What side effects are associated with this type of intervention?

The most common treatments for Huntington's Disease, particularly VMAT2 inhibitors like deutetrabenazine and tetrabenazine, are associated with side effects such as depression, anxiety, and akathisia. Deutetrabenazine is generally well tolerated, with adverse event rates similar to placebo in clinical trials.

What prior FDA approvals exist?

Deutetrabenazine and tetrabenazine are two VMAT2 inhibitors that have been approved by the FDA for the treatment of chorea associated with Huntington's Disease. Deutetrabenazine was found to be efficacious and generally well tolerated in clinical trials. Tetrabenazine, similarly, has been used to manage chorea symptoms in Huntington's Disease patients. Both drugs work by inhibiting the vesicular monoamine transporter 2 (VMAT2), which helps reduce the excessive movements characteristic of chorea.

What other types of research exist?

Promising novel alternatives to Valbenazine for Huntington's Disease patients include gene therapy approaches like AMT-130, RNA interference therapies such as tominersen, and other pharmacologic treatments like pridopidine. These therapies target the underlying genetic and molecular mechanisms of HD and have shown potential in clinical trials.

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Vesicular Monoamine Transporter 2 (VMAT2) Inhibitor

Valbenazine for Huntington's Disease

Phase 3

Waitlist Available

Research Sponsored by Neurocrine Biosciences

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Must not have

Known history of long QT syndrome, cardiac tachyarrhythmia, left bundle-branch block, atrioventricular (AV) block, uncontrolled bradyarrhythmia, or heart failure

Received gene therapy at any time

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 262 weeks

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing the safety of valbenazine, a medication that helps control involuntary movements in patients with Huntington disease who suffer from chorea. Valbenazine works by balancing brain chemicals to reduce these unwanted movements.

Who is the study for?

This trial is for individuals with Huntington's Disease and chorea who either completed a prior study (NBI-98854-HD3005) or have not used VMAT2 inhibitors in the last 30 days. Participants must be able to walk, understand English, consent to the study, and agree to use contraception if of childbearing potential.

What is being tested?

The trial is testing Valbenazine's long-term safety and effectiveness in treating chorea associated with Huntington’s Disease. It's an open-label Phase 3 study providing ongoing access to Valbenazine for those who need it.

What are the potential side effects?

While specific side effects are not listed here, Valbenazine may cause similar side effects as other medications that affect brain chemistry such as drowsiness, restlessness, nausea, and potentially heart-related issues.

Eligibility Criteria

Exclusion Criteria

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

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I have a history of heart rhythm problems or heart failure.

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I have received gene therapy.

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I have trouble swallowing.

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I have had severe liver problems or specific blood issues during a previous study.

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I do not have any serious or unstable health or mental conditions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 262 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 262 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 262 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change from Baseline in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: ValbenazineExperimental Treatment1 Intervention

Capsule, administered orally once daily.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Valbenazine

2018

Completed Phase 4

~880

0 / 5Eligibility 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

Valbenazine, a VMAT2 inhibitor, works by reducing the release of dopamine, which helps to manage chorea, a common motor symptom in Huntington's Disease (HD). By inhibiting the vesicular monoamine transporter 2 (VMAT2), Valbenazine decreases the excessive dopamine activity that contributes to involuntary movements. This is crucial for HD patients as it can significantly improve their motor function and quality of life. Other treatments, such as Deutetrabenazine, also target VMAT2 and have shown efficacy in reducing chorea with a similar mechanism. These treatments are important as they address one of the most debilitating symptoms of HD, helping to alleviate the burden of the disease on patients and their caregivers.

Indirect tolerability comparison of Deutetrabenazine and Tetrabenazine for Huntington disease.Huntington Disease: Linking Pathogenesis to the Development of Experimental Therapeutics.Effect of Deutetrabenazine on Chorea Among Patients With Huntington Disease: A Randomized Clinical Trial.