Trial Outcomes & Findings for Optimization of OnabotulinumtoxinA (BTX-A) Injection for the Treatment of Neurogenic Lower Urinary Tract Dysfunction (NCT NCT06059066)

Last Updated: 2025-05-09

Results Overview

Participants were asked to rate their willingness to repeat the procedure using an 11-point visual analog scale (VAS). A score of 0 ("Never") indicates complete unwillingness, representing the least favorable outcome, while a score of 11 ("Definitely") reflects strong willingness, representing the most favorable outcome.

Recruitment status

COMPLETED

Study phase

Target enrollment

78 participants

Primary outcome timeframe

Immediately post-procedure and 6 weeks post-procedure

Results posted on

2025-05-09

Participant Flow

Participant milestones

Participant milestones
Measure	Standard Injection Sites Standard number of injections Standard number of injection sites: 200 units or 300 units BTX-A reconstituted in 20 mL normal saline, injected into the bladder detrusor in 20 separate injections of 1 mL including the trigone.	Reduced Injection Sites Reduced number of injections Reduced number of injection sites: 200 units or 300 units BTX-A reconstituted in 5 mL normal saline, injected into the bladder detrusor in 5 separate injections of 1 mL including the trigone.
Overall Study STARTED	39	39
Overall Study COMPLETED	39	39
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Optimization of OnabotulinumtoxinA (BTX-A) Injection for the Treatment of Neurogenic Lower Urinary Tract Dysfunction

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Standard Injection Sites (20) n=39 Participants Standard number of injection sites: 200 units or 300 units BTX-A reconstituted in 20 mL normal saline, injected into the bladder detrusor in 20 separate injections of 1 mL including the trigone.	Reduced Injection Sites (5) n=39 Participants Reduced number of injection sites: 200 units or 300 units BTX-A reconstituted in 5 mL normal saline, injected into the bladder detrusor in 5 separate injections of 1 mL including the trigone.	Total n=78 Participants Total of all reporting groups
Age, Continuous	50.2 years STANDARD_DEVIATION 22.2 • n=5 Participants	47.7 years STANDARD_DEVIATION 19.4 • n=7 Participants	48.95 years STANDARD_DEVIATION 20.8 • n=5 Participants
Sex: Female, Male Female	31 Participants n=5 Participants	28 Participants n=7 Participants	59 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants	11 Participants n=7 Participants	19 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	11 Participants n=5 Participants	9 Participants n=7 Participants	20 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	28 Participants n=5 Participants	28 Participants n=7 Participants	56 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	6 Participants n=5 Participants	1 Participants n=7 Participants	7 Participants n=5 Participants
Race (NIH/OMB) White	27 Participants n=5 Participants	33 Participants n=7 Participants	60 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	4 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants
Medical comorbidities Multiple Sclerosis	8 participants n=5 Participants	7 participants n=7 Participants	15 participants n=5 Participants
Medical comorbidities Sacral Agenesis	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants
Medical comorbidities Spina Bifida	15 participants n=5 Participants	11 participants n=7 Participants	26 participants n=5 Participants
Medical comorbidities Spinal Cord Injury	4 participants n=5 Participants	11 participants n=7 Participants	15 participants n=5 Participants
Medical comorbidities Cerebral Vascular Accident	1 participants n=5 Participants	4 participants n=7 Participants	5 participants n=5 Participants
Medical comorbidities Parkinsons Disease	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Medical comorbidities Chronic Inflammatory Demyelinating Polyneuropathy	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Medical comorbidities Syringomyelia	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Medical comorbidities Transverse Myelitis	0 participants n=5 Participants	2 participants n=7 Participants	2 participants n=5 Participants

PRIMARY outcome

Timeframe: Immediately post-procedure and 6 weeks post-procedure

Population: Data at 6-weeks post-procedure were available for 54 participants. Loss to follow-up included 10 participants from the 20-injection group and 12 participants from the 5-injection group.

Outcome measures

Outcome measures
Measure	Standard Injection Sites (20) n=38 Participants 200 units or 300 units BTX-A reconstituted in 20 mL normal saline, injected into the bladder detrusor in 20 separate injections of 1 mL including the trigone.	Reduced Injection Sites (5) n=38 Participants 200 units or 300 units BTX-A reconstituted in 5 mL normal saline, injected into the bladder detrusor in 5 separate injections of 1 mL including the trigone.
Willingness to Repeat Procedure Post-injection willingness to repeat procedure rating (VAS score)	9.16 units on a scale Standard Deviation 1.46	9.34 units on a scale Standard Deviation 1.26
Willingness to Repeat Procedure 6-weeks post-procedure willingness to repeat procedure rating (VAS score)	9.73 units on a scale Standard Deviation 1.12	9.67 units on a scale Standard Deviation 0.99

SECONDARY outcome

Timeframe: Baseline and 6-weeks after BTX-A injections

Population: Data at 6-weeks post-procedure were available for 54 participants. Loss to follow-up included 10 participants from the 20-injection group and 12 participants from the 5-injection group.

