Trial Outcomes & Findings for Effects and Safety of Liposome Encapsulated Botulinum Toxin A for Overactive Bladder Syndrome (NCT NCT01167257)
NCT ID: NCT01167257
Last Updated: 2014-09-22
Results Overview
Efficacy: Mean change of the total frequency per 3 days from baseline to 4 weeks after the treatment day based on the 3-day voiding diary. Change = Week 4 minus Baseline value
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
62 participants
Primary outcome timeframe
Baseline to 4 weeks after initial treatment
Results posted on
2014-09-22
Participant Flow
Participant milestones
| Measure |
Experimental Arm
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80 mg/40 mL) in single intravesical instillation
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
Normal saline 50ml in single intravesical instillation
Normal saline instillation: Normal saline (BoNT-A/NS) 50ml in single intravesical instillation
|
|---|---|---|
|
Overall Study
STARTED
|
31
|
31
|
|
Overall Study
COMPLETED
|
28
|
28
|
|
Overall Study
NOT COMPLETED
|
3
|
3
|
Reasons for withdrawal
| Measure |
Experimental Arm
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80 mg/40 mL) in single intravesical instillation
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
Normal saline 50ml in single intravesical instillation
Normal saline instillation: Normal saline (BoNT-A/NS) 50ml in single intravesical instillation
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
2
|
2
|
|
Overall Study
Withdrew consent
|
1
|
1
|
Baseline Characteristics
Effects and Safety of Liposome Encapsulated Botulinum Toxin A for Overactive Bladder Syndrome
Baseline characteristics by cohort
| Measure |
Experimental Arm
n=31 Participants
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80mg/40ml) in single intravesical instillation
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
n=31 Participants
Normal saline 50 mL in single intravesical instillation
Normal saline instillation: Normal saline (BoNT-A/NS) 50ml in single intravesical instillation
|
Total
n=62 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
64.43 years
STANDARD_DEVIATION 12.49 • n=5 Participants
|
65.81 years
STANDARD_DEVIATION 14.48 • n=7 Participants
|
65.12 years
STANDARD_DEVIATION 13.49 • n=5 Participants
|
|
Sex: Female, Male
Female
|
18 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
33 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
29 Participants
n=5 Participants
|
|
Region of Enrollment
Taiwan
|
31 participants
n=5 Participants
|
31 participants
n=7 Participants
|
62 participants
n=5 Participants
|
|
Associated medical conditions
Diabetes Mellitus
|
3 participants
n=5 Participants
|
9 participants
n=7 Participants
|
12 participants
n=5 Participants
|
|
Associated medical conditions
Hypertension
|
5 participants
n=5 Participants
|
12 participants
n=7 Participants
|
17 participants
n=5 Participants
|
|
Associated medical conditions
Pelvic surgery
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Associated medical conditions
Other disease
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Associated medical conditions
Non disease
|
22 participants
n=5 Participants
|
8 participants
n=7 Participants
|
30 participants
n=5 Participants
|
|
Background or Prior disease
Any prior disease
|
31 participants
n=5 Participants
|
31 participants
n=7 Participants
|
62 participants
n=5 Participants
|
|
Background or Prior disease
None
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Prior therapy
Prior therapy taken
|
29 participants
n=5 Participants
|
29 participants
n=7 Participants
|
58 participants
n=5 Participants
|
|
Prior therapy
None
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
4 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to 4 weeks after initial treatmentPopulation: Control arm: subject number S025 was removed from analysis for not meeting the requirement for analysis, thus control arm 27 subjects
