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Recruiting NCT06785844

Intra-rectal Botulinum Toxin Injection for Intractable Non-retentive Fecal Incontinence in Children - an Open Label Pilot Study

Early Phase I Interventional Fecal Incontinence

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Botulinum Toxin A (Botox ).
Who it may be relevant to
Registry conditions: Fecal Incontinence. Basic parameters: 4 years — 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Israel
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Overview

Background: Fecal Incontinence (FI) is a frustrating and prevalent GI condition with profound social implications and a marked effect on quality of life. Treatment options are limited for children whose FI is not secondary to constipation (overflow incontinence), and they are defined as having non-retentive fecal incontinence (NRFI). Rectal botulinum injections (RBI) have recently shown promise for the treatment of FI in adults, following a large, randomized placebo-controlled trial, but no data exists regarding efficacy in children. Objectives: To evaluate the efficacy and safety of RBI in children with non-retentive fecal incontinence. Methods: A prospective open-label pilot study. Children with intractable NRFI will be screened using anorectal manometry and a colonic transit study. Eligible patients will receive one course of RBI and data regarding FI frequency will be prospectively collected during a 15-week period. Significance: New treatment options for children with intractable fecal incontinence are highly in need. The current study aims to introduce a new treatment modality into pediatric research and patient care.

Interventions

  • Drug Botulinum Toxin A (Botox )
    Botulinum Toxin injection

Primary outcome measures

  • median number of FI episodes/week at 1-month post-intervention compared to baseline [Time frame: 3-months post-intervention]
Secondary outcome measures (6)
  • Median number of FI episodes/week at 3-months post-intervention, compared to baseline diary. [Time frame: 3-months post-intervention]
  • Rate of patients with a 50% decrease in mean FI episodes/week compared to baseline diary. [Time frame: 3-months post-intervention]
  • Rate of patients with daily FI episodes (at least 1 episode/day) compared to baseline. [Time frame: 3-months post-intervention]
  • Mean number of complete bowel movements/week compared to baseline according to diary. [Time frame: 3-months post-intervention]
  • Parents and patient's general impression of the treatment using the following questions: "Was the treatment useful/beneficial?"; "Did the treatment improve your quality of life"? [Time frame: 3-months post-intervention]
  • Rate of RBI-associated major and minor adverse events. Patients will be questioned according to list, as well as allowed to add adverse events not listed. [Time frame: 3-months post-intervention]

Eligibility criteria

Inclusion criteria

  • Children 4-18 years old with fecal incontinence for a period greater than 6 months.
  • FI frequency of ≥ 3 episodes/week.
  • After appropriate medical evaluation, FI cannot be explained by another medical condition.
  • Normal colonic transit study, defined as passage of 80% of markers on day
  • Normal RAIR on anorectal manometry

Exclusion criteria

  • Patients currently fulfilling rome IV criteria for functional constipation.
  • Patients with evidence of fecal retention.
  • Patients who had had good response to treatment for overflow incontinence.
  • Absent RAIR on anorectal manometry.
  • Any radiologic evidence of dochylosigmoid or distended colon.
  • Any known organic condition that may affect bowel transit.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Israel · 1 center
  • Shaare Zedek Medical Center — Jerusalem

Publications

  • Rybak A, Martinelli M, Thapar N, Van Wijk MP, Vandenplas Y, Salvatore S, Staiano A, Benninga MA, Borrelli O. Colonic Function Investigations in Children: Review by the ESPGHAN Motility Working Group. J Pediatr Gastroenterol Nutr. 2022 May 1;74(5):681-692. doi: 10.1097/MPG.0000000000003429. Epub 2022 Feb 24. PMID 35262513
  • Athanasakos E, Cleeve S, Thapar N, Lindley K, Perring S, Cronin H, Borrelli O, Mutalib M. Anorectal manometry in children with defecation disorders BSPGHAN Motility Working Group consensus statement. Neurogastroenterol Motil. 2020 Jun;32(6):e13797. doi: 10.1111/nmo.13797. Epub 2020 Jan 27. PMID 31989766
  • Pascual-Pascual SI, Pascual-Castroviejo I. Safety of botulinum toxin type A in children younger than 2 years. Eur J Paediatr Neurol. 2009 Nov;13(6):511-5. doi: 10.1016/j.ejpn.2008.10.006. Epub 2008 Nov 25. PMID 19036619
  • Halleran DR, Lu PL, Ahmad H, Paradiso MM, Lehmkuhl H, Akers A, Hallagan A, Bali N, Vaz K, Yacob D, Di Lorenzo C, Levitt MA, Wood RJ. Anal sphincter botulinum toxin injection in children with functional anorectal and colonic disorders: A large institutional study and review of the literature focusing on complications. J Pediatr Surg. 2019 Nov;54(11):2305-2310. doi: 10.1016/j.jpedsurg.2019.03.020. E PMID 31060739
  • Leroi AM, Queralto M, Zerbib F, Siproudhis L, Vitton V, Amarenco G, Etienney I, Mion F, Bridoux V, Philip J, Brochard C, Damon H, Lacroix E, Gillibert A, Gourcerol G. Intrarectal injections of botulinum toxin versus placebo for the treatment of urge faecal incontinence in adults (FI-Toxin): a double-blind, multicentre, randomised, controlled phase 3 study. Lancet Gastroenterol Hepatol. 2024 Feb;9( PMID 38128556
  • Kaul A, Garza JM, Connor FL, Cocjin JT, Flores AF, Hyman PE, Di Lorenzo C. Colonic hyperactivity results in frequent fecal soiling in a subset of children after surgery for Hirschsprung disease. J Pediatr Gastroenterol Nutr. 2011 Apr;52(4):433-6. doi: 10.1097/MPG.0b013e3181efe551. PMID 21240024
  • Vriesman MH, Koppen IJN, Camilleri M, Di Lorenzo C, Benninga MA. Management of functional constipation in children and adults. Nat Rev Gastroenterol Hepatol. 2020 Jan;17(1):21-39. doi: 10.1038/s41575-019-0222-y. Epub 2019 Nov 5. PMID 31690829
  • de Bruijn CMA, Safder S, Rolle U, Mosiello G, Marshall D, Christiansen AB, Benninga MA. Development of a Bowel Management Scoring Tool in Pediatric Patients with Constipation. J Pediatr. 2022 May;244:107-114.e1. doi: 10.1016/j.jpeds.2022.01.036. Epub 2022 Feb 1. PMID 35114289

Identifiers

NCT: NCT06785844 · Intra-rectal Botulinum toxin

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