Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A prospective, randomized, double-blind, placebo-controlled, two-stage, multicenter study with an open-label extension period to investigate the efficacy and safety of NT 201 in the treatment of lower limb spasticity in children and adolescents with cerebral palsy

This research study is looking at a medicine called NT 201, also known as Xeomin. It's for children and teenagers who have cerebral palsy and experience stiffness in their leg muscles, making movement difficult. The study wants to find out if NT 201 can safely reduce this muscle stiffness and help them move more easily. Some participants will receive the active medicine, while others will get a placebo (a dummy treatment) in a way that neither doctors nor participants know who is getting what. This helps ensure fair results. Researchers will then compare how well both groups respond by checking muscle stiffness and overall progress over several weeks.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Merz Pharmaceuticals GmbH

Enrolment target

Start

06 May 2024

Pediatric lower limb (LL) spasticity (SP) caused by cerebral palsy (CP)

What is this study about?

This study is about helping children and teenagers who have cerebral palsy and experience muscle stiffness, especially in their legs. This stiffness, called spasticity, can make it hard to move around and do daily activities. The study is investigating a specific medicine, NT 201 (also known as Xeomin), to see if it can lessen this stiffness.

The main goal is to understand if NT 201 can safely and effectively reduce spasticity in these young people. Researchers will be looking closely at how much the leg muscles become less stiff over a few weeks after treatment. They'll also check if participants are moving better and achieving their personal treatment goals.

This is a Phase 3 study, which means the medicine has already been studied quite a bit and has shown some promise. This larger study is designed to confirm those findings and gather more detailed information about how well it works and if it has any side effects.

Key takeaways

Tests a medicine (NT 201/Xeomin) for leg muscle stiffness in children/teenagers with cerebral palsy.
Aims to reduce stiffness and improve movement.
Compares the medicine to a dummy treatment (placebo).
Neither participants nor doctors know who gets which treatment (double-blind).
Involves regular clinic visits for assessments.
Participants can leave the study at any time.

Who may be eligible?

This study is looking for children and teenagers of any age who have stiffness in their leg muscles (spasticity) because of cerebral palsy.

Both boys and girls can take part. The research team will carefully check each potential participant to make sure the study is a good fit for them and that it's safe for them to join.

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.

Do I/does my child have cerebral palsy?
Do I/does my child experience leg muscle stiffness (spasticity)?
Am I/is my child comfortable with regular clinic visits?
Am I/is my child able to follow study instructions?

Answer every question to see your result.

What does participation involve?

If you or your child decides to take part, the study will involve several visits to a clinic. You or your child would receive either the study medicine (NT 201/Xeomin) or a placebo (a dummy treatment that looks like the real medicine but contains no active drug). Neither you nor your doctor will know which one is being given, to ensure fair results.

Throughout the study, doctors will regularly check the stiffness in the leg muscles. They'll use special scales to measure changes and assess overall progress. There will be initial appointments, followed by check-ups at around 4 and 6 weeks after treatment. After this main part, there might be an option to continue with the open-label extension, where everyone would receive the active medicine. The total duration of the study would be explained in detail by the study team.

Potential risks and benefits

Taking part in any clinical study has potential benefits and potential risks. A possible benefit of this study is that NT 201 (Xeomin) might help reduce the leg muscle stiffness caused by cerebral palsy, potentially improving movement and comfort. However, there's no guarantee it will work for everyone, or work at all. As with any medicine, there could be side effects, which the study team will discuss in detail. You or your child can choose to leave the study at any time, for any reason, without it affecting your usual healthcare.

Locations (2)

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

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Unverified
Latvia
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Unverified
Poland

Common questions

What is cerebral palsy?

Cerebral palsy is a condition that affects movement and muscle tone or posture. It's caused by damage to the developing brain, often before or during birth.

What is spasticity?

Spasticity means that muscles are stiff and tight, making it difficult to move. It's a common symptom for people with cerebral palsy.

What is Xeomin (NT 201)?

Xeomin is a medicine that helps relax muscles by blocking nerve signals that tell them to contract. It's used in some conditions to reduce muscle stiffness.

What is a placebo?

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

What does 'double-blind' mean?

Double-blind means that neither the participants (or their parents) nor the doctors know who is receiving the active medicine and who is receiving the placebo. This helps prevent bias in the study results.