Efficacy: Mean change of the total frequency per 3 days from baseline to 4 weeks after the treatment day based on the 3-day voiding diary. Change = Week 4 minus Baseline value
Outcome measures
| Measure |
Experimental Arm
n=28 Participants
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80 mg/40 mL) in single intravesical instillation
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
n=27 Participants
Normal saline 50 mL in single intravesical instillation
Normal saline instillation: Normal saline (BoNT-A/NS) 50 mL in single intravesical instillation
|
|---|---|---|
|
Mean Change of the Total Frequency Per 3 Days
|
-4.64 Frequency per 3 days
Interval -7.46 to -1.82
|
-0.19 Frequency per 3 days
Interval -2.98 to 2.61
|
SECONDARY outcome
Timeframe: Baseline to 4 weeks after initial treatmentPopulation: Control arm: subject number S025 was removed from analysis for not meeting the requirement for analysis, thus control arm 27 subjects
Efficacy: Mean change of the Urgency episodes per 3 days from baseline to 4 weeks after the treatment day based on the 3-day voiding diary. Change = Week 4 minus Baseline value
Outcome measures
| Measure |
Experimental Arm
n=28 Participants
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80 mg/40 mL) in single intravesical instillation
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
n=27 Participants
Normal saline 50 mL in single intravesical instillation
Normal saline instillation: Normal saline (BoNT-A/NS) 50 mL in single intravesical instillation
|
|---|---|---|
|
Mean Change of the Urgency Episodes Per 3 Days
|
-7.43 Frequency per 3 days
Interval -11.68 to -3.18
|
-3.43 Frequency per 3 days
Interval -7.3 to 0.45
|
SECONDARY outcome
Timeframe: Baseline to 4 weeks after initial treatmentPopulation: Control arm: subject number S025 was removed from analysis for not meeting the requirement for analysis, thus control arm 27 subjects
Efficacy: Mean change of the urgency urinary incontinence (UUI) per 3 days from baseline to 4 weeks after the treatment day based on the 3-day voiding diary. Change = Week 4 minus Baseline value
Outcome measures
| Measure |
Experimental Arm
n=28 Participants
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80 mg/40 mL) in single intravesical instillation
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
n=27 Participants
Normal saline 50 mL in single intravesical instillation
Normal saline instillation: Normal saline (BoNT-A/NS) 50 mL in single intravesical instillation
|
|---|---|---|
|
Mean Change of the Urgency Urinary Incontinence (UUI) Per 3 Days
|
0.43 Frequency per 3 days
Interval -3.33 to 4.19
|
0.56 Frequency per 3 days
Interval -1.27 to 2.38
|
SECONDARY outcome
Timeframe: Baseline to 4 weeks after initial treatmentEfficacy:(measured the net change of variables from baseline to 1 month) Overactive bladder symptom score (OABSS) The OABSS is a 4-item questionnaire developed to evaluate OAB symptoms. The maximal scores are 2, 3, 5 and 5 for daytime frequency, nighttime frequency, urgency and urgency in continence, respectively. The OABSS range = 0 to 15 ((asymptomatic to very symptomatic). Change = Week 4 minus Baseline value
Outcome measures
| Measure |
Experimental Arm
n=28 Participants
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80 mg/40 mL) in single intravesical instillation
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
n=28 Participants
Normal saline 50 mL in single intravesical instillation
Normal saline instillation: Normal saline (BoNT-A/NS) 50 mL in single intravesical instillation
|
|---|---|---|
|
Net Change of the Overactive Bladder Symptom Score (OABSS)
|
-1.86 units on a scale
Interval -2.96 to -0.76
|
-0.75 units on a scale
Interval -2.01 to 0.51
|
SECONDARY outcome
