Study on Valbenazine for Treating Dyskinesia in Children and Adults with Cerebral Palsy
This trial is investigating the effectiveness and safety of valbenazine in treating involuntary movements in people with cerebral palsy. The study focuses on a specific type of movement problem called dyskinesia, which causes jerky, dance-like movements that can interfere with daily activities.
Who can participate:
- Children and teenagers between 4 and 17 years of age
- Both males and females
- Individuals with a confirmed diagnosis of dyskinesia caused by cerebral palsy, with movements that are jerky or dance-like in nature
- People whose medical condition is stable and not expected to worsen during the study period
Who cannot participate:
- Individuals who do not have dyskinesia related to cerebral palsy
- Those who do not experience the characteristic involuntary, jerky movements
- People outside the specified age range for the study
- Individuals who do not meet the specific clinical trial group requirements
What the trial involves:
The main goal of this study is to evaluate how well valbenazine reduces involuntary movements compared to a placebo. Participants will be randomly assigned to receive either the active medication or a placebo that looks identical but contains no active ingredient. Neither the participants nor the researchers will know who is receiving which treatment during the study, ensuring unbiased results.
The medication is taken orally as a capsule. Throughout the study, participants will be carefully monitored by healthcare professionals who will assess changes in their movement symptoms. The primary measurement involves tracking the Total Maximal Chorea score, which evaluates the severity of involuntary movements, from the beginning of the study through weeks 12 and 14.
The study also includes several additional assessments to get a complete picture of how the treatment affects participants. These include evaluations of overall symptom severity, movement disorder ratings, pain levels, and impressions from both patients and caregivers about whether the condition has improved. Healthcare providers will also assess overall improvement and track progress toward specific treatment goals.
The investigational drug:
Valbenazine works by selectively blocking a specific transporter in the brain called VMAT2, which helps reduce the release of dopamine. This action helps control the involuntary movements characteristic of this condition. The medication has been designed specifically to target movement disorders and is being evaluated for both its effectiveness and safety in this particular population.
The study began recruiting participants in October 2023 and is expected to conclude by January 2026. It is being conducted across five European countries: Spain, Poland, Belgium, Italy, and Portugal.