 2 consecutive days (normalized to a 12-hour daytime period) prior to the study visit. Daytime was defined as the time between waking up to start the day and first morning catheterization and going to bed to sleep for the night. The number of incontinence episodes were averaged daily during this period. A negative change from Baseline indicates improvement. Least squares estimates were based on an Analysis of Covariance (ANCOVA) model.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
114 participants
Primary outcome timeframe
Baseline (Day -28 to Day -1) to 2 consecutive days prior to Week 6
Results posted on
2019-11-21
Participant Flow
114 patients were enrolled and randomized into the study; 113 received study treatment.
Participant milestones
| Measure |
OnabotulinumtoxinA 50 U
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Overall Study
STARTED
|
39
|
45
|
30
|
|
Overall Study
mITT Population
|
38
|
45
|
30
|
|
Overall Study
Safety Population
|
38
|
45
|
30
|
|
Overall Study
COMPLETED
|
33
|
41
|
26
|
|
Overall Study
NOT COMPLETED
|
6
|
4
|
4
|
Reasons for withdrawal
| Measure |
OnabotulinumtoxinA 50 U
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
0
|
|
Overall Study
Lack of Efficacy
|
3
|
0
|
0
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
1
|
|
Overall Study
Other Miscellaneous Reasons
|
1
|
3
|
2
|
Baseline Characteristics
Modified Intent-to-Treat (mITT) population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received.
Baseline characteristics by cohort
| Measure |
OnabotulinumtoxinA 50 U
n=39 Participants
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=45 Participants
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=30 Participants
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
Total
n=114 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
11.4 years
STANDARD_DEVIATION 3.45 • n=39 Participants
|
10.8 years
STANDARD_DEVIATION 3.26 • n=45 Participants
|
11.9 years
STANDARD_DEVIATION 3.13 • n=30 Participants
|
11.3 years
STANDARD_DEVIATION 3.29 • n=114 Participants
|
|
Sex: Female, Male
Female
|
19 Participants
n=39 Participants
|
15 Participants
n=45 Participants
|
15 Participants
n=30 Participants
|
49 Participants
n=114 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=39 Participants
|
30 Participants
n=45 Participants
|
15 Participants
n=30 Participants
|
65 Participants
n=114 Participants
|
|
Race/Ethnicity, Customized
White
|
29 Participants
n=39 Participants
|
34 Participants
n=45 Participants
|
22 Participants
n=30 Participants
|
85 Participants
n=114 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
6 Participants
n=39 Participants
|
3 Participants
n=45 Participants
|
2 Participants
n=30 Participants
|
11 Participants
n=114 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=39 Participants
|
2 Participants
n=45 Participants
|
1 Participants
n=30 Participants
|
4 Participants
n=114 Participants
|
|
Race/Ethnicity, Customized
Hispanic
|
1 Participants
n=39 Participants
|
3 Participants
n=45 Participants
|
3 Participants
n=30 Participants
|
7 Participants
n=114 Participants
|
|
Race/Ethnicity, Customized
Other
|
2 Participants
n=39 Participants
|
3 Participants
n=45 Participants
|
2 Participants
n=30 Participants
|
7 Participants
n=114 Participants
|
|
Daily Daytime Average Frequency of Urinary Incontinence Episodes
|
2.81 urinary incontinence episodes per day
n=38 Participants • Modified Intent-to-Treat (mITT) population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received.
|
2.99 urinary incontinence episodes per day
n=45 Participants • Modified Intent-to-Treat (mITT) population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received.
|
3.68 urinary incontinence episodes per day
n=30 Participants • Modified Intent-to-Treat (mITT) population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received.
|
3.16 urinary incontinence episodes per day
n=113 Participants • Modified Intent-to-Treat (mITT) population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received.
|
PRIMARY outcome
Timeframe: Baseline (Day -28 to Day -1) to 2 consecutive days prior to Week 6Population: mITT population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received. Missing data are imputed up to Week 6 using Last Observation Carried Forward (LOCF) method.
Urinary incontinence was defined as involuntary loss of urine as recorded by the participant in a bladder diary during the 2 consecutive days (normalized to a 12-hour daytime period) prior to the study visit. Daytime was defined as the time between waking up to start the day and first morning catheterization and going to bed to sleep for the night. The number of incontinence episodes were averaged daily during this period. A negative change from Baseline indicates improvement. Least squares estimates were based on an Analysis of Covariance (ANCOVA) model.