Timeframe: Baseline to 4 weeks after initial treatmentPopulation: Control arm: subject number S025 was removed from analysis for not meeting the requirement for analysis, thus control arm 27 subjects
Efficacy:(measured the net change of variables from baseline and 1 month) Functional bladder capacity (FBC) Change = Week 4 minus Baseline value
Outcome measures
| Measure |
Experimental Arm
n=28 Participants
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80 mg/40 mL) in single intravesical instillation
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
n=27 Participants
Normal saline 50 mL in single intravesical instillation
Normal saline instillation: Normal saline (BoNT-A/NS) 50 mL in single intravesical instillation
|
|---|---|---|
|
Net Change of the Functional Bladder Capacity (FBC)
|
-12.86 mL
Interval -45.79 to 20.08
|
18.15 mL
Interval -16.37 to 52.67
|
SECONDARY outcome
Timeframe: Baseline to 4 weeks after initial treatmentPopulation: Control arm: subject number S025 was removed from analysis for not meeting the requirement for analysis, thus control arm 27 subjects
Efficacy:(measured the net change of variables from baseline and 1 month) Maximum flow rate (Qmax) Change = Week 4 minus Baseline value
Outcome measures
| Measure |
Experimental Arm
n=28 Participants
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80 mg/40 mL) in single intravesical instillation
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
n=27 Participants
Normal saline 50 mL in single intravesical instillation
Normal saline instillation: Normal saline (BoNT-A/NS) 50 mL in single intravesical instillation
|
|---|---|---|
|
Net Change of the Maximum Flow Rate (Qmax)
|
0.11 mL/s
Interval -2.02 to 2.25
|
-0.84 mL/s
Interval -4.3 to 2.62
|
SECONDARY outcome
Timeframe: Baseline and 1 month after initial treatmentEfficacy:(measured the net change of variables from baseline and 1 month) Postvoid residual volume (PVR) Change = Week 4 minus Baseline value
Outcome measures
| Measure |
Experimental Arm
n=28 Participants
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80 mg/40 mL) in single intravesical instillation
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
n=28 Participants
Normal saline 50 mL in single intravesical instillation
Normal saline instillation: Normal saline (BoNT-A/NS) 50 mL in single intravesical instillation
|
|---|---|---|
|
Net Change of the Postvoid Residual Volume (PVR)
|
-2.65 mL
Interval -20.13 to 14.84
|
-2.31 mL
Interval -19.43 to 14.81
|
SECONDARY outcome
Timeframe: Baseline to 4 weeks after initial treatmentEfficacy:(measured the net change of variables from baseline and 1 month) Urgency severity score (USS) within 3 days. The USS have 1-point scale ranging from 0 to 4. The USS grades urgency per toilet void as none, mild, moderate or severe.
Outcome measures
| Measure |
Experimental Arm
n=28 Participants
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80 mg/40 mL) in single intravesical instillation
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
n=28 Participants
Normal saline 50 mL in single intravesical instillation
Normal saline instillation: Normal saline (BoNT-A/NS) 50 mL in single intravesical instillation
|
|---|---|---|
|
Net Change of the Urgency Severity Score (USS) Within 3 Days
Improved
|
11 participants
|
4 participants
|
|
Net Change of the Urgency Severity Score (USS) Within 3 Days
No change
|
16 participants
|
20 participants
|
|
Net Change of the Urgency Severity Score (USS) Within 3 Days
Worsen
|
1 participants
|
4 participants
|
SECONDARY outcome
Timeframe: Baseline to 4 weeks after initial treatmentEfficacy:(measured the net change of variables from baseline and 1 month) The Global response assessment (GRA) have seven point scale is centered at zero (no change): markedly worse; moderately worse; slightly worse; no change; slightly improved; moderately improved; and markedly improved.
