Not yet recruitingPHASE1, PHASE2INTERVENTIONAL

Autologous Muscle Stem Cell Therapy for Treatment of Congenital Urinary Incontinence in Epispadias Patients

This study is looking for boys aged 3 to 17 who have a condition called epispadias, which means their bladder or urethra (the tube that carries urine out of the body) hasn't formed correctly. This often leads to urinary incontinence, where urine leaks out without control. The study will try a new treatment using the boy's own muscle stem cells. These cells will be taken from a muscle sample, grown, and then injected into the bladder muscle to help repair it. The main goals are to see if this treatment is safe and if it can help improve bladder control for these boys. The study will compare this new treatment to a placebo (a dummy treatment) to see how effective it is.

At a glance

Status

Not yet recruiting

Phase

PHASE1, PHASE2

Sponsor

Simone Spuler, MD

Enrolment target

Start

01 Jun 2024

Estimated completion

01 Oct 2026

Urinary Incontinence Epispadias, Male

What is this study about?

This study is about helping boys who have a condition called epispadias. Epispadias means the tube that carries urine out of the body (the urethra) or parts of the bladder haven't developed properly from birth. A common problem for boys with epispadias is that they can't control their urine, meaning they leak urine without meaning to. This happens because a special muscle called the urethral sphincter, which acts like a valve to hold urine in, isn't working as it should.

Researchers are testing a new approach using the boy's own muscle cells. The idea is to take a tiny piece of muscle, grow the special muscle cells from it, and then inject these new cells back into the urethral sphincter. The hope is that these cells will help repair the faulty muscle, making it stronger and better able to hold urine. This could significantly improve their bladder control and quality of life.

The study is in its early stages (Phases 1 and 2), which means the main goals are to check if this new treatment is safe for the boys and to see if it shows signs of working. They will also compare the treatment to a placebo, which is a dummy treatment that looks just like the real one but has no active ingredients. This helps researchers understand if any improvements are genuinely due to the new muscle cell treatment.

Key takeaways

This study is testing a new cell therapy for boys with epispadias and urine leaks.
It uses a boy's own muscle cells to try and fix bladder control problems.
The main goals are to check if the treatment is safe and if it works.
Participants will be carefully monitored for at least a year.
This is an early-stage study (Phases 1 & 2), so results are not guaranteed.

Who may be eligible?

This study is specifically for boys who meet certain requirements. Firstly, they must be male and have only epispadias, without other complex birth defects that might affect their bladder or other body parts. They need to be between 3 and 17 years old, and they must be experiencing urinary incontinence, meaning they consistently leak urine.

There are also reasons why someone might not be able to join the study. If a boy has any ongoing or long-term infections or inflammation in their body, or if they have a condition that affects how their blood clots, they would not be able to participate. Also, if they have had a bad reaction to anaesthesia before, or if they have certain heart conditions like a heart defect from birth or an irregular heartbeat, they would not be eligible. Finally, for a boy to join, his parents or legal guardians must understand and agree to the study by signing an informed consent form.

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 the person male?
Do they have only epispadias (no other complex birth defects)?
Are they between 3 and 17 years old?
Do they have urinary incontinence (urine leaks)?
Do they have any serious ongoing health issues like infections or heart problems?
Have their parents agreed to participate?

Answer every question to see your result.

What does participation involve?

If a boy joins this study, he would first have a small procedure called a cystoscopy. During this, the doctor will take a tiny sample of muscle tissue. This muscle tissue will then be used to grow the special muscle stem cells in a lab. Once enough cells are ready, the boy will have another cystoscopy where these grown cells (or a placebo) are carefully injected into the urethral sphincter muscle. This injection is done with the doctor watching inside the bladder.

After the injection, the boy will be closely monitored for at least 12 months. This monitoring will involve regular check-ups to make sure the treatment is safe and to see if it's helping with his bladder control. The exact number of visits and tests will be explained in detail, but the overall aim is to track his progress and health for at least a year after the treatment.

Potential risks and benefits

Like all medical studies, there are potential benefits and risks. The potential benefit is that the muscle stem cell treatment could help repair the bladder muscle, leading to better bladder control and improved quality of life. However, because this is a new treatment, the full risks aren't yet known. There could be risks associated with the procedures, such as infection or bleeding from the muscle biopsy or injection, or reactions to the cells themselves. Participants are free to withdraw from the study at any time, for any reason, without it affecting their future medical care.

Locations (2)

Clinic for Pediatric Urology in cooperation with University Hospital Regensburg, St. Hedwig Hospital
Verified postcode
Regensburg, Germany
Pediatric Urology, Department for Urology University of Ulm
Verified postcode
Ulm, Germany

Common questions

What is epispadias?

Epispadias is a birth condition where the tube that carries urine out of the body (urethra) hasn't formed correctly, often leading to problems with bladder control.

What are 'muscle stem cells'?

These are special cells found in your muscles that have the ability to repair and regrow muscle tissue. In this study, we use your own cells.

What is a placebo?

A placebo is a 'dummy' treatment that looks just like the real treatment but contains no active medicine. It helps researchers compare the real treatment's effects.

Will my child have to stay in hospital?

The procedures typically involve a short hospital stay for the biopsy and injection, but patients usually go home the same day or shortly after.

How long does the study last?

Participants will be followed for at least 12 months after the injection to monitor their progress and safety.

How to find out more

Simone Spuler, Prof. Dr.

simone.spuler@charite.de +49 (0) 30 450 540 501 Official trial record

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