The ICIQ-SF is a standardized survey that assesses the frequency, severity, and impact of urinary incontinence (UI) on quality of life (QoL). The total score, ranging from 0 to 21, is calculated from three questions, with higher scores indicating more severe symptoms. A score of 0 reflects no leakage and no impact on QoL. Question 1 measures how often leakage occurs (0-5), Question 2 assesses the amount of leakage (0-6), and Question 3 evaluates the impact on QoL (0-10), with higher scores indicating worse outcomes. We report the mean difference in total ICIQ and QoL scores from baseline to 6-weeks post-treatment.

Outcome measures

Outcome measures
Measure	Standard Injection Sites (20) n=28 Participants 200 units or 300 units BTX-A reconstituted in 20 mL normal saline, injected into the bladder detrusor in 20 separate injections of 1 mL including the trigone.	Reduced Injection Sites (5) n=26 Participants 200 units or 300 units BTX-A reconstituted in 5 mL normal saline, injected into the bladder detrusor in 5 separate injections of 1 mL including the trigone.
Change in Neurogenic Bladder Symptoms After BTX-A Treatment - International Consultation on Incontinence Questionnaire-Urinary Incontinence Short Form (ICIQ-UI SF) Mean difference in Total ICIQ score	-4.11 units on a scale Standard Deviation 8.25	-5.23 units on a scale Standard Deviation 6.55
Change in Neurogenic Bladder Symptoms After BTX-A Treatment - International Consultation on Incontinence Questionnaire-Urinary Incontinence Short Form (ICIQ-UI SF) Mean difference in ICIQ QoL score	-2.33 units on a scale Standard Deviation 4.38	-3.54 units on a scale Standard Deviation 3.70

SECONDARY outcome

Timeframe: Baseline and 6-weeks after BTX-A injections

Population: Data at 6-weeks post-procedure were available for 54 participants. Loss to follow-up included 10 participants from the 20-injection group and 12 participants from the 5-injection group.

The NBSS-SF is a validated survey with 10 questions that measure bladder symptoms across 3 different domains: incontinence (score range: 0-12), storage and voiding (score range: 0-9), and consequences (score range: 0-7); the highest score is associated with worse symptoms. It also includes one question to assess overall quality of life (QoL) scored from 0 (pleased) to 4 (unhappy). The total score, ranging from 0 to 28, is calculated from the three domains (Q3-Q10), with 0 being the best outcome and 28 the worst outcome. Here we report the mean difference in total NBSS-SF, each of the domains and QoL scores from baseline to 6-weeks post-treatment. The NBSS-SF is a validated 10-question survey that assesses bladder symptoms across three domains: incontinence (score range: 0-12), storage and voiding (score range: 0-9), and consequences (score range: 0-7), with higher scores indicating worse symptoms. It also includes a quality of life (QoL) question (Q2) scored from 0 (pleased)

Outcome measures

Outcome measures
Measure	Standard Injection Sites (20) n=28 Participants 200 units or 300 units BTX-A reconstituted in 20 mL normal saline, injected into the bladder detrusor in 20 separate injections of 1 mL including the trigone.	Reduced Injection Sites (5) n=26 Participants 200 units or 300 units BTX-A reconstituted in 5 mL normal saline, injected into the bladder detrusor in 5 separate injections of 1 mL including the trigone.
Change in Neurogenic Bladder Symptoms After BTX-A Treatment - Neurogenic Bladder Symptom Score - Short Form (NBSS-SF) Mean difference in Total NBSS-SF	5.44 units on a scale Standard Deviation 4.19	4.65 units on a scale Standard Deviation 5.46
Change in Neurogenic Bladder Symptoms After BTX-A Treatment - Neurogenic Bladder Symptom Score - Short Form (NBSS-SF) Mean difference in NBSS-SF QoL	-1.81 units on a scale Standard Deviation 1.54	-2.15 units on a scale Standard Deviation 1.46
Change in Neurogenic Bladder Symptoms After BTX-A Treatment - Neurogenic Bladder Symptom Score - Short Form (NBSS-SF) Mean difference in NBSS-SF incontinence domain	-2.93 units on a scale Standard Deviation 2.92	-2.15 units on a scale Standard Deviation 1.46
Change in Neurogenic Bladder Symptoms After BTX-A Treatment - Neurogenic Bladder Symptom Score - Short Form (NBSS-SF) Mean difference in NBSS-SF storage and voiding domain	-2.96 units on a scale Standard Deviation 2.47	-3.04 units on a scale Standard Deviation 3.39
Change in Neurogenic Bladder Symptoms After BTX-A Treatment - Neurogenic Bladder Symptom Score - Short Form (NBSS-SF) Mean difference in NBSS-SF consequence domain	-2.63 units on a scale Standard Deviation 2.41	-3.00 units on a scale Standard Deviation 3.19

SECONDARY outcome

Timeframe: 6 weeks after BTX-A injection

Population: Data at 6-weeks post-procedure were available for 54 participants. Loss to follow-up included 10 participants from the 20-injection group and 12 participants from the 5-injection group.

