Trial Outcomes & Findings for Botulin-A Toxin Instillations and Overactive Bladder (NCT NCT00583219)

NCT ID: NCT00583219

Last Updated: 2014-04-21

Results Overview

The study coordinator instructed the subject to keep record of any incontinence episodes during the 24-hour period prior to their baseline and follow-up visits.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 25 participants
• Primary outcome timeframe: baseline, 1 month, 3 months
• Results posted on: 2014-04-21

Participant Flow

Subjects were recruited from November 2006 to March 2009 at Mayo Clinic in Jacksonville, Florida.

Participant milestones

Measure	Botox/DMSO Solution Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Phase 1 STARTED	9
Phase 1 COMPLETED	9
Phase 1 NOT COMPLETED	0
Phase 2 STARTED	22
Phase 2 COMPLETED	21
Phase 2 NOT COMPLETED	1

Reasons for withdrawal

Measure

Botox/DMSO Solution Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.

Phase 2 Withdrawal by Subject

1

Baseline Characteristics

Botulin-A Toxin Instillations and Overactive Bladder

Baseline characteristics by cohort

Measure	Botox/DMSO Solution n=25 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Age, Continuous	74 years n=5 Participants
Sex: Female, Male Female	25 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Region of Enrollment United States	25 participants n=5 Participants
Body Mass Index	26.6 kg/m^2 n=5 Participants

PRIMARY outcome

Timeframe: baseline, 1 month, 3 months

The study coordinator instructed the subject to keep record of any incontinence episodes during the 24-hour period prior to their baseline and follow-up visits.

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Median Number of Incontinent Episodes During 24 Hours Baseline	4 number of incontinent episodes Interval 2.0 to 9.0
Median Number of Incontinent Episodes During 24 Hours 1 month	2 number of incontinent episodes Interval 0.0 to 5.0
Median Number of Incontinent Episodes During 24 Hours 3 months	4 number of incontinent episodes Interval 2.0 to 6.0

SECONDARY outcome

Timeframe: baseline, 1 month, 3 months

Prior to baseline and follow-up visits, the study coordinator weighed standard pads provided to the subject for the study time period. The study coordinator instructed the subject to bring in any pads used during the 24-hour period prior to baseline and follow up visits. The study coordinator recorded the 24-hour pad weights into the study dataset.

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Median 24 Hour Pad Weight Baseline	135 g Interval 6.0 to 259.0
Median 24 Hour Pad Weight 1 month	46 g Interval 9.0 to 183.0
Median 24 Hour Pad Weight 3 months	55 g Interval 11.0 to 318.0

SECONDARY outcome

Timeframe: baseline

The Blaivas-Groutz Anti-incontinence scale was used as a measure of urinary incontinence. This scale combines information on the number of incontinent episodes in a 24-hour period, 24-hour pad weights, and a qualitative rating by the patient into a single score ranging from 0 to 6. This score is then used to categorize incontinence as none (0), mild (1-2), moderate (3-4), or severe (5-6).

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Blaivas-Groutz Anti-Incontinence Score at Baseline None	0 participants (per category)
Blaivas-Groutz Anti-Incontinence Score at Baseline Mild	4 participants (per category)
Blaivas-Groutz Anti-Incontinence Score at Baseline Moderate	1 participants (per category)
Blaivas-Groutz Anti-Incontinence Score at Baseline Severe	16 participants (per category)

SECONDARY outcome

Timeframe: 1 month after treatment

The Blaivas-Groutz Anti-incontinence scale was used as a measure of urinary incontinence. This scale combines information on the number of incontinent episodes in a 24-hour period, 24-hour pad weights, and a qualitative rating by the patient into a single score ranging from 0 to 6. This score is then used to categorize incontinence as none (0), mild (1-2), moderate (3-4), or severe (5-6).

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Blaivas-Groutz Anti-Incontinence Score at 1 Month None	1 participants (per category)
Blaivas-Groutz Anti-Incontinence Score at 1 Month Mild	5 participants (per category)
Blaivas-Groutz Anti-Incontinence Score at 1 Month Moderate	5 participants (per category)
Blaivas-Groutz Anti-Incontinence Score at 1 Month Severe	10 participants (per category)

SECONDARY outcome

Timeframe: 3 months after treatment

The Blaivas-Groutz Anti-incontinence scale was used as a measure of urinary incontinence. This scale combines information on the number of incontinent episodes in a 24-hour period, 24-hour pad weights, and a qualitative rating by the patient into a single score ranging from 0 to 6. This score is then used to categorize incontinence as none (0), mild (1-2), moderate (3-4), or severe (5-6).

