RecruitingOBSERVATIONAL

Hirschsprung's Advances; Working Towards Autologous tIssue therapIes

This research is called "Hirschsprung's Advances" and focuses on Hirschsprung's Disease, a condition where babies are born without normal nerve cells in the lower part of their bowel. This can cause severe constipation and, if untreated, can be very serious. Current treatment involves surgery to remove the affected part of the bowel, which can lead to long-term issues like needing a stoma (a bag for poo). This study aims to find better treatments. Researchers have discovered special 'nerve stem cells' in the bowel of children with Hirschsprung’s. They want to see if these cells can be grown in the lab to form normal bowel nerves. The hope is to find ways to encourage these stem cells to develop into healthy nerves, possibly avoiding the need for surgery or stoma bags in the future.

At a glance

Status

Recruiting

Sponsor

Alder Hey Children's NHS Foundation Trust

Enrolment target

300

Start

05 Mar 2024

Estimated completion

01 Mar 2036

Hirschsprung Disease

What is this study about?

This study is called "Hirschsprung's Advances" and it's looking into new ways to help children with a condition called Hirschsprung's Disease. This condition means that some babies are born without the normal nerve cells in the end part of their bowel. These nerves are really important because they help the bowel move waste along. Without them, babies can get very constipated and feel very unwell. If it's not treated, it can be very dangerous.

At the moment, the main treatment is an operation where the part of the bowel without nerves is removed. While this can help, it sometimes leads to long-term problems, including some children needing a permanent stoma, which is an opening in the tummy with a bag to collect poo. Doctors and researchers are always looking for better ways to treat Hirschsprung’s Disease that could lead to fewer complications for children.

Researchers involved in this study have made an important discovery: even in the part of the bowel that doesn't have normal nerves in children with Hirschsprung's, there are still special 'nerve stem cells'. These are like basic building blocks that could potentially grow into proper nerve cells. The study aims to understand how these stem cells grow in the lab and if they can be encouraged to form the kind of nerves that make the bowel work properly. They will also look at whether certain medicines can help these stem cells develop into normal nerves. The overall goal is to see if these findings could lead to new treatments where children with Hirschsprung's Disease might not need such extensive surgery or a stoma, giving them a much better quality of life.

Key takeaways

Researches new ways to treat Hirschsprung's Disease.
Uses existing bowel tissue from planned surgeries, no extra procedures.
Focuses on special 'nerve stem cells' to grow new nerves.
Aims to find treatments that could avoid surgery and stoma bags.
No direct risks to your child's health from participation.
Open to children aged 0-15 years already having specific bowel surgery.

Who may be eligible?

This study is open to children who are already having bowel surgery for medical reasons related to their condition. It's important to know that no extra tissue will be taken from your child beyond what is medically necessary for their operation.

Parents or guardians of children aged from newborn up to 15 years old, of any gender, can consider taking part in this study. The main requirement is that your child is already undergoing an operation where a piece of their bowel needs to be removed.

There are no specific reasons that would prevent a child from taking part, as long as they meet the age and surgery criteria. The researchers are not asking for any additional procedures or changes to your child's medical care to be part of this study.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is my child aged between newborn and 15 years old?
Is my child already scheduled for a bowel operation?
Will a small piece of bowel tissue be removed during this operation that isn't essential for other medical checks?
Do I understand that no extra procedures will be performed on my child for this study?

Answer every question to see your result.

What does participation involve?

This is an 'observational study', which means researchers will be looking at existing tissue specimens. Your child won't need any extra tests, procedures, or medications as part of this study. If you choose to participate, it simply means that a small part of the bowel tissue that is already being removed during your child's planned surgery (and wouldn't be needed for other medical checks) will be used for this research. There are no additional hospital visits or follow-up appointments required for the study itself, nor will there be any changes to your child's treatment plan. The total duration of your child's involvement is simply the time it takes for the researchers to collect the tissue after their surgery.

Potential risks and benefits

There are no direct medical risks to your child from taking part in this study because no extra tissue is removed or procedures performed beyond their planned surgery. The potential benefit is that your child's tissue could contribute to very important research that might lead to better treatments for Hirschsprung's Disease in the future, potentially helping many other children avoid serious operations or a stoma. You are completely free to decide whether or not to participate, and changing your mind at any time will not affect your child's medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Alder Hey Children's NHS Foundation Trust
Verified postcode
Liverpool, United Kingdom· Recruiting

Common questions

What is Hirschsprung's Disease?

It's a condition where babies are born without normal nerve cells in part of their bowel, making it hard for them to pass stools.

Will my child have any extra procedures if we join this study?

No, your child will not have any extra tests, procedures, or medications. Researchers will only use tissue already being removed during their planned surgery.

What do the researchers hope to find out?

They hope to learn if special 'nerve stem cells' found in the bowel can be grown to form normal nerves, potentially leading to new treatments that avoid surgery.

Who can take part in this study?

Children aged newborn to 15 years old who are already having bowel surgery where some tissue is removed for medical reasons.

Will my child's medical care be affected if we don't join the study?

No, your child's medical care will not be affected at all, regardless of whether you choose to participate or not.

How to find out more

Rachel Harwood, MBBS

rachel.harwood@alderhey.nhs.uk 01512824998 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.