Outcome measures
| Measure |
Experimental Arm
n=28 Participants
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80 mg/40 mL) in single intravesical instillation
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
n=28 Participants
Normal saline 50 mL in single intravesical instillation
Normal saline instillation: Normal saline (BoNT-A/NS) 50 mL in single intravesical instillation
|
|---|---|---|
|
Net Change of the Global Response Assessment (GRA)
Improved
|
21 participants
|
19 participants
|
|
Net Change of the Global Response Assessment (GRA)
No change
|
7 participants
|
9 participants
|
Adverse Events
Experimental Arm
Serious events: 1 serious events
Other events: 10 other events
Deaths: 0 deaths
Control Arm
Serious events: 2 serious events
Other events: 16 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Experimental Arm
n=31 participants at risk
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80 mg/40 mL) in single intravesical instillation
Liposome encapsulated botulinum toxin A'
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
n=31 participants at risk
Normal saline 50 mL in single intravesical instillation
Normal saline instillation'
Normal saline instillation: Normal saline (BoNT-A/NS) 50 mL in single intravesical instillation
|
|---|---|---|
|
General disorders
Hyponatremia
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
General disorders
Right Knee osteoarthritis
|
3.2%
1/31 • 3 months
|
0.00%
0/31 • 3 months
|
|
Nervous system disorders
C-spine whiplash injury with radiculopathy
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
Other adverse events
| Measure |
Experimental Arm
n=31 participants at risk
Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL in Liposome 80 mg/40 mL) in single intravesical instillation
Liposome encapsulated botulinum toxin A'
Liposome encapsulated botulinum toxin A: Liposome encapsulated BoNT-A (mixed BOTOX 200 U/10 mL water in Liposome 80 mg/40 mL water) in single intravesical instillation, one time treatment at the treatment day
|
Control Arm
n=31 participants at risk
Normal saline 50 mL in single intravesical instillation
Normal saline instillation'
Normal saline instillation: Normal saline (BoNT-A/NS) 50 mL in single intravesical instillation
|
|---|---|---|
|
Musculoskeletal and connective tissue disorders
Cervical spondylosis
|
3.2%
1/31 • 3 months
|
0.00%
0/31 • 3 months
|
|
Eye disorders
Foreign body in conjunctival SAC
|
3.2%
1/31 • 3 months
|
0.00%
0/31 • 3 months
|
|
Endocrine disorders
Diabetes mellitus
|
3.2%
1/31 • 3 months
|
0.00%
0/31 • 3 months
|
|
Gastrointestinal disorders
Irritable bowel syndrome
|
3.2%
1/31 • 3 months
|
0.00%
0/31 • 3 months
|
|
Ear and labyrinth disorders
Unspecified otitis media
|
3.2%
1/31 • 3 months
|
0.00%
0/31 • 3 months
|
|
Reproductive system and breast disorders
Chronic mastoiditis
|
3.2%
1/31 • 3 months
|
0.00%
0/31 • 3 months
|
|
General disorders
Dizziness and giddiness
|
3.2%
1/31 • 3 months
|
0.00%
0/31 • 3 months
|
|
Gastrointestinal disorders
Abdominal fullness pain
|
3.2%
1/31 • 3 months
|
0.00%
0/31 • 3 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory infection upper tract
|
3.2%
1/31 • 3 months
|
0.00%
0/31 • 3 months
|
|
Respiratory, thoracic and mediastinal disorders
Couch
|
3.2%
1/31 • 3 months
|
0.00%
0/31 • 3 months
|
|
Cardiac disorders
Ventricular tachycardia
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
Skin and subcutaneous tissue disorders
Unspecified local infections of skin and subcutaneous tissue
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
Respiratory, thoracic and mediastinal disorders
Chest pain
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
Musculoskeletal and connective tissue disorders
Fracture of ankle
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
Musculoskeletal and connective tissue disorders
Traumatic arthropathy
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
Musculoskeletal and connective tissue disorders
Fracture of clavicle
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
Nervous system disorders
Paralysis
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
Skin and subcutaneous tissue disorders
Contusion
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
Gastrointestinal disorders
Functional disorder of stomach disturbance irritation
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
Skin and subcutaneous tissue disorders
Herpes zoster
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
General disorders
Fever
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
General disorders
Oral ulcer
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
Eye disorders
Blepharitis
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
|
General disorders
Rhinitis
|
0.00%
0/31 • 3 months
|
6.5%
2/31 • 3 months
|
|
Skin and subcutaneous tissue disorders
Dermatophytosis
|
0.00%
0/31 • 3 months
|
3.2%
1/31 • 3 months
|
Additional Information
Dr. Hann-Chorng Kuo
Department of Urology, Buddhist Tzu Chi General Hospital and Tzu Chi University
Phone: 886-3-8561825
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place