Ignite Creation Date:
2024-10-26 @ 3:40 PM
Last Modification Date:
2024-10-26 @ 3:40 PM
Study NCT ID:
NCT06592534
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-09-10
Brief Title:
Babies With Enterocolitis - A Study of Faecal Calprotectin in Hirschsprung Disease The BEACH Study
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Babies With Enterocolitis - A Study of Faecal Calprotectin in Hirschsprung Disease The BEACH Study
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
BEACH
Brief Summary:
Babies with Hirschsprungs Disease are born without normal nerves to the end of their bowel which means they cannot poo properly and are at high risk of infection of the bowel called enterocolitis 1 in 4 children with Hirschsprungs Disease develop enterocolitis and if not treated quickly it can lead to death The symptoms include a swollen tummy temperatures and diarrhoea but it can be hard to spot especially in the early stages and there is no test for it
In some diseases a substance called calprotectin is found in the poo when the bowel is inflamed The investigators plan to collect poo samples from children with Hirschsprungs Disease and measure the calprotectin to see if it can help the investigators predict which children are at highest risk of enterocolitis When the investigators collect the poo we will ask parents some questions about their childs diet and poos over the week before and how easy it was to collect the sample
This is a pilot study which means the investigators dont expect to get a definite answer to whether measuring calprotectin levels will change treatment for children with Hirschsprungs disease However it will help the investigators find out if calprotectin levels are a useful test for bowel inflammation in these children and will tell us what parents views are on collecting poo samples regularly If it does look like measuring calprotectin is a useful test in Hirschsprungs disease the investigators will do more studies to find out if some children may benefit from more intensive treatment
In some diseases a substance called calprotectin is found in the poo when the bowel is inflamed The investigators plan to collect poo samples from children with Hirschsprungs Disease and measure the calprotectin to see if it can help the investigators predict which children are at highest risk of enterocolitis When the investigators collect the poo we will ask parents some questions about their childs diet and poos over the week before and how easy it was to collect the sample
This is a pilot study which means the investigators dont expect to get a definite answer to whether measuring calprotectin levels will change treatment for children with Hirschsprungs disease However it will help the investigators find out if calprotectin levels are a useful test for bowel inflammation in these children and will tell us what parents views are on collecting poo samples regularly If it does look like measuring calprotectin is a useful test in Hirschsprungs disease the investigators will do more studies to find out if some children may benefit from more intensive treatment
Detailed Description:
Background
Hirschsprungs Disease is a rare congenital condition affecting 1 in 5000 children and characterised by an absence of ganglia in the distal bowel1 Affected infants cannot stool normally and require an intensive treatment programme of regular rectal washouts or a stoma to ensure that their bowel is emptied and avoid bowel perforation Once they are a few months old definitive surgery is performed to remove the affected segment of bowel
Infants with Hirschsprungs Disease have a high 1 in 4 risk of developing Hirschsprungs associated enterocolitis HAEC an inflammatory and infective condition of the bowel which can cause severe acute illness and death in 1-10 of affected children2 Although the incidence of HAEC is reduced by surgery the risk persists throughout childhood3 Signs and symptoms are non-specific including fevers irritability poor feeding abdominal distension and explosive stool and deterioration can be rapid The mainstay of treatment is intravenous antibiotic therapy with colonic lavage per rectum In severe cases emergency surgery to remove the affected bowel is required with long term implications for continence A biomarker to identify children at higher risk of HAEC or its life-threatening complications would enable clinicians to consider employing additional or earlier treatments for these patients
Faecal calprotectin is a biomarker currently used in both the diagnostic work-up of inflammatory bowel disease IBD and to monitor disease progression and remission adding to the clinical decision making around escalation and de-escalation of IBD treatments4 5 Previous studies have demonstrated that faecal calprotectin levels reduce with age and have wide variability in early infancy6
Faecal calprotectin has not previously been described as a marker of enterocolitis in infants with Hirschsprungs Disease We have received written communication of an unpublished retrospective study of single faecal calprotectin measurement in children with Hirschsprungs Disease and suspected HAEC from a UK centre Using clinical radiological haematological and biochemical features this study divided 27 children into low risk and high risk groups Faecal calprotectin level was significantly higher in the high risk group 285μgg compared to the low risk group 70μgg p amplt001 Whilst these data suggest a potential role for faecal calprotectin assay in the diagnosis of acute HAEC the difference might simply represent association due to demographics baseline faecal calprotectin is known to be higher in younger children likewise HAEC is more common in younger children with Hirschsprungs Disease Further it is unknown whether the high risk group had a more profound acute calprotectin rise reflective of acute disease severity or a higher baseline Serial faecal calprotectin monitoring could benefit children both for earlier diagnosis and earlier initiation of more intensive therapies
