Valbenazine for Torticollis

Overseen By Ananna Zaman

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Virginia Commonwealth University

Must be taking: Botulinum toxin

Must not be taking: Antipsychotics, VMAT2 inhibitors

Disqualifiers: Deep brain stimulation, Depression, Liver disease, Tardive dyskinesia, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 1

Duration: 12 weeks

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called Valbenazine for individuals with cervical dystonia (CD), a condition that causes neck muscle spasms and pain. Current treatments, such as botulinum toxin (BoNT) injections, often fail to fully relieve symptoms and can cause side effects, while other options may be invasive. The trial aims to determine if Valbenazine can improve symptoms and quality of life for those with CD. Participants should have experienced CD symptoms starting at age 18 or older, with symptoms that are neither too mild nor too severe, and must have been stable on their current treatment for at least 90 days. As a Phase 2 trial, this research focuses on measuring Valbenazine's effectiveness in an initial, smaller group, offering participants a chance to potentially benefit from an innovative treatment.

Will I have to stop taking my current medications?

The trial requires that participants are stable on their current neuroactive medications and have not taken certain medications like dopaminergic or anti-dopaminergic drugs in the last 30 days. If you are on these medications, you may need to stop them before joining the trial.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research suggests that valbenazine could be a safe option for people with cervical dystonia, especially when combined with botulinum toxin injections. One study found that adding valbenazine to this treatment might help relieve symptoms without major safety concerns. Previous studies indicate that it is generally well-tolerated, with no serious side effects reported. While more research is needed, these findings offer hope for better management of this condition. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Valbenazine is unique because it targets the underlying neurological dysfunction in torticollis, a condition characterized by involuntary muscle contractions. Unlike current treatments such as botulinum toxin injections, which primarily focus on symptom relief by temporarily paralyzing the affected muscles, Valbenazine works by modulating neurotransmitters involved in muscle movement. This new mechanism of action offers the potential for more sustained symptom control and fewer side effects. Researchers are excited about Valbenazine because it could provide a more comprehensive approach to managing torticollis, potentially improving quality of life for patients.

What evidence suggests that Valbenazine might be an effective treatment for cervical dystonia?

Research has shown that valbenazine may help treat cervical dystonia (CD), a condition causing neck pain and stiffness. In this trial, participants will receive either valbenazine or a placebo during different injection cycles. Studies have found that combining valbenazine with botulinum toxin (BoNT) treatments can ease CD symptoms, particularly in severe cases. This combination might improve neck movement and reduce pain for people with CD. Valbenazine works by altering certain brain chemicals, which may help control dystonia symptoms. Although more research is needed, early results suggest valbenazine could be a helpful option for those seeking relief from this ongoing condition. ^{1 2 3 4 6}

Who Is on the Research Team?

Brian Berman

Principal Investigator

Virginia Commonwealth University

Are You a Good Fit for This Trial?

This trial is for adults with cervical dystonia, a condition affecting neck muscles, causing pain and limited motion. Participants must be currently receiving botulinum toxin injections but still experiencing symptoms. Those with other forms of dystonia or who can't tolerate the study procedures are excluded.

Inclusion Criteria

My dystonia started after I turned 18 and I don't have a genetic mutation related to movement disorders.

My medication for brain or nerve conditions is not changing.

I am between 18 and 75 years old and my dystonia started after I turned 18.

Exclusion Criteria

History of deep brain stimulation

I have a history of involuntary muscle movements or dystonia.

I get botulinum toxin shots not every 3 months but at different times.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either Valbenazine or Placebo for the first injection cycle, followed by a switch to the alternate treatment for the next cycle

12 weeks

Baseline, Week 1, Week 6, Week 12

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Valbenazine

Trial Overview

The trial tests Valbenazine as an additional treatment to botulinum toxin injections in managing cervical dystonia symptoms. It's a randomized study where some participants will receive Valbenazine while others will get a placebo to compare effectiveness.

How Is the Trial Designed?

