Ultrasound and EMG Guided Botox Injection for the Treatment of Non-Relaxing Puborectalis Syndrome

Phase 2

Terminated

Conditions: Non-relaxing Puborectalis Syndrome

Interventions: Drug: botulinum toxin

Registration Number: NCT01780636

Lead Sponsor: Karen Noblett

Brief Summary: Our study aims to determine the efficacy of ultrasound and EMG guided Botox injections into the puborectalis muscle in women with non-relaxing puborectalis syndrome. With use of this technique, we believe we will find that Botox injections decrease symptom severity and improve quality of life (QOL) in patients with NRPS. All enrolled patients will receive ultrasound and EMG guided Botox injections at three points along the puborectalis muscle. Post-injection data will be obtained with a defecation diary and questionnaire data at the 2-week, 3-month, and 6-month intervals.

Detailed Description: Non-relaxing puborectalis syndrome (NRPS) is an obstructive defecatory disorder that presents with chronic constipation, anismus, or a sensation of incomplete rectal emptying. The specific etiology of this syndrome is unknown. The true incidence of NRPS is unknown, but it is estimated that 4% of patients with defecatory disorders have NRPS. Previous therapies for non-relaxing PRS have included biofeedback, pelvic floor physical therapy, dietary changes, laxatives, enemas, and surgical division of the puborectalis muscle. Unfortunately, responses to these treatments have been variable at best. Direct injection of botulinum toxin (Botox) into the puborectalis muscle is an alternative approach for treating NRPS and has recently been reported in a limited number of studies. Botox blocks cholinergic nerve endings in the autonomic nervous system and has been used to weaken the puborectalis muscle in constipated patients.

Our study aims to determine the efficacy of ultrasound and EMG guided Botox injections into the puborectalis muscle in women with NRPS. With use of this technique, we believe we will find that Botox injections decrease symptom severity and improve quality of life (QOL) in patients with NRPS.

We plan to compare patient reported symptoms, symptom severity, and QOL prior to Botox injections with symptoms and QOL at 2-week, 3-month, and 6-month intervals after Botox injection into the puborectalis muscle in patients with NRPS.

All enrolled patients will receive ultrasound and EMG guided Botox injections at three points along the puborectalis muscle. Post-injection data will be obtained with a defecation diary and questionnaire data at the 2-week, 3-month, and 6-month intervals. Additional injections will be offered to patients who fail the initial treatment.

Recruitment & Eligibility

Status: TERMINATED

Sex: Female

Target Recruitment: 7

Inclusion Criteria

Female
18 years of age or older
Diagnosis of non-relaxing puborectalis syndrome

Exclusion Criteria

Allergy or sensitivity to botulinum toxin
Pregnancy or plan to become pregnant in the subsequent 6 months after injection
Undiagnosed ano-rectal mass, lesion, or infection
Stage II or greater rectocele

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Botulinum toxin (Botox) injection	botulinum toxin	All patients will be given the active Botox injection and thus this study will remain open-label and non-randomized as this is a pilot study to determine initial efficacy.

Primary Outcome Measures

Name	Time	Method
PAC-SYM and Wexner Scores	Baseline to 6 months	Scores on a scale The baseline scores were compared to scores at 6 months to evaluate the degree of improvement
Change in Symptoms Based on Scores Using the PAC-SYM and Wexner Constipation Questionnaires.	Baseline to 6 months post-injection	To assess whether Botox injections into the puborectalis muscle will improve patient's symptoms based on scores using the PAC-SYM and Wexner Constipation validated questionnaires from baseline to 6 months post-Botox injection to determine severity of symptoms. The PAC-SYM questionnaire unabbreviated title is Patient Assessment of Constipation-Symptoms and the total score is summed ranging from 0 to 44. The Wexner Constipation questionnaire total score is summed ranging from 0 to 30. Both questionnaires have higher values representing a worse outcome.

Secondary Outcome Measures

Name	Time	Method
Change in Pressures on Anal Manometry	Baseline to 6 months post-injection	To assess the change in pressures on anal manometry before and after Botox injections into the puborectalis muscle from baseline to 6 months post-Botox injection. Anal manometry is a test done in clinic to assess bowel function. Average resting pressure is recorded using the advanced diagnostic equipment. Above 40 mmHg is normal for resting pressure. Also, average squeeze pressure will be recorded. Greater than 100 mmHg is normal for average squeeze pressure.