Aim
This study aims to describe the longitudinal changes observed in faecal calprotectin levels in infants and children with Hirschsprungs Disease This pilot study will also be used to determine the acceptability of regular faecal calprotectin monitoring for parents using published and accepted methods7 and will be used to power future studies
Design
This is a pragmatic research pilot study Children under 5 years of age with biopsy proven Hirschsprungs Disease will be identified by the clinical team and invited to participate Stool samples will be requested to be provided at every routine clinic appointment and elective admission and during any emergency admissions over the two year study period The faecal calprotectin level will be analysed using standard laboratory protocols for each sample At the time of each stool sample parents will be given a short questionnaire regarding the acceptability of collecting a stool sample and regarding their childs diet bowel habits and recent illnesses
The study will run for 2 years in 4 UK centres with an expectation that each patient provides a minimum of 4 samples to the study during this time A control population has not been included as previous studies have shown that there is a very wide variability in faecal calprotectin in normal infants
Descriptive analyses of the findings will be undertaken including analysis of the change in faecal calprotectin level over time including describing any changes associated with changes in diet association with the number of episodes of HAEC and the faecal calprotectin level at the time of admission with HAEC compared to the baseline level
Key goals
To describe the change in faecal calprotectin in early childhood of children with Hirschsprungs Disease An understanding of how an individuals faecal calprotectin levels change during infancy has not previously been described particularly in children with Hirschsprungs Disease This study will enable the investigators to track the faecal calprotectin levels over two years and to collect preliminary data to assess whether changes in diet and stooling patterns are associated with changes in faecal calprotectin
To gain preliminary data to assess whether there may be an associated between faecal calprotectin and frequency or severity of HAEC episodes in young children with enterocolitis This pilot study will enable the investigators to undertake an initial assessment of whether there is any association between faecal calprotectin levels and HAEC and whether there is any association between faecal calprotectin level and severity of HAEC during an emergency admission
To assess the acceptability to parents of taking stool samples regularly It is important to understand whether this is an acceptable sample to be collected by parents Therefore evaluating the acceptability of collecting a stool sample within the study will help the investigators to determine whether monitoring of faecal calprotectin levels would be possible within clinical practice Children with Hirschsprungs Disease frequently stool with additional help either through using rectal therapy or into a stoma bag and therefore extrapolating the acceptability from people who do not have stooling difficulties is not appropriate
Outcomes
The results of this study will be presented in an international conference and will be published in a peer reviewed journal The findings of this study are intended to determine whether faecal calprotectin has the potential to be used as a predictive biomarker for enterocolitis If it is we plan to undertake a definitive study to demonstrate this using this study to power the sample size required If faecal calprotectin proves a useful biomarker for determining the risk or severity of HAEC this will have important implications for patients enabling them to be treated more intensively and effectively Faecal calprotectin levels could also be used within the design of studies considering the efficacy of novel treatments of HAEC as a recruitment criteria or as a method of stratifying HAEC severity
Hirschsprungs Disease is a rare congenital condition affecting 1 in 5000 children and characterised by an absence of ganglia in the distal bowel1 Affected infants cannot stool normally and require an intensive treatment programme of regular rectal washouts or a stoma to ensure that their bowel is emptied and avoid bowel perforation Once they are a few months old definitive surgery is performed to remove the affected segment of bowel
Infants with Hirschsprungs Disease have a high 1 in 4 risk of developing Hirschsprungs associated enterocolitis HAEC an inflammatory and infective condition of the bowel which can cause severe acute illness and death in 1-10 of affected children2 Although the incidence of HAEC is reduced by surgery the risk persists throughout childhood3 Signs and symptoms are non-specific including fevers irritability poor feeding abdominal distension and explosive stool and deterioration can be rapid The mainstay of treatment is intravenous antibiotic therapy with colonic lavage per rectum In severe cases emergency surgery to remove the affected bowel is required with long term implications for continence A biomarker to identify children at higher risk of HAEC or its life-threatening complications would enable clinicians to consider employing additional or earlier treatments for these patients