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Blaivas-Groutz Anti-Incontinence Score at 3 Months Severe	13 participants (per category)
Blaivas-Groutz Anti-Incontinence Score at 3 Months None	0 participants (per category)
Blaivas-Groutz Anti-Incontinence Score at 3 Months Mild	2 participants (per category)
Blaivas-Groutz Anti-Incontinence Score at 3 Months Moderate	6 participants (per category)

SECONDARY outcome

Timeframe: baseline, 1 month, 3 months

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Mean Number of Pads Per Day Baseline	4 pads per day Interval 2.0 to 7.0
Mean Number of Pads Per Day 1 month	3 pads per day Interval 1.0 to 5.0
Mean Number of Pads Per Day 3 months	3 pads per day Interval 2.0 to 5.0

SECONDARY outcome

Timeframe: baseline, 1 month, 3 months

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Postvoid Residual Baseline	8 mL Interval 0.0 to 24.0
Postvoid Residual 1 month	19 mL Interval 0.0 to 32.0
Postvoid Residual 3 months	24 mL Interval 9.0 to 65.0

SECONDARY outcome

Timeframe: baseline, 1 month, 3 months

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Urine Culture baseline negative	16 participants
Urine Culture baseline positive	5 participants
Urine Culture 1 month negative	16 participants
Urine Culture 1 month positive	5 participants
Urine Culture 3 months negative	13 participants
Urine Culture 3 months positive	8 participants

SECONDARY outcome

Timeframe: baseline

Urinary urgency was measured by the Indevus Urgency Severity Scale (IUSS). The IUSS asks patients to assess the severity of 'urgency' at each void. The scale employs the following wording: "Degree of urgency is meant to describe your urge to urinate. Sometimes you may feel a very strong urge to urinate and at other times, you may feel a milder urge prior to the onset of a toilet void. Rate this feeling by circling 0, 1, 2, or 3, defined as: 0: NONE - no urgency, 1: MILD - awareness of urgency, but it is easily tolerated and you can continue with your usual activity or tasks, 2: MODERATE - enough urgency discomfort that it interferes with or shortens your usual activity or tasks, 3: SEVERE - extreme urgency discomfort that abruptly stops all activity or tasks."

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Urinary Urgency at Baseline None	0 participants (per category)
Urinary Urgency at Baseline Mild	2 participants (per category)
Urinary Urgency at Baseline Moderate	8 participants (per category)
Urinary Urgency at Baseline Severe	11 participants (per category)

SECONDARY outcome

Timeframe: 1 month after treatment

Urinary urgency was measured by the Indevus Urgency Severity Scale (IUSS). The IUSS asks patients to assess the severity of 'urgency' at each void. The scale employs the following wording: "Degree of urgency is meant to describe your urge to urinate. Sometimes you may feel a very strong urge to urinate and at other times, you may feel a milder urge prior to the onset of a toilet void. Rate this feeling by circling 0, 1, 2, or 3, defined as: 0: NONE - no urgency, 1: MILD - awareness of urgency, but it is easily tolerated and you can continue with your usual activity or tasks, 2: MODERATE - enough urgency discomfort that it interferes with or shortens your usual activity or tasks, 3: SEVERE - extreme urgency discomfort that abruptly stops all activity or tasks."

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Urinary Urgency at 1 Month None	0 participants (per category)
Urinary Urgency at 1 Month Mild	13 participants (per category)
Urinary Urgency at 1 Month Moderate	7 participants (per category)
Urinary Urgency at 1 Month Severe	1 participants (per category)

SECONDARY outcome

Timeframe: 3 months after treatment

Urinary urgency was measured by the Indevus Urgency Severity Scale (IUSS). The IUSS asks patients to assess the severity of 'urgency' at each void. The scale employs the following wording: "Degree of urgency is meant to describe your urge to urinate. Sometimes you may feel a very strong urge to urinate and at other times, you may feel a milder urge prior to the onset of a toilet void. Rate this feeling by circling 0, 1, 2, or 3, defined as: 0: NONE - no urgency, 1: MILD - awareness of urgency, but it is easily tolerated and you can continue with your usual activity or tasks, 2: MODERATE - enough urgency discomfort that it interferes with or shortens your usual activity or tasks, 3: SEVERE - extreme urgency discomfort that abruptly stops all activity or tasks."

