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Comparison of intrathecal baclofen pump and selective dorsal rhizotomy for children with cerebral palsy who cannot walk

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study is looking at cerebral palsy, a condition that affects movement and muscle control. The study focuses on children with cerebral palsy who cannot walk and who have muscle stiffness that is not well controlled with other treatments. Two different treatment approaches will be compared in this study. The first treatment involves using an intrathecal baclofen pump, which is a device that delivers a medication called baclofen directly into the fluid around the spinal cord to help reduce muscle stiffness. The second treatment is called selective dorsal rhizotomy, which is a surgical procedure on the nerves in the spine to reduce muscle stiffness. The study will use different forms of baclofen medication given through the pump system.

The purpose of this study is to compare how these two treatments affect quality of life, safety, and costs one year after the treatment. The study will look at which treatment works better for children with cerebral palsy who cannot walk and have problems with muscle stiffness. Both treatments aim to help reduce the muscle stiffness that makes movement difficult and affects daily activities.

During the study, children will receive either the baclofen pump or the surgical procedure. The study will follow participants for one year after treatment to measure various outcomes. These include changes in quality of life, any side effects or problems that occur, muscle stiffness levels, movement ability, communication, sleep, and other health factors. The study will also track how much medication is being used and whether the treatments help achieve the goals set for each child. Information about costs related to healthcare will also be collected to understand which treatment provides better value.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial enrollment and consent
Before any procedures begin, written informed consent will be obtained from your legally authorized representative. If you are able to understand based on your age and development, your agreement will also be obtained in a way that is appropriate for you.
You must be between 4 and 18 years old and have been diagnosed with spastic cerebral palsy. Cerebral palsy is a condition that affects movement and posture, and spastic means your muscles are tight or stiff.
You must have significant difficulty with movement, classified as level 4 or 5 on a scale that measures how well you can move around. This means you cannot walk on your own.
Your muscle stiffness must not be well controlled with standard treatments like physical therapy or oral medications, or these treatments cause side effects that are difficult to tolerate.
Step 2
Treatment assignment
You will be assigned to receive one of two treatments for your muscle stiffness. The first option is an intrathecal baclofen pump, which is a device placed under your skin that delivers medication directly into the fluid around your spinal cord. The second option is selective dorsal rhizotomy, which is a surgical procedure on nerves in your spine.
The medication used in the pump is called baclofen, and it helps relax tight muscles. The pump delivers this medication continuously into the space around your spinal cord, called the intrathecal space.
The available formulations of baclofen for the pump include solutions with different concentrations: 10 mg in 5 ml, 10 mg in 20 ml, or 2 mg per ml.
Step 3
Treatment period
If you receive the intrathecal baclofen pump, the device will be surgically implanted and will deliver medication continuously. The dosage and rate of medication delivery will be adjusted by your medical team to find the right amount for you.
You will be allowed to continue taking your regular medications during the study. Your medical team will keep track of all medications you take to see if any can be reduced over time.
During the first year after your treatment, you will not be allowed to switch to the other treatment option or have the other surgical procedure. This is important to understand how well each treatment works on its own.
Step 4
Follow-up assessments
Your health-related quality of life will be measured before your treatment and then again one year after your treatment. This will be done using a questionnaire completed by your caregiver.
Throughout the study, various aspects of your health will be monitored, including: muscle stiffness, range of motion in your joints, trunk control, ability to communicate, mouth and throat function, sleep quality, urinary symptoms, spine and hip alignment, and any medications you are taking.
Any side effects or complications you experience will be carefully recorded and monitored.
Your caregiver's stress levels and satisfaction with the treatment will also be assessed.
The costs related to your healthcare will be tracked to understand the financial impact of each treatment.
Step 5
Long-term follow-up
The study will continue to collect information about your progress for several years after your treatment.
The overall study is expected to continue until April 2039, though your active participation will primarily focus on the first year after treatment.

Who can join the trial?

8 criteria

You or your legal representative must sign a consent form before any study procedures begin. If you are a child, you will be asked to agree in a way that matches your age and understanding.
You can be male or female.
You must be at least 4 years old and younger than 18 years old.
You must have spastic Cerebral Palsy, which is a condition that causes tight and stiff muscles.
You must be classified as GMFCS level 4 or 5, which means you have significant difficulty with movement and require support for mobility.
Your spasticity, which is the muscle tightness and stiffness, must not be well controlled with regular treatments such as therapy or a medication called baclofen taken by mouth, or these treatments cause side effects that are hard to tolerate.
You are allowed to continue taking your regular medications during the study, and the study team will keep track of these medications.
You cannot have certain surgical procedures during the first year of the study, specifically switching from one type of treatment to another type of treatment being studied.

Who cannot join the trial?

4 criteria

The study does not list specific reasons why a patient cannot participate
However, this study is designed only for children who cannot walk on their own (called non-ambulatory) and who have a specific type of Cerebral Palsy that causes muscle stiffness (called spastic Cerebral Palsy)
Children who can walk independently would not be suitable for this study
Children who have other types of Cerebral Palsy without muscle stiffness would not be suitable for this study

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Cliniques Universitaires Saint-Luc
Woluwe-Saint-Lambert, Belgium
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Katholieke Universiteit te Leuven
Leuven, Belgium
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Universitair Ziekenhuis Gent
Gent, Belgium

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Investigated drugs

Intrathecal baclofen
is a muscle relaxant medication that is delivered directly into the fluid around the spinal cord through a pump that is surgically placed under the skin. This medication helps reduce muscle stiffness and spasms in children with cerebral palsy who have difficulty walking. The pump continuously releases small amounts of the medication to help control tight muscles and improve comfort and movement.
Selective dorsal rhizotomy
is a surgical procedure, not a medication, where doctors carefully cut specific nerve roots in the spinal cord that are causing muscle stiffness. This surgery is performed to permanently reduce tightness in the muscles of children with cerebral palsy who cannot walk on their own. The goal is to help improve their comfort, positioning, and ability to participate in daily activities.

Baclofen

What is already known about the treatment

Intrathecal Baclofen – This medication is delivered directly into the fluid surrounding the spinal cord through a surgically implanted pump device that releases the medicine continuously. Baclofen is an established treatment used to reduce severe muscle stiffness and spasms in conditions like cerebral palsy, particularly in children who cannot walk. It works by attaching to specific receptors in the spinal cord and brain that help calm overactive nerve signals responsible for muscle tightness. This medication belongs to a class of drugs called muscle relaxants or antispastic agents, and when given through a pump, it allows for much smaller doses compared to oral tablets while providing better control of symptoms.

Investigated diseases

Cerebral Palsy – Cerebral palsy is a group of disorders that affect a person's ability to move and maintain balance and posture. It is caused by damage to the developing brain, most often before birth, during delivery, or shortly after birth. The condition affects muscle tone, movement, and motor skills, leading to difficulties with coordination and control of voluntary movements. In spastic cerebral palsy, muscles become stiff and tight, making movement difficult and sometimes painful. The severity can vary greatly, with some individuals experiencing mild symptoms while others may have significant physical limitations affecting daily activities. Non-ambulatory forms mean that affected children are unable to walk independently and may require wheelchairs or other mobility aids.