The PGI-I is a validated tool for assessing patient satisfaction after therapy. It consists of a single question using a Likert scale to measure perceived improvement or worsening after BTX-A treatment, ranging from 1 ("Very much worse") to 7 ("Very much better"). Data reported here reflect average scores obtained at the six-week follow-up.

Outcome measures

Outcome measures
Measure	Standard Injection Sites (20) n=28 Participants 200 units or 300 units BTX-A reconstituted in 20 mL normal saline, injected into the bladder detrusor in 20 separate injections of 1 mL including the trigone.	Reduced Injection Sites (5) n=26 Participants 200 units or 300 units BTX-A reconstituted in 5 mL normal saline, injected into the bladder detrusor in 5 separate injections of 1 mL including the trigone.
Patient Impression of Clinical Improvement After BTX-A - Patient Global Impression of Improvement (PGI-I) Scale	5.26 units on a scale Standard Deviation 1.04	4.88 units on a scale Standard Deviation 1.09

SECONDARY outcome

Timeframe: Prior to and immediately after BTX-A injections

Participants rated their pain using the NPRS to assess discomfort during the BTX-A procedure. The NPRS is an 11-point scale (0-10), where 0 represents no pain and 10 indicates the worst pain ever experienced. Higher scores reflect greater pain intensity. We report the change in NPRS scores from before to immediately after the procedure.

Outcome measures

Outcome measures
Measure	Standard Injection Sites (20) n=38 Participants 200 units or 300 units BTX-A reconstituted in 20 mL normal saline, injected into the bladder detrusor in 20 separate injections of 1 mL including the trigone.	Reduced Injection Sites (5) n=38 Participants 200 units or 300 units BTX-A reconstituted in 5 mL normal saline, injected into the bladder detrusor in 5 separate injections of 1 mL including the trigone.
Patient Reported Procedural Discomfort - Numeric Pain Rating Scale (NPRS)	3.39 units on a scale Standard Deviation 3.01	0.57 units on a scale Standard Deviation 2.07

Adverse Events

Standard Injection Sites (20)

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Reduced Injection Sites (5)

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Standard Injection Sites (20) n=28 participants at risk Standard number of injections (20 injections): 200 units or 300 units BTX-A reconstituted in 20 mL normal saline, injected into the bladder detrusor in 20 separate injections of 1 mL including the trigone.	Reduced Injection Sites (5) n=26 participants at risk Reduced number of injections (5 injections): 200 units or 300 units BTX-A reconstituted in 5 mL normal saline, injected into the bladder detrusor in 5 separate injections of 1 mL including the trigone.
Renal and urinary disorders Urinary Tract Infection (UTI)	21.4% 6/28 • Number of events 6 • 6-8 weeks post-injection	26.9% 7/26 • Number of events 7 • 6-8 weeks post-injection
Renal and urinary disorders Catheter Clogging	0.00% 0/28 • 6-8 weeks post-injection	3.8% 1/26 • Number of events 1 • 6-8 weeks post-injection
Renal and urinary disorders New onset gross hematuria	0.00% 0/28 • 6-8 weeks post-injection	3.8% 1/26 • Number of events 1 • 6-8 weeks post-injection
Renal and urinary disorders Increased bladder spasms	0.00% 0/28 • 6-8 weeks post-injection	3.8% 1/26 • Number of events 1 • 6-8 weeks post-injection
Endocrine disorders Decreased cold tolerance	0.00% 0/28 • 6-8 weeks post-injection	3.8% 1/26 • Number of events 1 • 6-8 weeks post-injection
Endocrine disorders Hyperglycemia requiring emergency evaluation	3.6% 1/28 • Number of events 1 • 6-8 weeks post-injection	0.00% 0/26 • 6-8 weeks post-injection
Blood and lymphatic system disorders Plasmapheresis for CIDP	3.6% 1/28 • Number of events 1 • 6-8 weeks post-injection	0.00% 0/26 • 6-8 weeks post-injection

Additional Information

Julu Benoy

Houston Methodist Hospital

Phone: (713) 441-6455

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place