Group I: Placebo for their initial injection cycle then switch onto Valbenazine for next injections. Experimental Treatment 2 Interventions

Subject will receive the placebo for their initial injection cycle (for 3 months) and then switch onto Valbenazine at the time of their next injections.(next 3 months)

Group II: Albenazine for the first injection cycle then switch to Placebo for next injections Experimental Treatment 2 Interventions

Subject will receive albenazine for the first injection cycle (duration of 3 months) and then switch onto Placebo at the time of their next injections (remain on it for the next 3 months).

Who Is Running the Clinical Trial?

Virginia Commonwealth University

Lead Sponsor

Trials

732

Recruited

22,900,000+

Neurocrine Biosciences

Industry Sponsor

Trials

78

Recruited

6,600+

Kyle W. Gano

Neurocrine Biosciences

Chief Executive Officer since 2024

PhD in Pharmacology

Dr. Sanjay Keswani

Neurocrine Biosciences

Chief Medical Officer

MD

Published Research Related to This Trial

Valbenazine is the only FDA-approved treatment for tardive dyskinesia (TD), showing a significant response rate of 40% compared to 8.7% for placebo, with a number needed to treat (NNT) of 4, indicating it is effective for this condition.

While valbenazine has a low discontinuation rate due to adverse events (2.9% vs. 1.6% for placebo), it can cause somnolence in 10.9% of patients, and it may prolong the ECG QT interval, highlighting the need for monitoring.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Valbenazine for tardive dyskinesia: A systematic review of the efficacy and safety profile for this newly approved novel medication-What is the number needed to treat, number needed to harm and likelihood to be helped or harmed? Citrome, L.[2022]

Valbenazine is the first FDA-approved medication specifically for treating tardive dyskinesia (TD), showing significant improvement in symptoms at a dose of 80 mg/day with a clinically meaningful effect size of 0.90 in short-term trials.

Compared to tetrabenazine, valbenazine offers advantages such as once-a-day dosing and a better short-term side effect profile, although further long-term studies are needed to fully understand its benefits and risks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Valbenazine for Tardive Dyskinesia. Freudenreich, O., Remington, G.[2022]

Citations

1.

neurology.org

neurology.org/doi/10.1212/WNL.0000000000205482

Efficacy of Valbenazine in the Treatment of Cervical Dystonia

This exploratory study suggests a potential benefit of VBZ for the treatment of cervical dystonia when added to BTX treatment.

2.

frontierspartnerships.org

frontierspartnerships.org/journals/dystonia/articles/10.3389/dyst.2025.13923/full

Efficacy and safety of valbenazine in the treatment of cervical ...

This exploratory study demonstrates a potential benefit in the addition of VBZ for the treatment of cervical dystonia associated with severe pain and posturing.

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06771323

NCT06771323 | Safety and Effectiveness of Valbenazine ...

Dopamine depleting medications such as the VMAT2 inhibitor tetrabenazine has also been found helpful in some patients with dystonia, particularly those with ...

4.

parkinsons.vcu.edu

parkinsons.vcu.edu/news/news-posts/vcu-parkinsons-and-movement-disorders-2024-by-the-numbers--1.html

PMDC study to explore a potential cervical dystonia treatment

... valbenazine could be a safe and effective treatment for chronic dystonia symptoms when paired with botulinum toxin injections. “New ...

5.

ctv.veeva.com

ctv.veeva.com/study/safety-and-effectiveness-of-valbenazine-as-adjunct-therapy-to-botulinum-toxin-injections-in-cervical

Safety and Effectiveness of Valbenazine as Adjunct Therapy ...

Dystonia severity more than minimal and not very severe as defined by Toronto Western Spasmodic Torticollis Rating Scale-2 Motor Severity ( ...

6.

trialx.com

trialx.com/clinical-trials/listings/315946/safety-and-effectiveness-of-valbenazine-as-adjunct-therapy-to-botulinum-toxin-injections-in-cervical-dystonia/

Safety and Effectiveness of Valbenazine as Adjunct ...

Dystonia severity more than minimal and not very severe as defined by Toronto Western Spasmodic Torticollis Rating Scale-2 Motor Severity ( ...