NCT05450991

Brief Summary

Approximately 340 babies are born with Hirschsprung's disease (HSCR) or an anorectal malformation (ARM) per year in the UK. Most require corrective surgery in the newborn or early infancy period. In both conditions, there is both variability in the severity of the condition and the type of operative technique used. Many children do well following surgery and have good continence in later life. However a significant proportion of children suffer from a lifetime of constipation or incontinence. This has a significant impact on their social and psychological welfare and is a significant burden on healthcare resources. Due to the variation in practice, there are limited data on long-term outcomes following surgery for children with HSCR or ARMs. Where studies have been performed, they often include small numbers, non-standardised outcome measures and short follow-up periods. It is therefore difficult to ascertain the effectiveness of different management strategies. However, a recent Delphi process has been carried out to establish 10 core measures in HSCR to improve outcome reporting. This research group has a long track record in this research area, specifically in the determination of long-term outcomes of children with HSCR, leading to well cited papers within the literature. In the last 2 decades there has been a significant change in the surgical techniques used in HSCR, however the underlying evidence base for this is still lacking. This study seeks to follow-on from previous studies looking specifically at the long-term outcomes in children with HSCR. Furthermore, the aim is to widen the study to encompass all children treated at Alder Hey and also to use the same methodology to assess long-term outcomes for children with ARMs, as both groups of patients experience similar long term morbidity. This will provide qualitative and quantitative data aiding counselling parents of children with HSCR/ARM.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1,200

participants targeted

Target at P75+ for all trials

Timeline
31mo left

Started Jan 2022

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress62%
Jan 2022Dec 2028

Study Start

First participant enrolled

January 25, 2022

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

June 18, 2022

Completed
23 days until next milestone

First Posted

Study publicly available on registry

July 11, 2022

Completed
6.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2028

Expected
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2028

Last Updated

January 30, 2024

Status Verified

January 1, 2024

Enrollment Period

6.5 years

First QC Date

June 18, 2022

Last Update Submit

January 29, 2024

Conditions

Hirschsprung DiseaseAnorectal Malformations

Outcome Measures

Primary Outcomes (20)

  • Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls

    PedsQL score for children. Compared to pre-defined controls. Score 0 (best)-92 (worst)

    1 year of age

  • Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls

    PedsQL score for children. Compared to pre-defined controls. Score 0 (best)-92 (worst)

    5 years of age

  • Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls

    PedsQL score for children. Compared to pre-defined controls. Score 0 (best)-92 (worst)

    10 years of age

  • Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls

    PedsQL score for children. Compared to pre-defined controls Score 0 (best)-92 (worst)

    15 years of age

  • Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls

    GI-QOL score. Compared to pre-defined controls. 0 (worst)-144 (best)

    20 years of age

  • Comparison of the Quality of life of children and adults with HSCR and ARM to pre-defined controls

    GI-QOL score. Compared to pre-defined controls. 0 (worst)-144 (best)

    30 years of age

  • Description of urinary function of children and adults with HSCR and ARM

    Proportion requiring urinary catheterisation or experiencing urinary incontinence

    5 years of age

  • Description of urinary function of children and adults with HSCR and ARM

    Proportion requiring urinary catheterisation or experiencing urinary incontinence

    10 years of age

  • Description of urinary function of children and adults with HSCR and ARM

    Proportion requiring urinary catheterisation or experiencing urinary incontinence

    15 years of age

  • Description of urinary function of children and adults with HSCR and ARM

    Proportion requiring urinary catheterisation or experiencing urinary incontinence

    20 years of age

  • Description of urinary function of children and adults with HSCR and ARM

    Proportion requiring urinary catheterisation or experiencing urinary incontinence

    30 years of age

  • Comparison of the bowel function of children and adults with HSCR and ARM to pre-defined controls

    Paediatric incontinence and constipation score

    5 years 0 (worst)-52 (best)

  • Comparison of the bowel function of children and adults with HSCR and ARM to pre-defined controls

    Paediatric incontinence and constipation score

    10 years 0 (worst)-52 (best)

  • Comparison of the bowel function of children and adults with HSCR and ARM to pre-defined controls

    Paediatric incontinence and constipation score

    15 years 0 (worst)-52 (best)

  • Description of the Mortality rate of children and adults with ARM and HSCR

    Death rate with cause given

    1year of age

  • Description of the Mortality rate of children and adults with ARM and HSCR

    Death rate with cause given

    5 years of age

  • Description of the Mortality rate of children and adults with ARM and HSCR

    Death rate with cause given

    10 years of age

  • Description of the Mortality rate of children and adults with ARM and HSCR

    Death rate with cause given

    15 years of age

  • Description of the Mortality rate of children and adults with ARM and HSCR

    Death rate with cause given

    20 years of age

  • Description of the Mortality rate of children and adults with ARM and HSCR

    Death rate with cause given

    30 years of age

Secondary Outcomes (2)

  • Comparison of the sexual health of adults with HSCR and ARM compared to pre-defined controls and obstetric health

    30 years of age

  • Description of the obstetric health of adults with HSCR and ARM

    30 years

Study Arms (2)

Hirschsprung's Disease

Infants, children and adults cared for in Alder Hey Children's Hospital from the neonatal period onward with histologically confirmed Hirschsprung's disease.

Procedure: Pull through

Anorectal Malformations

Infants, children and adults cared for in Alder Hey Children's Hospital from the neonatal period onward with an anorectal malformation.

Procedure: Pull through

Interventions

Pull throughPROCEDURE

Children diagnosed with Hirschpsrung's Disease or an anorectal malformation, with or without operative intervention will be included

Also known as: PSARP, Anoplasty, Soave Procedure, Swenson's Procedure, Duhammel Procedure, Cut-back
Anorectal MalformationsHirschsprung's Disease

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Children and adults diagnosed with Hirschsprung's disease or Anorectal Malformation since 1991 cared for in a single centre.

You may qualify if:

  • All children treated at either center with histologically confirmed Hirschsprung's Disease since diagnosis from 1991
  • All children diagnosed with anorectal malformation on the basis of position in relation to the external sphincter and size

You may not qualify if:

  • Patients with non-histologically diagnosed Hirschsprung's Disease
  • Patients whose initial primary treatment or majority of follow-up for either condition has been external to either center
  • Patients with funnel anus
  • Adults who are unable to consent for themselves

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Alder Hey Children's Hospital

Liverpool, Merseyside, L12 2AP, United Kingdom

RECRUITING

MeSH Terms

Conditions

Hirschsprung DiseaseAnorectal Malformations

Condition Hierarchy (Ancestors)

Digestive System AbnormalitiesDigestive System DiseasesMegacolonColonic DiseasesIntestinal DiseasesGastrointestinal DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
OTHER
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 18, 2022

First Posted

July 11, 2022

Study Start

January 25, 2022

Primary Completion (Estimated)

August 1, 2028

Study Completion (Estimated)

December 1, 2028

Last Updated

January 30, 2024

Record last verified: 2024-01

Data Sharing

IPD Sharing
Will not share

Locations

GB(1)