Faecal calprotectin is a biomarker currently used in both the diagnostic work-up of inflammatory bowel disease IBD and to monitor disease progression and remission adding to the clinical decision making around escalation and de-escalation of IBD treatments4 5 Previous studies have demonstrated that faecal calprotectin levels reduce with age and have wide variability in early infancy6
Faecal calprotectin has not previously been described as a marker of enterocolitis in infants with Hirschsprungs Disease We have received written communication of an unpublished retrospective study of single faecal calprotectin measurement in children with Hirschsprungs Disease and suspected HAEC from a UK centre Using clinical radiological haematological and biochemical features this study divided 27 children into low risk and high risk groups Faecal calprotectin level was significantly higher in the high risk group 285μgg compared to the low risk group 70μgg p amplt001 Whilst these data suggest a potential role for faecal calprotectin assay in the diagnosis of acute HAEC the difference might simply represent association due to demographics baseline faecal calprotectin is known to be higher in younger children likewise HAEC is more common in younger children with Hirschsprungs Disease Further it is unknown whether the high risk group had a more profound acute calprotectin rise reflective of acute disease severity or a higher baseline Serial faecal calprotectin monitoring could benefit children both for earlier diagnosis and earlier initiation of more intensive therapies
Aim
This study aims to describe the longitudinal changes observed in faecal calprotectin levels in infants and children with Hirschsprungs Disease This pilot study will also be used to determine the acceptability of regular faecal calprotectin monitoring for parents using published and accepted methods7 and will be used to power future studies
Design
This is a pragmatic research pilot study Children under 5 years of age with biopsy proven Hirschsprungs Disease will be identified by the clinical team and invited to participate Stool samples will be requested to be provided at every routine clinic appointment and elective admission and during any emergency admissions over the two year study period The faecal calprotectin level will be analysed using standard laboratory protocols for each sample At the time of each stool sample parents will be given a short questionnaire regarding the acceptability of collecting a stool sample and regarding their childs diet bowel habits and recent illnesses
The study will run for 2 years in 4 UK centres with an expectation that each patient provides a minimum of 4 samples to the study during this time A control population has not been included as previous studies have shown that there is a very wide variability in faecal calprotectin in normal infants
Descriptive analyses of the findings will be undertaken including analysis of the change in faecal calprotectin level over time including describing any changes associated with changes in diet association with the number of episodes of HAEC and the faecal calprotectin level at the time of admission with HAEC compared to the baseline level
Key goals
To describe the change in faecal calprotectin in early childhood of children with Hirschsprungs Disease An understanding of how an individuals faecal calprotectin levels change during infancy has not previously been described particularly in children with Hirschsprungs Disease This study will enable the investigators to track the faecal calprotectin levels over two years and to collect preliminary data to assess whether changes in diet and stooling patterns are associated with changes in faecal calprotectin
To gain preliminary data to assess whether there may be an associated between faecal calprotectin and frequency or severity of HAEC episodes in young children with enterocolitis This pilot study will enable the investigators to undertake an initial assessment of whether there is any association between faecal calprotectin levels and HAEC and whether there is any association between faecal calprotectin level and severity of HAEC during an emergency admission
To assess the acceptability to parents of taking stool samples regularly It is important to understand whether this is an acceptable sample to be collected by parents Therefore evaluating the acceptability of collecting a stool sample within the study will help the investigators to determine whether monitoring of faecal calprotectin levels would be possible within clinical practice Children with Hirschsprungs Disease frequently stool with additional help either through using rectal therapy or into a stoma bag and therefore extrapolating the acceptability from people who do not have stooling difficulties is not appropriate
Outcomes
The results of this study will be presented in an international conference and will be published in a peer reviewed journal The findings of this study are intended to determine whether faecal calprotectin has the potential to be used as a predictive biomarker for enterocolitis If it is we plan to undertake a definitive study to demonstrate this using this study to power the sample size required If faecal calprotectin proves a useful biomarker for determining the risk or severity of HAEC this will have important implications for patients enabling them to be treated more intensively and effectively Faecal calprotectin levels could also be used within the design of studies considering the efficacy of novel treatments of HAEC as a recruitment criteria or as a method of stratifying HAEC severity
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None