Dyskinesia Trials in Disease

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, jerky, or dance-like movements that can affect different parts of the body and interfere with daily activities. The study aims to determine how well Valbenazine works in reducing these involuntary movements and how safe it is for use in both children and adults.

Who can participate:

• Patients with a confirmed diagnosis of dyskinesia due to cerebral palsy, specifically those experiencing choreiform movements (jerky, dance-like involuntary movements)
• Children and teenagers between 4 and 17 years of age
• Patients whose medical condition is stable and not expected to worsen during the study period
• Both males and females are eligible to participate

Who cannot participate:

• Patients who do not have dyskinesia due to cerebral palsy
• Patients who do not experience choreiform movements
• Patients outside the specified age range of 4 to 17 years
• Patients who do not meet the specific clinical trial group requirements

What the trial involves:

Participants will be randomly assigned to receive either Valbenazine or a placebo (an inactive substance that looks like the medication). The medication is taken orally in capsule form. Neither the participants nor the researchers will know who is receiving the actual medication or the placebo during the study, ensuring unbiased results.

The main goal of the trial is to measure changes in involuntary movements using the Total Maximal Chorea score from the beginning of the study to week 12 and week 14. Additional assessments will include evaluations of overall symptom severity, improvement in movement disorders, pain levels, and motor function. The study will also examine how the treatment affects patients' and caregivers' perceptions of improvement and goal achievement.

Investigational drug:

Valbenazine is the medication being tested in this trial. It works by selectively blocking a protein called vesicular monoamine transporter 2 (VMAT2), which helps reduce the release of dopamine in the brain. This mechanism helps control involuntary movements. Valbenazine is administered as an oral capsule and is being evaluated for its effectiveness and safety in reducing choreiform movements associated with cerebral palsy.

The study began recruiting participants in October 2023 and is expected to conclude by January 2026. The trial is being conducted across five European countries: Spain, Poland, Belgium, Italy, and Portugal.