Effects of Botox in Obstructed Defecation Syndrome
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| ClinicalTrials.gov Identifier: NCT02160288 |
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Recruitment Status :
Recruiting
First Posted : June 10, 2014
Last Update Posted : October 18, 2017
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Obstructed Defecation Syndrome (ODS) | Drug: Botulinum Toxin-A Drug: Normal Saline | Phase 2 |
Constipation represents one of the five most common physician diagnoses for gut disorders. Obstructed defecation syndrome (ODS) is an under-treated condition which accounts for 30%- 50% of all patients with constipation and it is more common as people age. ODS is due to the abnormal contraction of the puborectalis muscle (a muscle around the anus that should relax during defecation).
Biofeedback therapy and medical management are the standards of care for ODS. Typically patients are first managed with dietary modifications (fiber supplementation, increased fluids) and medication (laxatives, enemas). If constipation is not improved, they will undergo biofeedback, which lasts from 3-8 sessions on average. Biofeedback acts on the cause of ODS and it has good short-term success, but around 50%-70% of treated patients re-experience constipation after one year. The main drawbacks of biofeedback for ODS are the facts that it is expensive, time-consuming, available in few select-centers and its success depends very much on the provider. Biofeedback is delivered in multiple 1-hour clinic sessions, so many patients don't finish all recommended sessions and their constipation may recur faster.
Botox also acts on the cause of ODS and was shown to improve constipation within 1-3 weeks after the injection. Botox is delivered as a one-time injection in the puborectalis muscle and external anal sphincter (the muscle right around the anus). The injection can be performed in the clinic under local anesthesia, and the patient goes home afterwards. Currently, Botox is used for treatment of patients who fail biofeedback and medical management, to avoid the options of last resort (resection of the colon with stoma). To this day, no adequately designed study has confirmed that Botox is indeed superior to placebo (normal saline) for the treatment of ODS. The results from this study will provide valuable data on the ability of Botox to improve symptoms of constipation and the duration of its effect. This project has the potential to increase the availability of effective treatments for ODS.
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 46 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Triple (Participant, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | Effects of Type A Botulinum Toxin in Obstructed Defecation Syndrome: a Phase II Randomized, Parallel-Group, Triple-Blind, Placebo-Controlled Trial |
| Actual Study Start Date : | February 2016 |
| Estimated Primary Completion Date : | August 2018 |
| Estimated Study Completion Date : | August 2019 |
| Arm | Intervention/treatment |
|---|---|
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Active Comparator: 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.
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Drug: Botulinum Toxin-A
100 Units diluted in a 5 cc syringe at a concentration of 20U/mL
Other Name: Botox |
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Placebo Comparator: Normal saline
Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter.
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Drug: Normal Saline
Dispensed in a 5 cc syringe
Other Name: 0.9% Sodium Chloride |
- Changes in the Altomare ODS score (ODS-S) and Patient Assessment of Constipation- Quality of Life score (PAC-QoL) [ Time Frame: Baseline, 1 month after injection ]The primary outcome measure will be the percent 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.
- Changes in the Altomare ODS score (ODS-S) [ Time Frame: Baseline, 1, 3, 6 and 12 months after injection ]This is a measure of efficacy.
- Changes in the Patient Assessment of Constipation- Quality of Life score (PAC-QoL) [ Time Frame: Baseline, 1, 3, 6 and 12 months after injection ]This is a measure of efficacy.
- Changes in Health-Related Quality of Life [ Time Frame: Baseline, 1, 3, 6 and 12 months after injection ]SF-36 version 1 will be used to measure changes in the health-related quality of life.This is a measure of efficacy.
- Changes in Cleveland Clinic Fecal Incontinence Score (CCFI) [ Time Frame: Baseline, 1, 3, 6 and 12 months after injection ]This is a measure of Botox-specific adverse events.
- Changes in Fecal Incontinence Quality of Life Scale (FIQoL) [ Time Frame: Baseline, 1, 3, 6 and 12 months after injection ]This is a measure of Botox-specific adverse events.
- Relaxation of puborectalis with push measured by EMG [ Time Frame: Baseline, 1 month follow-up visit ]This is a measure of efficacy.
- Success of balloon expulsion test [ Time Frame: Baseline, 1 month follow-up visit ]This is a measure of efficacy.
- Change in anal sphincter function [ Time Frame: Baseline, 1 month follow-up visit ]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.
- Change in defecation index [ Time Frame: Baseline, 1 month follow-up visit ]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.
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| Ages Eligible for Study: | 18 Years to 90 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Males and females 18 years or older of all races and backgrounds
- Competent to give informed consent
- Meet the Rome III diagnostic criteria for functional constipation
- Inability to relax the puborectalis muscle at electromyography
- Altomare Obstructed Defecation Syndrome score of 15 points or above
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Failure of treatment with 2 conservative measures which may be as follows:
- 1 laxative (osmotic or stimulant) for 2 weeks
- 1 fiber supplement for one month
- And/or trial of biofeedback for at least 4 sessions
Exclusion Criteria:
- Previous treatment with Botox (possible antibodies)
- Known hypersensitivity to any of the components of the toxin
- Medication regimen includes narcotics
- Previous radiation therapy to the anal canal and rectum
- Prior proctectomy
- Presence of unhealed and symptomatic anal fissure
- Presence of anal pain
- Presence of fecal incontinence
- Presence of full thickness rectal prolapse
- Presence of internal sphincter myopathy
- Inflammatory bowel disease or proctitis
- Pregnancy or breast-feeding
- Subject is currently enrolled/ just finished participating in a clinical trial in which the intervention/ its carry-over effect may interact with the intervention in this trial
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02160288
| Contact: Liliana Bordeianou, M.D. | 617-643-0541 | lbordeianou@partners.org | |
| Contact: Lieba Savitt, N.P. | 617-643-5580 | MGHpelvicfloorcenter@partners.org |
| United States, Massachusetts | |
| Massachusetts General Hospital | Recruiting |
| Boston, Massachusetts, United States, 02114 | |
| Contact: Liliana Bordeianou, M.D. 617-643-0541 lbordeianou@partners.org | |
| Contact: Lieba Savitt, N.P. 617-643-5580 MGHpelvicfloorcenter@partners.org | |
| Principal Investigator: Liliana Bordeianou, M.D. | |
| Principal Investigator: | Liliana Bordeianou, M.D. | Massachusetts General Hospital |
Publications:
| Responsible Party: | Liliana Bordeianou, Assistant Professor of Surgery, Division of General and Gastrointestinal Surgery, Massachusetts General Hospital |
| ClinicalTrials.gov Identifier: | NCT02160288 History of Changes |
| Other Study ID Numbers: |
MGH2014-P-000406 |
| First Posted: | June 10, 2014 Key Record Dates |
| Last Update Posted: | October 18, 2017 |
| Last Verified: | October 2017 |
Keywords provided by Liliana Bordeianou, Massachusetts General Hospital:
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Botox Obstructed Defecation Syndrome ODS Outlet-Type Constipation injection |
Additional relevant MeSH terms:
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Syndrome Disease Pathologic Processes Botulinum Toxins Botulinum Toxins, Type A abobotulinumtoxinA Acetylcholine Release Inhibitors |
Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action Cholinergic Agents Neurotransmitter Agents Physiological Effects of Drugs Neuromuscular Agents Peripheral Nervous System Agents |