What is the mechanism of action of this treatment?

VMAT2 inhibitors, such as Valbenazine, work by regulating the release of dopamine in the brain. Dyskinesia often results from excessive or dysregulated dopamine activity, and by inhibiting VMAT2, these medications reduce the amount of dopamine released into the synaptic cleft. This helps stabilize dopamine levels and reduce involuntary movements. This mechanism is crucial for dyskinesia patients as it directly addresses the neurochemical imbalance contributing to their symptoms, offering a targeted approach to manage and potentially alleviate their condition.

What side effects are associated with this type of intervention?

Valbenazine, a VMAT2 inhibitor, is primarily used to treat tardive dyskinesia and has side effects that include somnolence, fatigue, and dry mouth. Other common treatments for dyskinesia, such as antipsychotics and dopaminergic agents, can cause side effects like weight gain, gastrointestinal disturbances, and extrapyramidal symptoms. It is important to monitor these side effects closely and discuss them with a healthcare provider.

What prior FDA approvals exist?

Valbenazine, a VMAT2 inhibitor, has been approved by the FDA for the treatment of tardive dyskinesia. Another VMAT2 inhibitor, deutetrabenazine, has also received FDA approval for the same condition. These drugs work by modulating the release of neurotransmitters, thereby reducing involuntary movements associated with dyskinesia. Both medications represent significant advancements in the management of dyskinesia, offering targeted treatment options for patients suffering from these movement disorders.

What other types of research exist?

Promising novel alternatives to Valbenazine for dyskinesia include Deutetrabenazine, which has shown safety and efficacy in treating chorea associated with Huntington's disease, and Tetrabenazine, which has been used for tardive tremor. Additionally, therapies targeting DNA and RNA, such as antisense oligonucleotides, are being explored for conditions like Huntington's disease and may offer future potential for dyskinesia treatment.

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Vesicular Monoamine Transporter 2 (VMAT2) Inhibitor

Valbenazine for Cerebral Palsy

Phase 3

Recruiting

Research Sponsored by Neurocrine Biosciences

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Medically confirmed diagnosis of DCP (hyperkinetic movement disorder due to cerebral palsy [CP]) with choreiform movements

Must not have

Clinical diagnosis or history of dyskinesia due to condition other than CP

Inability to swallow soft solids, unless medications can be administered via a gastrostomy tube

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial is testing valbenazine, a medication that may help reduce involuntary movements. It targets children and adults with cerebral palsy who have these movements. The drug works by balancing chemicals in the brain to control movement better. Valbenazine has a longer duration of action and improved side effect profile compared to other similar medications.

Who is the study for?

This trial is for people aged 6-70 with dyskinesia due to cerebral palsy, specifically those with choreiform movements. Participants should have stable medical conditions and be able to swallow soft solids or take medication via a gastrostomy tube. It's not for pregnant/breastfeeding individuals, those with other causes of dyskinesia, recent suicidal behavior/ideation, substance abuse, or certain heart conditions.

What is being tested?

The study is testing the effectiveness of Valbenazine compared to a placebo in improving involuntary movement (chorea) in participants with dyskinesia from cerebral palsy. The goal is to see if Valbenazine can help control these movements better than no treatment.

What are the potential side effects?

Valbenazine may cause side effects such as sleepiness, balance problems (risk of falls), nausea, and potentially long QT syndrome which affects heart rhythm. Not everyone will experience these side effects and they could vary in severity.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with a movement disorder due to cerebral palsy.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have experienced involuntary movements not caused by cerebral palsy.

Select...

I cannot swallow soft solids but can take medications through a tube.

Select...

I have a history of heart rhythm problems or abnormal ECG results.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: ValbenazineExperimental Treatment1 Intervention

Valbenazine dosed for 14 weeks (double-blind treatment period) followed by open-label valbenazine treatment. Participants may opt to enter an open-label valbenazine treatment extension phase.

Group II: PlaceboPlacebo Group2 Interventions

Placebo dosed for 14 weeks (double-blind treatment period) followed by open-label valbenazine treatment. Participants may opt to enter an open-label valbenazine treatment extension phase.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Valbenazine

2018

Completed Phase 4

~880

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

VMAT2 inhibitors, such as Valbenazine, work by regulating the release of dopamine in the brain. Dyskinesia often results from excessive or dysregulated dopamine activity, and by inhibiting VMAT2, these medications reduce the amount of dopamine released into the synaptic cleft. This helps stabilize dopamine levels and reduce involuntary movements. This mechanism is crucial for dyskinesia patients as it directly addresses the neurochemical imbalance contributing to their symptoms, offering a targeted approach to manage and potentially alleviate their condition.

Postthalamic stroke dystonic choreoathetosis responsive to tetrabenazine.