Apply to Trial

Compensation: Varies

Number of Visits: 1

Duration: 12 weeks

20 Participants Needed

Valbenazine for Torticollis

Overseen ByCaileigh Dintino

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 GroupAll trial participants will receive the active study treatment (no placebo)

Prior Safety DataThis treatment has passed at least one previous human trial

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

Trial Summary

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.

What data supports the effectiveness of the drug Valbenazine for treating torticollis?

Valbenazine is effective in reducing involuntary movements in tardive dyskinesia, a condition similar to torticollis, by decreasing excessive movement. While tetrabenazine, a similar drug, showed limited effectiveness for torticollis, Valbenazine's success in treating other movement disorders suggests potential benefits.¹ ² ³ ⁴ ⁵

How does the drug valbenazine differ from other treatments for torticollis?

Valbenazine is unique because it is a selective VMAT2 inhibitor, originally approved for treating tardive dyskinesia, a different movement disorder. Unlike other treatments like tetrabenazine, valbenazine is taken once daily and has a better side effect profile, potentially leading to fewer and less severe side effects.¹ ³ ⁶ ⁷ ⁸

What is the purpose of this trial?

The most common form of idiopathic dystonia is adult-onset cervical dystonia (CD), a focal form of dystonia affecting the muscles of the neck. CD is often associated with pain and limited range of motion, and frequently leads to reduced quality of life and disability. Effective long-term treatment options are extremely limited. Recurring botulinum neurotoxin (BoNT) injections can ease the symptoms of CD, but they frequently provide only partial relief and can be associated with intolerable side effects. Deep brain stimulation can be used to treat more severe cases of CD, but this neurosurgical procedure is invasive, on average only about 50% effective and may lead to serious adverse effects. Novel treatment approaches for CD are desperately needed to alleviate symptoms and improve the quality of life for the many who suffer from this chronic and disabling neurological disorder.

Research Team

Brian Berman

Principal Investigator

Virginia Commonwealth University

Eligibility Criteria

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.

See 3 more

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.

See 7 more

Timeline

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

Treatment Details

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.

Participant Groups

2Treatment groups

Experimental Treatment

Group I: Placebo for their initial injection cycle then switch onto Valbenazine for next injections.Experimental Treatment2 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 injectionsExperimental Treatment2 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).

Find a Clinic Near You

Who Is Running the Clinical Trial?

Virginia Commonwealth University

Lead Sponsor

Trials

732

Recruited

22,900,000+

Neurocrine Biosciences

Industry Sponsor

Trials

Recruited

6,600+

Kyle W. Gano

Neurocrine Biosciences

Chief Executive Officer since 2024

PhD in Pharmacology

Dr. Sanjay Keswani

Neurocrine Biosciences

Chief Medical Officer

Findings from Research

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]