Outcome measures
| Measure |
OnabotulinumtoxinA 50 U
n=38 Participants
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=45 Participants
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=30 Participants
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Change From Baseline in Daily Average Frequency of Daytime Urinary Incontinence Episodes
|
-1.30 urinary incontinence episodes per day
Standard Error 0.205
|
-1.30 urinary incontinence episodes per day
Standard Error 0.189
|
-1.34 urinary incontinence episodes per day
Standard Error 0.245
|
SECONDARY outcome
Timeframe: First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)Population: Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
An adverse event is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A TEAE is defined as any new adverse event or worsening of an existing condition after initiation of treatment.
Outcome measures
| Measure |
OnabotulinumtoxinA 50 U
n=38 Participants
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=45 Participants
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=30 Participants
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Number of Participants With Treatment Emergent Adverse Events (TEAE)
|
27 Participants
|
33 Participants
|
23 Participants
|
SECONDARY outcome
Timeframe: Baseline (Day -28 to Day -1) to 2 consecutive days prior to Week 6Population: mITT population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received. Number analyzed is number of participants with non-missing values at the specified Visit.
The change in urine volume at first morning catherization was recorded by the participant in a bladder diary in the 2 consecutive days during the week prior to the study visit. A positive change from Baseline indicates improvement. Least squares estimates were based on an ANCOVA model.
Outcome measures
| Measure |
OnabotulinumtoxinA 50 U
n=36 Participants
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=43 Participants
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=27 Participants
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Change From Baseline in Average Urine Volume at First Morning Catheterization
|
21.93 milliliters (mL)
Standard Error 14.676
|
34.90 milliliters (mL)
Standard Error 13.580
|
87.49 milliliters (mL)
Standard Error 17.808
|
SECONDARY outcome
Timeframe: Baseline (Day -28 to Day -1), Week 6Population: mITT population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received. Number analyzed is number of participants with non-missing values at the specified Visit.
Urinary incontinence was defined as involuntary loss of urine and the presence or absence of night time urinary incontinence was recorded by the participant in a bladder diary in the 2 consecutive days (normalized to a 12-hour daytime period) during the week prior to the study visit. Night time was defined as the time between going to bed to sleep for the night and waking up to start the day. The percentage of participants with night time urinary incontinence is presented in categories (0, 1, 2 nights).
Outcome measures
| Measure |
OnabotulinumtoxinA 50 U
n=38 Participants
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=45 Participants
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=28 Participants
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Percentage of Participants With Night Time Urinary Incontinence
Baseline (BL): 0 nights of incontinence
|
0.0 percentage of participants
|
13.3 percentage of participants
|
3.6 percentage of participants
|
|
Percentage of Participants With Night Time Urinary Incontinence
BL: 1 night of incontinence
|
13.2 percentage of participants
|
2.2 percentage of participants
|
14.3 percentage of participants
|
|
Percentage of Participants With Night Time Urinary Incontinence
BL: 2 nights of incontinence
|
86.8 percentage of participants
|
84.4 percentage of participants
|
82.1 percentage of participants
|
|
Percentage of Participants With Night Time Urinary Incontinence
Week 6: 0 nights of incontinence
|
30.6 percentage of participants
|
32.6 percentage of participants
|
28.6 percentage of participants
|
|
Percentage of Participants With Night Time Urinary Incontinence
Week 6: 1 night of incontinence
|
16.7 percentage of participants
|
16.3 percentage of participants
|
28.6 percentage of participants
|
|
Percentage of Participants With Night Time Urinary Incontinence
Week 6: 2 nights of incontinence
|
52.8 percentage of participants
|
51.2 percentage of participants
|
42.9 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline (Day -28 to Day -1) to Week 6Population: mITT population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received. Number analyzed is number of participants with non-missing values at the specified Visit.
The MCC was defined by urodynamics, as the volume infused before the participant felt they could no longer delay micturition (has a strong desire to void), had a leakage, or 500 mL was instilled. A positive change from Baseline indicates improvement (increase) in the maximum volume of urine the bladder holds. Least squares estimates were based on an ANCOVA model.
Outcome measures
| Measure |
OnabotulinumtoxinA 50 U
n=34 Participants
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=38 Participants
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=28 Participants
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Change From Baseline in Maximum Cystometric Capacity (MCC)
|
62.06 mL
Standard Error 14.339
|
48.57 mL
Standard Error 13.549
|
63.55 mL
Standard Error 17.363
|
SECONDARY outcome
Timeframe: Baseline (Day -28 to -1) and Week 6Population: mITT population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received. Number analyzed is number of participants with non-missing values at the specified Visit.
Urodynamic tests were performed by site personnel qualified for performing pressure/flow cystometry. The results were verified by an independent central reviewer. Cystometry was used to measures the presence of involuntary detrusor contractions upon filling. A reduction in IDCs from Baseline to Week 6 indicates improvement.