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Urinary Urgency at 3 Months None	0 participants (per category)
Urinary Urgency at 3 Months Mild	11 participants (per category)
Urinary Urgency at 3 Months Moderate	7 participants (per category)
Urinary Urgency at 3 Months Severe	3 participants (per category)

SECONDARY outcome

Timeframe: baseline, 1 month, 3 months

The UDI-6 was one measure of urinary-associated quality of life. The UDI-6 questionnaire has 6 items, scored from 0 (not at all) to 3 (greatly), with total scores ranging from 0 to 18, a higher score indicating greater distress.

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Median Urogenital Distress Inventory (UDI-6) Scores Baseline	10 units on a scale Interval 8.0 to 12.0
Median Urogenital Distress Inventory (UDI-6) Scores 1 month	5 units on a scale Interval 4.0 to 9.0
Median Urogenital Distress Inventory (UDI-6) Scores 3 months	6 units on a scale Interval 4.0 to 8.0

SECONDARY outcome

Timeframe: baseline, 1 month, 3 months

The bothersomeness refers to the question: On a scale of 1-10 (0 is not at all; 10 is intolerable), how badly does loss of urinary control bother you?

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Bothersomeness Baseline	9 units on a scale Interval 8.0 to 10.0
Bothersomeness 1 month	5 units on a scale Interval 3.0 to 8.0
Bothersomeness 3 months	5 units on a scale Interval 2.0 to 7.0

SECONDARY outcome

Timeframe: baseline, 1 month, 3 months

The IIQ-7 was one measure of urinary-associated quality of life. The IIQ-7 questionnaire has 7 items, scored from 0 (not at all) to 3 (greatly), with total scores ranging from 0 to 21, a higher score indicating greater distress.

Outcome measures

Measure	Botox/DMSO Solution n=21 Participants Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. The 6 subjects in the Phase 1 trial who received 300 units of botulinum-A toxin and 50cc DMSO also continued in the Phase 2 trial. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Incontinence Impact Questionnaire-short Form (IIQ-7) Scores Baseline	13 units on a scale Interval 10.0 to 18.0
Incontinence Impact Questionnaire-short Form (IIQ-7) Scores 1 month	7 units on a scale Interval 2.0 to 11.0
Incontinence Impact Questionnaire-short Form (IIQ-7) Scores 3 months	6 units on a scale Interval 2.0 to 12.0

