Trial Outcomes & Findings for Effects of Botox in Obstructed Defecation Syndrome (NCT NCT02160288)

NCT ID: NCT02160288

Last Updated: 2020-05-12

Results Overview

The primary outcome measure will be the change from baseline in the sum of ODS-S and PAC-QoL at 1 month after the Botox injection. This measures the symptomatic improvement in ODS. ODS (altomare obstructed defecation syndrome score) scores range from 0 (minimum) to 31 (maximum) where 31 is the most severe (worse outcome) and 0 is no symptoms (better outcome). The scale for PAC-QoL (Patient Assessment of Constipation- Quality of life score) is a minimum of 0 and a maximum of 112 where 0 is highest quality of life (better outcome) and 112 is lowest quality of life (worse outcome) Larger number for change would suggest a better outcome

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 1 participants
• Primary outcome timeframe: Baseline, 1 month after injection
• Results posted on: 2020-05-12

Participant Flow

Participant milestones

Measure	Botulinum Toxin-A Patients will receive Botulinum Toxin-A (Botox) injected in the puborectalis muscle and external anal sphincter. We will use 100 units of Botox, a dose with a good safety profile that has been proven to work in previous studies performed in adults. Botulinum Toxin-A: 100 Units diluted in a 5 cc syringe at a concentration of 20U/mL	Normal Saline Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter. Normal Saline: Dispensed in a 5 cc syringe
Overall Study STARTED	1	0
Overall Study COMPLETED	1	0
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	Botulinum Toxin-A n=1 Participants Patients will receive Botulinum Toxin-A (Botox) injected in the puborectalis muscle and external anal sphincter. We will use 100 units of Botox, a dose with a good safety profile that has been proven to work in previous studies performed in adults. Botulinum Toxin-A: 100 Units diluted in a 5 cc syringe at a concentration of 20U/mL	Normal Saline Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter. Normal Saline: Dispensed in a 5 cc syringe	Total n=1 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=1 Participants	0 Participants	0 Participants n=1 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=1 Participants	0 Participants	1 Participants n=1 Participants
Age, Categorical >=65 years	0 Participants n=1 Participants	0 Participants	0 Participants n=1 Participants
Sex: Female, Male Female	0 Participants n=1 Participants	0 Participants	0 Participants n=1 Participants
Sex: Female, Male Male	1 Participants n=1 Participants	0 Participants	1 Participants n=1 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment United States	1 Participants n=1 Participants	0 Participants	1 Participants n=1 Participants
ODS score	20 units on a scale n=1 Participants	—	20 units on a scale n=1 Participants

PRIMARY outcome

Timeframe: Baseline, 1 month after injection

Population: since there was only one participant, analysis of the data is limited

The primary outcome measure will be the change from baseline in the sum of ODS-S and PAC-QoL at 1 month after the Botox injection.

This measures the symptomatic improvement in ODS. ODS (altomare obstructed defecation syndrome score) scores range from 0 (minimum) to 31 (maximum) where 31 is the most severe (worse outcome) and 0 is no symptoms (better outcome).

The scale for PAC-QoL (Patient Assessment of Constipation- Quality of life score) is a minimum of 0 and a maximum of 112 where 0 is highest quality of life (better outcome) and 112 is lowest quality of life (worse outcome)

Larger number for change would suggest a better outcome

Outcome measures

Measure	Botulinum Toxin-A n=1 Participants Patients will receive Botulinum Toxin-A (Botox) injected in the puborectalis muscle and external anal sphincter. We will use 100 units of Botox, a dose with a good safety profile that has been proven to work in previous studies performed in adults. Botulinum Toxin-A: 100 Units diluted in a 5 cc syringe at a concentration of 20U/mL	Normal Saline Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter. Normal Saline: Dispensed in a 5 cc syringe
Changes in the Altomare Obstructed Defecation Syndrome- Score (ODS-S) and Patient Assessment of Constipation- Quality of Life Score (PAC-QoL)	3 change in score on a scale	—

SECONDARY outcome

Timeframe: Assessed at baseline, 1, 3, 6 and 12 months after injection and value compared from baseline to 12 months

Population: since there was only one participant, analysis of the data is limited

The scale title is obstructed defecation score, the minimum value is 0 and the maximum value is 31 for the ODS score. This measures the symptomatic improvement in ODS. ODS scores range from 0-31 where 31 is the most severe and 0 is no symptoms. A higher score would suggest a better outcome.