Outcome measures
| Measure |
OnabotulinumtoxinA 50 U
n=38 Participants
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=45 Participants
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=30 Participants
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Percentage of Participants With Involuntary Detrusor Contractions (IDC)
Baseline
|
94.4 percentage of participants
Interval 81.34 to 99.32
|
88.1 percentage of participants
Interval 74.37 to 96.02
|
92.6 percentage of participants
Interval 75.71 to 99.09
|
|
Percentage of Participants With Involuntary Detrusor Contractions (IDC)
Week 6
|
61.8 percentage of participants
Interval 43.56 to 77.83
|
44.7 percentage of participants
Interval 28.62 to 61.7
|
46.4 percentage of participants
Interval 27.51 to 66.13
|
SECONDARY outcome
Timeframe: Baseline (Day-28 to Day-1) to Week 6Population: mITT population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received. Only participants who experienced an IDC are included in the analysis.
Urodynamic tests were performed by site personnel qualified for performing pressure/flow cystometry. The results were verified by an independent central reviewer. Cystometry was used to measures the pressure inside of the bladder to see how well the bladder was working. A negative change from Baseline indicates improvement. Least squares estimates were based on an ANCOVA model.
Outcome measures
| Measure |
OnabotulinumtoxinA 50 U
n=21 Participants
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=17 Participants
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=12 Participants
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Change From Baseline in Maximum Detrusor Pressure During the First IDC (PdetMax1stIDC) in Participants With IDC
|
-7.64 centimeters of water (cm H2O)
Standard Error 5.301
|
-12.13 centimeters of water (cm H2O)
Standard Error 5.573
|
-5.46 centimeters of water (cm H2O)
Standard Error 8.267
|
SECONDARY outcome
Timeframe: Baseline (Day 1) to Week 6Population: mITT population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received. Number analyzed is number of participants with non-missing values at the specified Visit.
Urodynamic tests were performed by site personnel qualified for performing pressure/flow cystometry. The results were verified by an independent central reviewer. Cystometry was used to measures the pressure inside of the bladder to see how well the bladder was working. A negative change from Baseline indicates improvement. Least squares estimates were based on an ANCOVA model.
Outcome measures
| Measure |
OnabotulinumtoxinA 50 U
n=34 Participants
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=38 Participants
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=28 Participants
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Change From Baseline in Maximum Detrusor Pressure (PdetMax) During the Storage Phase
|
-12.88 cm H2O
Standard Error 3.793
|
-20.09 cm H2O
Standard Error 3.632
|
-27.31 cm H2O
Standard Error 4.557
|
SECONDARY outcome
Timeframe: Baseline (Day -28 to -1) to Week 6Population: mITT population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received. Only participants who experienced a leak during urodynamics are included in the analysis.
DLPP was defined as the lowest detrusor pressure at which urine leakage occurs in the absence of either a detrusor contraction or increased intra-abdominal pressure. Urodynamic tests were performed by site personnel qualified for performing pressure/flow cystometry. The results were verified by an independent central reviewer. Cystometry was used to measures the pressure inside of the bladder to see how well the bladder was working. A negative change from Baseline indicates improvement. Least squares estimates are based on an ANCOVA model.
Outcome measures
| Measure |
OnabotulinumtoxinA 50 U
n=2 Participants
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=1 Participants
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=1 Participants
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Change From Baseline in Detrusor Leak Point Pressure (DLPP) During the Storage Phase
|
9.50 cm H2O
Standard Deviation 2.121
|
-39.00 cm H2O
Standard Deviation 0.000
|
12.00 cm H2O
Standard Deviation 0.000
|
SECONDARY outcome
Timeframe: 48 weeksPopulation: mITT population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received. Number analyzed is number of participants with non-missing values at the specified Visit.
Time from treatment on Day 1 to request for retreatment was estimated. For those participants who did not request retreatment, their data was censored using the date of their last study visit.
Outcome measures
| Measure |
OnabotulinumtoxinA 50 U
n=27 Participants
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=35 Participants
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=23 Participants
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Time to Participant Request for Retreatment
|
30.6 weeks
Interval 23.1 to 39.1
|
24.1 weeks
Interval 18.1 to 27.6
|
29.6 weeks
Interval 16.3 to 37.3
|
SECONDARY outcome
Timeframe: 48 weeksPopulation: mITT population included participants who received study drug on Day 1, analyzed on as-randomized basis, except those who received less than their randomized dose due to weight and dose limit of 6 U/kg, allocated to nearest dose group based on dose received. Number analyzed is number of participants with non-missing values at the specified Visit.