Adverse Events

Botox/DMSO Solution

Serious events: 0 serious events

Other events: 18 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Botox/DMSO Solution n=25 participants at risk Subjects received Botulinum-A toxin and Dimethyl sulfoxide solution. The first 3 subjects in Phase 1 underwent bladder instillation of 50 cc of the solution utilizing 200 units of botulinum-A toxin and 50cc DMSO. The next 6 subjects in the Phase 1 trial received 300 units of botulinum-A toxin and 50cc DMSO. All subjects in the Phase 2 trial received 300 units of botulinum-A toxin and 50cc DMSO. Botulinum-A toxin: A simple bladder catheterization in the office with a standard urinary catheter and instill the Botulinum-A toxin/DMSO solution. Dimethyl sulfoxide (DMSO): Subjects received 50 cc of DMSO in an aqueous solution for intravesical instillation with botulinum-A toxin. Each cc contained 0.54 gm DMSO.
Renal and urinary disorders Pelvic soreness in first 24 hours	4.0% 1/25 • Number of events 1 • Phase 1 patients (9) were assess immediately, 4 hours 24 hours, and 7 days after instillation. Phase 2 patients were assessed immediately, 2 hours, and 4 hours after instillation. All patients were assessed at 1 month and 3 month follow-up intervals.
General disorders Positional light-headedness in first 24 hours	4.0% 1/25 • Number of events 1 • Phase 1 patients (9) were assess immediately, 4 hours 24 hours, and 7 days after instillation. Phase 2 patients were assessed immediately, 2 hours, and 4 hours after instillation. All patients were assessed at 1 month and 3 month follow-up intervals.
Psychiatric disorders Anxiety in first 24 hours	4.0% 1/25 • Number of events 1 • Phase 1 patients (9) were assess immediately, 4 hours 24 hours, and 7 days after instillation. Phase 2 patients were assessed immediately, 2 hours, and 4 hours after instillation. All patients were assessed at 1 month and 3 month follow-up intervals.
Renal and urinary disorders Bladder spasms in first 24 hours	8.0% 2/25 • Number of events 2 • Phase 1 patients (9) were assess immediately, 4 hours 24 hours, and 7 days after instillation. Phase 2 patients were assessed immediately, 2 hours, and 4 hours after instillation. All patients were assessed at 1 month and 3 month follow-up intervals.
Renal and urinary disorders Urethral discomfort in first 24 hours	8.0% 2/25 • Number of events 2 • Phase 1 patients (9) were assess immediately, 4 hours 24 hours, and 7 days after instillation. Phase 2 patients were assessed immediately, 2 hours, and 4 hours after instillation. All patients were assessed at 1 month and 3 month follow-up intervals.
Renal and urinary disorders Dysuria in first 24 hours	8.0% 2/25 • Number of events 2 • Phase 1 patients (9) were assess immediately, 4 hours 24 hours, and 7 days after instillation. Phase 2 patients were assessed immediately, 2 hours, and 4 hours after instillation. All patients were assessed at 1 month and 3 month follow-up intervals.
Renal and urinary disorders Urinary tract infection after 24 hours	12.0% 3/25 • Number of events 3 • Phase 1 patients (9) were assess immediately, 4 hours 24 hours, and 7 days after instillation. Phase 2 patients were assessed immediately, 2 hours, and 4 hours after instillation. All patients were assessed at 1 month and 3 month follow-up intervals.
Reproductive system and breast disorders Perineal rash 7 days after injection	4.0% 1/25 • Number of events 1 • Phase 1 patients (9) were assess immediately, 4 hours 24 hours, and 7 days after instillation. Phase 2 patients were assessed immediately, 2 hours, and 4 hours after instillation. All patients were assessed at 1 month and 3 month follow-up intervals.
Renal and urinary disorders Throbbing with urination 7 days after injection	4.0% 1/25 • Number of events 1 • Phase 1 patients (9) were assess immediately, 4 hours 24 hours, and 7 days after instillation. Phase 2 patients were assessed immediately, 2 hours, and 4 hours after instillation. All patients were assessed at 1 month and 3 month follow-up intervals.
Renal and urinary disorders Dysuria after 24 hours	16.0% 4/25 • Number of events 4 • Phase 1 patients (9) were assess immediately, 4 hours 24 hours, and 7 days after instillation. Phase 2 patients were assessed immediately, 2 hours, and 4 hours after instillation. All patients were assessed at 1 month and 3 month follow-up intervals.
Renal and urinary disorders Bladder spasms after 24 hours	4.0% 1/25 • Number of events 1 • Phase 1 patients (9) were assess immediately, 4 hours 24 hours, and 7 days after instillation. Phase 2 patients were assessed immediately, 2 hours, and 4 hours after instillation. All patients were assessed at 1 month and 3 month follow-up intervals.
Renal and urinary disorders Urethral discomfort after 24 hours	4.0% 1/25 • Number of events 1 • Phase 1 patients (9) were assess immediately, 4 hours 24 hours, and 7 days after instillation. Phase 2 patients were assessed immediately, 2 hours, and 4 hours after instillation. All patients were assessed at 1 month and 3 month follow-up intervals.
Renal and urinary disorders Pelvic tenderness after 24 hours	8.0% 2/25 • Number of events 2 • Phase 1 patients (9) were assess immediately, 4 hours 24 hours, and 7 days after instillation. Phase 2 patients were assessed immediately, 2 hours, and 4 hours after instillation. All patients were assessed at 1 month and 3 month follow-up intervals.

Additional Information

Dr. Steven P. Petrou

Mayo Clinic

Phone: 904-953-7330

Email: petrou.steven@mayo.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place