Outcome measures

Measure	Botulinum Toxin-A n=1 Participants Patients will receive Botulinum Toxin-A (Botox) injected in the puborectalis muscle and external anal sphincter. We will use 100 units of Botox, a dose with a good safety profile that has been proven to work in previous studies performed in adults. Botulinum Toxin-A: 100 Units diluted in a 5 cc syringe at a concentration of 20U/mL	Normal Saline Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter. Normal Saline: Dispensed in a 5 cc syringe
Changes in the Altomare ODS Score (ODS-S)	18 score on a scale	—

SECONDARY outcome

Timeframe: Assessed at baseline, 1, 3, 6 and 12 months after injection and value compared from baseline to 12 months

Population: since there was only one participant, analysis of the data is limited

PAC-QoL is Patient Assessment of Constipation- Quality of Life Score. The scale for PAC-QoL is 0 minimum score to 112 maximum score where 0 is highest quality of life (better outcome) and 112 is lowest quality of life (worse outcome). Since we are looking for a change in score, the minimum change would be 0 and the maximum change would be 112. A higher change would suggest a better outcome.

Outcome measures

Measure	Botulinum Toxin-A n=1 Participants Patients will receive Botulinum Toxin-A (Botox) injected in the puborectalis muscle and external anal sphincter. We will use 100 units of Botox, a dose with a good safety profile that has been proven to work in previous studies performed in adults. Botulinum Toxin-A: 100 Units diluted in a 5 cc syringe at a concentration of 20U/mL	Normal Saline Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter. Normal Saline: Dispensed in a 5 cc syringe
Changes in the Patient Assessment of Constipation- Quality of Life Score (PAC-QoL)	21 change in score on a scale	—

SECONDARY outcome

Timeframe: Baseline, 1, 3, 6 and 12 months after injection and value compared from baseline to 12 months

Population: since there was only one participant, analysis of the data is limited

Medical outcomes survey short form (SF-36 version 1) will be used to measure changes in the health-related quality of life.This is a measure of efficacy. The score ranges from 0-100 where 0 is maximum impact on health and 100 is no impact on health

Outcome measures

Measure	Botulinum Toxin-A n=1 Participants Patients will receive Botulinum Toxin-A (Botox) injected in the puborectalis muscle and external anal sphincter. We will use 100 units of Botox, a dose with a good safety profile that has been proven to work in previous studies performed in adults. Botulinum Toxin-A: 100 Units diluted in a 5 cc syringe at a concentration of 20U/mL	Normal Saline Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter. Normal Saline: Dispensed in a 5 cc syringe
Changes in Health-Related Quality of Life	1 change in score on a scale	—

SECONDARY outcome

Timeframe: Assessed at baseline, 1, 3, 6 and 12 months after injection and value compared from baseline to 12 months

Population: since there was only one participant, analysis of the data is limited

This is a measure of Botox-specific adverse events. The score is a total of answers of questions. The score range is 0-20 where 0 is perfect incontinence and 20 is complete incontinence.

Outcome measures

Measure	Botulinum Toxin-A n=1 Participants Patients will receive Botulinum Toxin-A (Botox) injected in the puborectalis muscle and external anal sphincter. We will use 100 units of Botox, a dose with a good safety profile that has been proven to work in previous studies performed in adults. Botulinum Toxin-A: 100 Units diluted in a 5 cc syringe at a concentration of 20U/mL	Normal Saline Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter. Normal Saline: Dispensed in a 5 cc syringe
Changes in Cleveland Clinic Fecal Incontinence Score (CCFI)	2 change in score on a scale	—

SECONDARY outcome

Timeframe: Assessed at baseline, 1, 3, 6 and 12 months after injection and value compared from baseline to 12 months

Population: since there was only one participant, analysis of the data is limited

This is a measure of Botox-specific adverse events. This fecal incontinence quality of life scale (FIQoL) scale ranges from 4 as the minimum and 20 as maximum. lower score indicates lower quality of life (worse outcome) and higher score has better quality of life (better outcome).

