Effects of Botox in Obstructed Defecation Syndrome

NCT02160288 | Terminated Phase 2 Results

• 1 other identifier: MGH2014-P-000406
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine if Botulinum Toxin-A (Botox) injection will improve symptoms of constipation in obstructed defecation syndrome (ODS).

Conditions

Obstructed Defecation Syndrome (ODS)

Keywords

Botox; Obstructed Defecation Syndrome; ODS; Outlet-Type Constipation; injection

Outcome Measures

Primary Outcomes (1)

• Changes in the Altomare Obstructed Defecation Syndrome- Score (ODS-S) and Patient Assessment of Constipation- Quality of Life Score (PAC-QoL)

  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

  Baseline, 1 month after injection

Secondary Outcomes (9)

• Changes in the Altomare ODS Score (ODS-S)

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

• Changes in the Patient Assessment of Constipation- Quality of Life Score (PAC-QoL)

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

• Changes in Health-Related Quality of Life

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

• Changes in Cleveland Clinic Fecal Incontinence Score (CCFI)

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

• Changes in Fecal Incontinence Quality of Life Scale (FIQoL)

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

Study Arms (2)

Botulinum Toxin-A

ACTIVE COMPARATOR

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.

Drug: Botulinum Toxin-A

Normal saline

PLACEBO COMPARATOR

Patients will receive normal saline (placebo) injected in the puborectalis muscle and external anal sphincter.

Drug: Normal Saline

Interventions

Botulinum Toxin-A DRUG

100 Units diluted in a 5 cc syringe at a concentration of 20U/mL

Also known as: Botox

Botulinum Toxin-A

Normal Saline DRUG

Dispensed in a 5 cc syringe

Also known as: 0.9% Sodium Chloride

Normal saline

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• Failure of treatment with 2 conservative measures which may be as follows:
• laxative (osmotic or stimulant) for 2 weeks
• fiber supplement for one month
• And/or trial of biofeedback for at least 4 sessions

You may not qualify if:

• 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

Sponsors & Collaborators

• Massachusetts General Hospital: lead

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Related Publications (4)

• Shaheen NJ, Hansen RA, Morgan DR, Gangarosa LM, Ringel Y, Thiny MT, Russo MW, Sandler RS. The burden of gastrointestinal and liver diseases, 2006. Am J Gastroenterol. 2006 Sep;101(9):2128-38. doi: 10.1111/j.1572-0241.2006.00723.x. Epub 2006 Jul 18.

  PMID: 16848807 BACKGROUND

• Suares NC, Ford AC. Prevalence of, and risk factors for, chronic idiopathic constipation in the community: systematic review and meta-analysis. Am J Gastroenterol. 2011 Sep;106(9):1582-91; quiz 1581, 1592. doi: 10.1038/ajg.2011.164. Epub 2011 May 24.

  PMID: 21606976 BACKGROUND

• American Gastroenterological Association; Bharucha AE, Dorn SD, Lembo A, Pressman A. American Gastroenterological Association medical position statement on constipation. Gastroenterology. 2013 Jan;144(1):211-7. doi: 10.1053/j.gastro.2012.10.029. No abstract available.

  PMID: 23261064 BACKGROUND

• 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. doi: 10.1111/j.1572-0241.2006.00791.x. Epub 2006 Oct 4.

  PMID: 17029615 BACKGROUND

MeSH Terms

Interventions

Botulinum Toxins, Type A; Saline Solution; Sodium Chloride

Intervention Hierarchy (Ancestors)

Botulinum Toxins; Metalloendopeptidases; Endopeptidases; Peptide Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Metalloproteases; Bacterial Proteins; Proteins; Amino Acids, Peptides, and Proteins; Bacterial Toxins; Toxins, Biological; Biological Factors; Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Results Point of Contact

• Title: Dr Liliana Bordeianou
• Organization: Massachusetts General Hospital

lsavitt@mgh.harvard.edu

6176430541

Study Officials

• Liliana Bordeianou, M.D.

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor of Surgery, Division of General and Gastrointestinal Surgery

Study Record Dates

• First Submitted: June 3, 2014
• First Posted: June 10, 2014
• Study Start: February 1, 2016
• Primary Completion: June 11, 2019
• Study Completion: June 11, 2019
• Last Updated: May 12, 2020
• Results First Posted: May 12, 2020

Record last verified: 2020-05

Locations

US (1)