In order to qualify for retreatment, the criteria listed below must be fulfilled at the qualification for retreatment visit: Participant/parent/caregiver requests retreatment, participant has a total of at least 2 daytime urinary incontinence episodes over the 2-day bladder diary collection period, at least 12 weeks has elapsed since treatment 1 and participant has not experienced a serious treatment-related adverse event at any time.
Outcome measures
| Measure |
OnabotulinumtoxinA 50 U
n=27 Participants
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=35 Participants
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=23 Participants
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Time to Participant Qualification for Retreatment
|
35.0 weeks
Interval 23.1 to 39.1
|
25.0 weeks
Interval 20.0 to 32.1
|
29.6 weeks
Interval 16.3 to 38.0
|
Adverse Events
OnabotulinumtoxinA 50 U
Serious events: 4 serious events
Other events: 27 other events
Deaths: 0 deaths
OnabotulinumtoxinA 100 U
Serious events: 3 serious events
Other events: 33 other events
Deaths: 0 deaths
OnabotulinumtoxinA 200 U
Serious events: 2 serious events
Other events: 23 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
OnabotulinumtoxinA 50 U
n=38 participants at risk
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=45 participants at risk
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=30 participants at risk
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Infections and infestations
Urinary tract infection
|
5.3%
2/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
4.4%
2/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Infections and infestations
Cystitis
|
2.6%
1/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Infections and infestations
Postoperative wound infection
|
2.6%
1/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Infections and infestations
Encephalitis viral
|
0.00%
0/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
2.2%
1/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Infections and infestations
Epididymitis
|
0.00%
0/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
3.3%
1/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Infections and infestations
Orchitis
|
0.00%
0/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
3.3%
1/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Injury, poisoning and procedural complications
Arteriovenous fistula thrombosis
|
2.6%
1/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Nervous system disorders
Hydrocephalus
|
0.00%
0/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
2.2%
1/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Vascular disorders
Hypertension
|
0.00%
0/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
3.3%
1/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
Other adverse events
| Measure |
OnabotulinumtoxinA 50 U
n=38 participants at risk
OnabotulinumtoxinA (botulinum toxin Type A) 50 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 (NCT01852058) if qualified.
|
OnabotulinumtoxinA 100 U
n=45 participants at risk
OnabotulinumtoxinA 100 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
OnabotulinumtoxinA 200 U
n=30 participants at risk
OnabotulinumtoxinA 200 U (not to exceed 6 U/kg) injected into the detrusor wall on Day 1. Participants were eligible for retreatment in study 191622-121 if qualified.
|
|---|---|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
2.6%
1/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
6.7%
3/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
6.7%
2/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Gastrointestinal disorders
Abdominal pain
|
5.3%
2/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
2.2%
1/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
3.3%
1/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Gastrointestinal disorders
Vomiting
|
2.6%
1/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
6.7%
3/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
General disorders
Pyrexia
|
5.3%
2/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
8.9%
4/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
General disorders
Suprapubic pain
|
5.3%
2/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
3.3%
1/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Infections and infestations
Urinary tract infection
|
23.7%
9/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
28.9%
13/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
23.3%
7/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Infections and infestations
Bacteriuria
|
15.8%
6/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
15.6%
7/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
20.0%
6/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Infections and infestations
Pharyngitis
|
7.9%
3/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
6.7%
3/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Infections and infestations
Gastroenteritis
|
5.3%
2/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
6.7%
3/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
3.3%
1/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
2.2%
1/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
13.3%
4/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
6.7%
3/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
3.3%
1/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Infections and infestations
Sinusitis
|
5.3%
2/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
3.3%
1/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Injury, poisoning and procedural complications
Limb injury
|
0.00%
0/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
2.2%
1/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
6.7%
2/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Nervous system disorders
Headache
|
2.6%
1/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
15.6%
7/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
6.7%
2/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Renal and urinary disorders
Leukocyturia
|
2.6%
1/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
6.7%
3/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
13.3%
4/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Renal and urinary disorders
Hydronephrosis
|
5.3%
2/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
2.2%
1/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.3%
2/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
2.2%
1/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
0.00%
0/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
|
Skin and subcutaneous tissue disorders
Acne
|
5.3%
2/38 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
2.2%
1/45 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
3.3%
1/30 • First study treatment to 12 weeks after last treatment (Up to 48 weeks after first study injection)
Safety population included participants who underwent treatment procedure and received study drug on randomization/Day 1, except those who received less dose due to 6 U/kg weight cap, were allocated to nearest dose group based on the actual dose received.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee A disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 90 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER