Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3, Open Label, Multicenter, Baseline-controlled Sequential Dose Titration Study Followed by a Fixed Dose Observation Period to Evaluate Pharmacokinetics, Efficacy and Safety of Mirabegron Prolonged-release Microgranula-based Suspension in Children from 6 Months to Less Than 3 Years of Age with Neurogenic Detrusor Overactivity

This research study is for very young children, from 6 months old up to just under 3 years old, who have a bladder condition called neurogenic detrusor overactivity. This condition means their bladder muscles squeeze too often or at the wrong times, leading to leaks. The study is testing a medication called Mirabegron, which is given as a liquid. Researchers want to find the right dose for these children, and then see how well it helps control their bladder, reduce leaks, and if there are any side effects. The aim is to make daily life easier for these children and their families.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Astellas Pharma Global Development Inc.

Enrolment target

Start

07 Dec 2023

Neurogenic detrusor overactivity (NDO)

What is this study about?

This study is about a particular bladder problem called neurogenic detrusor overactivity, which sometimes affects very young children, from 6 months up to just under 3 years old. This condition means the bladder muscle (called the detrusor) contracts too often or without warning, which can lead to wetting accidents and can be quite challenging for children and their families.

The study is looking at a medicine called Mirabegron, which is already used for similar bladder problems in older children and adults. In this study, the medicine is given as a liquid that's easy for young children to take. The main goal is to find out the best dosage for these very young children, and then to see if it helps to improve their bladder control and reduce leakage over time. Researchers also want to be sure that the medicine is safe for this age group.

Understanding how this medicine works in very young children and finding a safe, effective dose is really important. It could potentially help many children with this condition lead more comfortable lives, giving them better control over their bladders and reducing the impact this condition has on their daily routines.

Key takeaways

The study is for children aged 6 months to under 3 years with a specific bladder problem.
It's testing a liquid medicine called Mirabegron to improve bladder control.
Researchers want to find the best dose and check if the medicine is safe and effective.
Participation involves regular clinic visits, special bladder tests, and blood checks.
The study aims to help young children with bladder control issues.

Who may be eligible?

This study is for very young children who have been diagnosed with a specific bladder condition called neurogenic detrusor overactivity.

Children can join the study if they are between 6 months old and just under 3 years old.

Both boys and girls are able to participate.

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 your child between 6 months and under 3 years old?
Has your child been diagnosed with neurogenic detrusor overactivity?
Are you able to attend regular appointments and follow study instructions?
Are you comfortable with your child taking a new liquid medication?

Answer every question to see your result.

What does participation involve?

If a child joins this study, they will receive the study medicine, Mirabegron, as a liquid. Initially, the dose will be adjusted carefully to find the best amount for your child. After this, your child will take a set dose for a longer period.

Throughout the study, specific checks will be done. This includes regular appointments where doctors will check vital signs like heart rate and blood pressure, and perform blood and urine tests. They will also do special bladder tests (called urodynamics) and ultrasound scans of the kidneys and bladder to see how things are working.

You and your child might also be asked to keep a diary of how often your child leaks or needs to be catheterised. The study will involve several visits over about a year.

Potential risks and benefits

Taking part in any study has potential benefits and risks. The potential benefit is that the study medicine, Mirabegron, might help improve your child's bladder control and reduce wetting incidents. This could lead to a better quality of life for your child. However, like all medicines, Mirabegron can have side effects, which will be carefully monitored. There might also be some discomfort from the tests involved, such as blood draws or bladder tests. It's important to remember that you are free to withdraw your child from the study at any time, for any reason, without it affecting their regular medical care.

Locations (4)

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

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Unverified
Denmark
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Unverified
Belgium
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Unverified
Poland
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Unverified
Germany

Common questions

What is neurogenic detrusor overactivity?

It's a bladder condition where the bladder muscles squeeze too often or at the wrong times, leading to problems with bladder control and leakage, especially in children with certain nerve conditions.

What is Mirabegron?

Mirabegron is a medicine that helps the bladder muscle relax, which can calm an overactive bladder and help it hold more urine, reducing leaks.

How will my child take the medicine?

The medicine will be given as a liquid suspension, which is easier for very young children to take.

Will my child need any special tests?

Yes, they will have some special bladder tests (urodynamics), blood tests, urine tests, and ultrasounds to check how the medicine is affecting their body and bladder.

How long does the study last?

The study involves regular check-ups and assessments over a period of about a year.