Safety and Effectiveness of Valbenazine as Adjunct Therapy to Botulinum Toxin Injections in Cervical Dystonia

NCT06771323 | Recruiting Phase 2 FDA Drug

• 1 other identifier: HM20029848
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Cervical Dystonia

Outcome Measures

Primary Outcomes (1)

• Toronto Western Spasmodic Torticollis Rating Scale-2 (TWSTRS)-severity score change at 12 weeks of treatment

  TWSTRS-2-severity score change at 12 weeks of treatment. The isolated TWSTRS-2-Severity score change was chosen as the primary outcome because the TWSTRS-2-Severity had items dropped from the original TWSTRS as they failed to meet criteria for utility in clinimetric testing,11 and because the primary effect of Valbenazine is expected to be on the motor symptoms and less on pain and disability. The Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS-2) uses the following scoring system for severity: 0: Absent, 1: Mild, which is less than one-third of the possible range and can be intermittent or constant 2: Moderate, which is one-third to two-thirds of the possible range and constant, or severe, which is more than two-thirds of the possible range and intermittent, and 3: Severe and constant

  baseline and 12 weeks of treatment

Secondary Outcomes (9)

• Global Dystonia Severity Rating Scale (GDSRS)

  Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)

• Hospital Anxiety and Depression Scale (HADS)

  Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)

• Pittsburgh Sleep Quality Index (PSQI)

  Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)

• Epworth Sleepiness Scale (ESS)

  Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)

• Cervical Dystonia Impact Profile - 58 item (CDIP-58)

  Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)

Study Arms (2)

Placebo for their initial injection cycle then switch onto Valbenazine for next injections.

EXPERIMENTAL

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)

Drug: Valbenazine; Drug: Placebo

Albenazine for the first injection cycle then switch to Placebo for next injections

EXPERIMENTAL

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).

Drug: Valbenazine; Drug: Placebo

Interventions

Valbenazine DRUG

To assess if valbenazine 80mg daily improves motor symptoms in idiopathic CD patients with persistent symptoms despite current treated with botulinum toxin injections.

Albenazine for the first injection cycle then switch to Placebo for next injections; Placebo for their initial injection cycle then switch onto Valbenazine for next injections.

Placebo DRUG

Placebo

Albenazine for the first injection cycle then switch to Placebo for next injections; Placebo for their initial injection cycle then switch onto Valbenazine for next injections.

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Idiopathic CD (neck musculature first and most prominently affected)
• years old (participants excluded if their dystonia symptoms began before age 18 as childhood-onset dystonia typically represents a genetic and/or primary generalized form of dystonia)
• Onset of dystonia ≥18 years old, no known hyperkinetic movement disorder-related genetic mutation
• Dystonia severity more than minimal and not very severe as defined by Toronto Western Spasmodic Torticollis Rating Scale-2 Motor Severity (TWSTRS-2-Severity) score ≥ 5 and ≤ 20.
• Stable on botulinum toxin injections last 90 days (BoNT dose change \<10% and patient reported stability of response over last two injection cycles)
• Stable on other neuroactive medications.

You may not qualify if:

• History of deep brain stimulation
• History of uncontrolled or untreated depression in the prior 3 months, suicidality, or history of suicide attempts
• History of uncontrolled liver disease or failure
• History of tardive dyskinesia or tardive dystonia
• Currently taking dopaminergic and/or anti-dopaminergic medications including VMAT2 inhibitors or other antipsychotic medications
• Exposure to dopaminergic and/or anti-dopaminergic medications including VMAT2 inhibitors or other antipsychotic medications in the last 30 days -Presence of parkinsonism or other movement disorder other than dystonia on exam -Receiving botulinum toxin injections at a planned frequency other than every 3 months or typically receive injections at intervals \<11 weeks or \>13 weeks -Known history of long QT syndrome or cardiac tachyarrhythmia or any clinically significant cardiac abnormality.
• Prolonged QTc as defined by \> 450 msec for men and \> 470 msec for women

Sponsors & Collaborators

• Virginia Commonwealth University: lead

Study Sites (1)

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

RECRUITING

MeSH Terms

Conditions

Torticollis

Interventions

valbenazine

Condition Hierarchy (Ancestors)

Dystonia; Dyskinesias; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Brian Berman

  Virginia Commonwealth University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ananna Zaman

CONTACT

8043686610; Ananna.Zaman@vcuhealth.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Randomization assignment (protocol biostatistician) will be provided to the pharmacy, who will remain unblinded and dispense study drug. The study team and subject will be blinded to the order of procedures.
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Investigator-Initiated prospective double-blind crossover clinical trial

Study Record Dates

• First Submitted: January 8, 2025
• First Posted: January 13, 2025
• Study Start: December 17, 2024
• Primary Completion: April 1, 2026
• Study Completion: April 1, 2026
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)