Ongoing Clinical Trials for Dystonia
This article provides information about an ongoing clinical trial investigating treatments for dystonia, specifically dyskinesia related to cerebral palsy. The trial is testing valbenazine, a medication designed to reduce involuntary movements in both children and adults with this condition. The study is being conducted across multiple European countries.
Clinical trial locations
- Belgium
- Italy
- Poland
- Portugal
- Spain
Study on Valbenazine for Treating Dyskinesia in Children and Adults with Cerebral Palsy
This clinical trial is investigating valbenazine as a treatment for dyskinesia in people with cerebral palsy. Dyskinesia refers to involuntary movements that can be jerky or dance-like, and these movements can significantly affect daily activities and quality of life.
Who can participate:
- Children and teenagers between 4 and 17 years old
- People with a confirmed medical diagnosis of dyskinesia due to cerebral palsy, characterized by choreiform (dance-like) movements
- Patients whose medical conditions are stable and expected to remain stable during the study
- Both males and females are welcome to participate
Who cannot participate:
- People who do not have dyskinesia due to cerebral palsy
- Those who do not experience choreiform movements
- Individuals outside the specified age range for the study
- Those who do not meet the specific clinical trial group criteria
What the trial involves:
The main goal of this study is to test how well valbenazine works in reducing involuntary movements and to evaluate its safety. Valbenazine is taken orally as a capsule and works by affecting dopamine release in the brain, which helps control unwanted movements.
Participants will be randomly assigned to receive either valbenazine or a placebo (an inactive look-alike capsule). Neither the participants nor the researchers will know who is receiving which treatment during the study. This design helps ensure that the results are unbiased and accurate.
The study will measure changes in movement using several assessment tools, including the Total Maximal Chorea score, which tracks involuntary movements at the beginning of the study and then again at weeks 12 and 14. Other assessments will look at overall severity of symptoms, improvement as noted by patients and caregivers, pain levels, and overall motor function.
Study medication:
Valbenazine is a medication classified as a VMAT2 inhibitor. It works by selectively inhibiting a protein called vesicular monoamine transporter 2, which reduces dopamine release in the brain. This mechanism helps control the involuntary movements seen in dyskinesia. The medication is taken as an oral capsule, and the dosage is determined by the study protocol.
The trial began recruiting participants in October 2023 and is expected to conclude in January 2026. The study is being conducted across five European countries: Spain, Poland, Belgium, Italy, and Portugal.
Summary
Currently, there is one clinical trial available for patients with dystonia, specifically focusing on dyskinesia related to cerebral palsy. This trial is testing valbenazine, a medication that targets involuntary movements by affecting dopamine regulation in the brain. The study is notable for its focus on a younger population, including children as young as 4 years old, and is being conducted across multiple European countries. The trial uses a rigorous double-blind, placebo-controlled design to ensure reliable results about the medication’s effectiveness and safety. Patients interested in participating should consult with their healthcare providers to determine if they meet the eligibility criteria and to discuss whether participation would be appropriate for their individual circumstances.