Outcome measures

Measure	Botulinum Toxin-A n=1 Participants Patients will receive Botulinum Toxin-A (Botox) injected in the puborectalis muscle and external anal sphincter. We will use 100 units of Botox, a dose with a good safety profile that has been proven to work in previous studies performed in adults. Botulinum Toxin-A: 100 Units diluted in a 5 cc syringe at a concentration of 20U/mL	Normal Saline Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter. Normal Saline: Dispensed in a 5 cc syringe
Changes in Fecal Incontinence Quality of Life Scale (FIQoL)	18 change in score on a scale	—

SECONDARY outcome

Timeframe: Baseline, 1 month follow-up visit

Population: since there was only one participant, analysis of the data is limited

This is a measure of efficacy.

Outcome measures

Measure	Botulinum Toxin-A n=1 Participants Patients will receive Botulinum Toxin-A (Botox) injected in the puborectalis muscle and external anal sphincter. We will use 100 units of Botox, a dose with a good safety profile that has been proven to work in previous studies performed in adults. Botulinum Toxin-A: 100 Units diluted in a 5 cc syringe at a concentration of 20U/mL	Normal Saline Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter. Normal Saline: Dispensed in a 5 cc syringe
Relaxation of Puborectalis With Push Measured by EMG	0 participants	—

SECONDARY outcome

Timeframe: Baseline, 1 month follow-up visit

Population: since there was only one participant, analysis of the data is limited

This is a measure of efficacy.

Outcome measures

Measure	Botulinum Toxin-A n=1 Participants Patients will receive Botulinum Toxin-A (Botox) injected in the puborectalis muscle and external anal sphincter. We will use 100 units of Botox, a dose with a good safety profile that has been proven to work in previous studies performed in adults. Botulinum Toxin-A: 100 Units diluted in a 5 cc syringe at a concentration of 20U/mL	Normal Saline Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter. Normal Saline: Dispensed in a 5 cc syringe
Success of Balloon Expulsion Test	1 Participants	—

SECONDARY outcome

Timeframe: Baseline, 1 month follow-up visit

Population: since there was only one participant, analysis of the data is limited

The changes in anal sphincter function will be assessed by anorectal manometry. This test will measure the resting sphincter pressure, maximal resting anal pressure, maximum squeeze pressure. The change in anal sphincter function is a measure of treatment efficacy. The minimum score for resting sphincter pressure is 0 and the maximum is 200. The minimum squeeze pressure is 10 and the maximum is 200. The lower score is suggested of decreased tone and worse outcome, where high scores may suggest worse outcome in terms of sphincter function.

Outcome measures

Measure	Botulinum Toxin-A n=1 Participants Patients will receive Botulinum Toxin-A (Botox) injected in the puborectalis muscle and external anal sphincter. We will use 100 units of Botox, a dose with a good safety profile that has been proven to work in previous studies performed in adults. Botulinum Toxin-A: 100 Units diluted in a 5 cc syringe at a concentration of 20U/mL	Normal Saline Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter. Normal Saline: Dispensed in a 5 cc syringe
Change in Anal Sphincter Function	56.3 change of score	—

SECONDARY outcome

Timeframe: Baseline, 1 month follow-up visit

Population: since there was only one participant, analysis of the data is limited

The defecation index=maximum rectal pressure during attempted defecation/minimum anal residual pressure during attempted defecation. This is calculated based on measurements obtained from anorectal manometry and is a measure of treatment efficacy. Scores can change from 0 (meaning no change- least effective) to 1 meaning that the change shows complete relaxation with defecation.

Outcome measures

Measure	Botulinum Toxin-A n=1 Participants Patients will receive Botulinum Toxin-A (Botox) injected in the puborectalis muscle and external anal sphincter. We will use 100 units of Botox, a dose with a good safety profile that has been proven to work in previous studies performed in adults. Botulinum Toxin-A: 100 Units diluted in a 5 cc syringe at a concentration of 20U/mL	Normal Saline Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter. Normal Saline: Dispensed in a 5 cc syringe
Change in Defecation Index	0 change in score on a scale	—

Adverse Events

Botulinum Toxin-A

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Normal Saline

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr Liliana Bordeianou

Massachusetts General Hospital

Phone: 6176430541

Email: lsavitt@mgh.harvard.edu

Results disclosure agreements

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