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

• ClinicalTrials.gov Identifier: NCT01780636
• Recruitment Status: Unknown
• First Posted: January 31, 2013
• Last Update Posted: January 31, 2013
• Sponsor: University of California, Irvine
• Collaborators: American Urogynecologic Society; Allergan
• Information provided by (Responsible Party): Samantha Reiter, University of California, Irvine

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Non-relaxing Puborectalis Syndrome	Drug: botulinum toxin	Phase 2; Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 30 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Ultrasound and EMG Guided Botox Injection for the Treatment of Non-Relaxing Puborectalis Syndrome
• Study Start Date: January 2013
• Estimated Primary Completion Date: January 2014
• Estimated Study Completion Date: July 2014

Arms and Interventions

Arm	Intervention/treatment
Experimental: Botulinum toxin (Botox) injection; 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.	Drug: botulinum toxin; Other Name: Botox (Allergan, Irvine, CA)

Outcome Measures

Primary Outcome Measures :

1. Change in symptoms based on scores using the PAC-SYM and Wexner Constipation questionnaires. [ Time Frame: 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 at baseline, 2 weeks, 3 months, and 6 months post-Botox injection to determine severity of symptoms.

Secondary Outcome Measures :

1. Change in pressures on anal manometry [ Time Frame: Baseline to 3 months post-injection ]
   To assess the change in pressures on anal manometry before and after Botox injections into the puborectalis muscle at baseline and 3 months post-Botox injection.

Other Outcome Measures:

1. Change in quality of life based on scores using the PAC-QOL questionnaire. [ Time Frame: Baseline to 6 months post-Botox injection ]
   To assess whether Botox injections into the puborectalis muscle will improve patient's quality of life based on scores using the PAC-QOL validated questionnaire at 2 weeks, 3 months, and 6 months post-Botox injection.
2. Change in the anorectal angle during defecation using defecography [ Time Frame: Baseline to 3 months post-injection ]
   To assess the change in the anorectal angle during defecation using defecography at baseline and 3 months after Botox injections into the puborectalis muscle

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: Female
• Accepts Healthy Volunteers: No

Criteria

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

Contacts and Locations

Contacts

Contact: Anthony Gaddi, MD; 714-456-8564; tgaddi@uci.edu

Contact: Samantha Reiter, MS; 714-456-6846; reiters@uci.edu

Locations

United States, California

University of California, Irvine Medical Center; Recruiting

Orange, California, United States, 92868

Principal Investigator: Karen Noblett, MD

Sub-Investigator: Anthony Gaddi, MD

Sub-Investigator: Felicia Lane, MD

Sub-Investigator: Stephanie Jacobs, MD

Sub-Investigator: Lauren Cadish, MD

Sub-Investigator: Allison Serra, MD

Sponsors and Collaborators

University of California, Irvine

American Urogynecologic Society

Allergan

Investigators

• Principal Investigator: Karen Noblett, MD; University of California, Irvine

More Information

Additional Information:

American Urogynecologic Society 

Publications:

Times ML, Reickert CA. Functional anorectal disorders. Clin Colon Rectal Surg. 2005 May;18(2):109-15. doi: 10.1055/s-2005-870892.

WASSERMAN IF. PUBORECTALIS SYNDROME (RECTAL STENOSIS DUE TO ANORECTAL SPASM). Dis Colon Rectum. 1964 Mar-Apr;7:87-98.

Kuijpers HC, Bleijenberg G. The spastic pelvic floor syndrome. A cause of constipation. Dis Colon Rectum. 1985 Sep;28(9):669-72.

Karlbom U, Hållden M, Eeg-Olofsson KE, Påhlman L, Graf W. Results of biofeedback in constipated patients: a prospective study. Dis Colon Rectum. 1997 Oct;40(10):1149-55.

Meunier P, Marechal JM, de Beaujeu MJ. Rectoanal pressures and rectal sensitivity studies in chronic childhood constipation. Gastroenterology. 1979 Aug;77(2):330-6.

MacDonald A, Shearer M, Paterson PJ, Finlay IG. Relationship between outlet obstruction constipation and obstructed urinary flow. Br J Surg. 1991 Jun;78(6):693-5.

Mellgren A, Bremmer S, Johansson C, Dolk A, Udén R, Ahlbäck SO, Holmström B. Defecography. Results of investigations in 2,816 patients. Dis Colon Rectum. 1994 Nov;37(11):1133-41. Review.

Wexner SD, Jorge JM. Colorectal physiological tests: use or abuse of technology? Eur J Surg. 1994 Mar;160(3):167-74.

Halligan S, Malouf A, Bartram CI, Marshall M, Hollings N, Kamm MA. Predictive value of impaired evacuation at proctography in diagnosing anismus. AJR Am J Roentgenol. 2001 Sep;177(3):633-6.

Salzano A, Cavallo G, De Rosa A, De Luca LE, Serpe A, Losco M. [Diagnosis with defecography of puborectal muscle syndrome]. Radiol Med. 1997 Apr;93(4):396-400. Italian.

Ganeshan A, Anderson EM, Upponi S, Planner AC, Slater A, Moore N, D'Costa H, Bungay H. Imaging of obstructed defecation. Clin Radiol. 2008 Jan;63(1):18-26. Epub 2007 Sep 14. Review.

Maria G, Brisinda G, Bentivoglio AR, Cassetta E, Albanese A. Botulinum toxin in the treatment of outlet obstruction constipation caused by puborectalis syndrome. Dis Colon Rectum. 2000 Mar;43(3):376-80.

Maria G, Cadeddu F, Brandara F, Marniga G, Brisinda G. Experience with type A botulinum toxin for treatment of outlet-type constipation. Am J Gastroenterol. 2006 Nov;101(11):2570-5. Epub 2006 Oct 4.

Ron Y, Avni Y, Lukovetski A, Wardi J, Geva D, Birkenfeld S, Halpern Z. Botulinum toxin type-A in therapy of patients with anismus. Dis Colon Rectum. 2001 Dec;44(12):1821-6.

Shafik A, El-Sibai O. Botulin toxin in the treatment of nonrelaxing puborectalis syndrome. Dig Surg. 1998;15(4):347-51.

• Responsible Party: Samantha Reiter, Karen Noblett, MD, University of California, Irvine
• ClinicalTrials.gov Identifier: NCT01780636
• Other Study ID Numbers: ALLERGAN-72570
• First Posted: January 31, 2013
• Last Update Posted: January 31, 2013
• Last Verified: January 2013

Keywords provided by Samantha Reiter, University of California, Irvine:

Non relaxing puborectalis syndrome; Botulinum toxin A; Botox; Constipation

Additional relevant MeSH terms:

Syndrome; Disease; Pathologic Processes; Botulinum Toxins; Acetylcholine Release Inhibitors; Membrane Transport Modulators; Molecular Mechanisms of Pharmacological Action; Cholinergic Agents; Neurotransmitter Agents; Physiological Effects of Drugs