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

NCT ID: NCT06785844

Last Updated: 2025-01-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

EARLY_PHASE1

Total Enrollment

14 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-02-01

Study Completion Date

2030-09-30

Brief Summary

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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.

Detailed Description

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Conditions

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Fecal Incontinence

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Botulinum toxin

Group Type EXPERIMENTAL

Botulinum Toxin A (Botox )

Intervention Type DRUG

Botulinum Toxin injection

Interventions

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Botulinum Toxin A (Botox )

Botulinum Toxin injection

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

4 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shaare Zedek Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Yogev Dotan

Dr Dotan Yogev

Responsibility Role PRINCIPAL_INVESTIGATOR

Central Contacts

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Dotan Yogev, MD

Role: CONTACT

972 025645027

References

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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.

Reference Type BACKGROUND
PMID: 35262513 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 31989766 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 19036619 (View on PubMed)

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. Epub 2019 Apr 23.

Reference Type BACKGROUND
PMID: 31060739 (View on PubMed)

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(2):147-158. doi: 10.1016/S2468-1253(23)00332-1. Epub 2023 Dec 18.

Reference Type BACKGROUND
PMID: 38128556 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 21240024 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 31690829 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 35114289 (View on PubMed)

Koppen IJ, von Gontard A, Chase J, Cooper CS, Rittig CS, Bauer SB, Homsy Y, Yang SS, Benninga MA. Management of functional nonretentive fecal incontinence in children: Recommendations from the International Children's Continence Society. J Pediatr Urol. 2016 Feb;12(1):56-64. doi: 10.1016/j.jpurol.2015.09.008. Epub 2015 Oct 21.

Reference Type BACKGROUND
PMID: 26654481 (View on PubMed)

Popescu M, Mutalib M. Bowel transit studies in children: evidence base, role and practicalities. Frontline Gastroenterol. 2021 May 10;13(2):152-159. doi: 10.1136/flgastro-2020-101719. eCollection 2022.

Reference Type BACKGROUND
PMID: 35300467 (View on PubMed)

Lambregts AP, Nieuwhof-Leppink AJ, Klijn AJ, Schroeder RPJ. Intravesical botulinum-A toxin in children with refractory non-neurogenic overactive bladder. J Pediatr Urol. 2022 Jun;18(3):351.e1-351.e8. doi: 10.1016/j.jpurol.2022.02.007. Epub 2022 Feb 18.

Reference Type BACKGROUND
PMID: 35283021 (View on PubMed)

Voskuijl WP, Reitsma JB, van Ginkel R, Buller HA, Taminiau JA, Benninga MA. Longitudinal follow-up of children with functional nonretentive fecal incontinence. Clin Gastroenterol Hepatol. 2006 Jan;4(1):67-72. doi: 10.1016/j.cgh.2005.10.001.

Reference Type BACKGROUND
PMID: 16431307 (View on PubMed)

Rajindrajith S, Devanarayana NM, Benninga MA. Review article: faecal incontinence in children: epidemiology, pathophysiology, clinical evaluation and management. Aliment Pharmacol Ther. 2013 Jan;37(1):37-48. doi: 10.1111/apt.12103. Epub 2012 Oct 28.

Reference Type BACKGROUND
PMID: 23106105 (View on PubMed)

Other Identifiers

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Intra-rectal Botulinum toxin

Identifier Type: -

Identifier Source: org_study_id

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