Cervical DystoniaPhase 2NCT06771323
Safety and Effectiveness of Valbenazine as Adjunct Therapy to Botulinum Toxin Injections in Cervical Dystonia
A Phase 2 study testing Valbenazine in people with Cervical Dystonia, run by Virginia Commonwealth University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Cervical Dystonia
- What is being tested?
- Valbenazine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Dec 2024to Jun 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Idiopathic CD (neck musculature first and most prominently affected)
- 18-75 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Virginia Commonwealth University
Richmond, Virginia, 23298
Caileigh Dintino, 804-220-0970, caileigh.dintino@vcuhealth.org
Brian Berman, (804)-628-5276, bermanbd2@vcu.edu
About this study
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.
Read the detailed description
The dopaminergic system has been implicated in the pathophysiology of hyperkinetic movement disorders including dystonia. For example, it is well established that acute and chronic dystonia syndromes can be caused by dopamine receptor-blocking drugs. Several animal and human imaging studies also support the presence of abnormal dopaminergic function in idiopathic forms of dystonia. Although historically dopamine receptor-blocking medications have been used to treat dystonia, early clinical trials have been limited and the results mixed. The atypical neuroleptic clozapine was found to be moderately effective in treating segmental and generalized dystonia, but its usefulness was limited by potential adverse effects. Another atypical neuroleptic risperidone has been reported to be effective in a 4-week trial of five patients with various forms of dystonia. Dopamine depleting medications such as the VMAT2 inhibitor tetrabenazine has also been found helpful in some patients with dystonia, particularly those with tardive dystonia, which has overlapping phenomenology with idiopathic dystonia. The more recently developed VMAT2 inhibitor valbenazine has demonstrated clear benefit in tardive dyskinesia, but their therapeutic benefit in idiopathic dystonia has not been evaluated in a placebo-controlled clinical trial. Off label uses and an open-label study of valbenazine suggest it could provide some benefit and be well tolerated in idiopathic CD patient populations.
- Conditions
- Cervical Dystonia
- Being tested
- Valbenazine, Placebo
- Medical subject headings
- Torticollis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ValbenazineDrug
To assess if valbenazine 80mg daily improves motor symptoms in idiopathic CD patients with persistent symptoms despite current treated with botulinum toxin injections.
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: 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). Receives: Drug: Valbenazine; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: baseline and 12 weeks of treatment
Secondary outcomes (9)
Global Dystonia Severity Rating Scale (GDSRS)
Pain in Dystonia Scale (PIDS), The GDS is a Likert type scale with ratings from 0 to 10 (0 is no dystonia, 1 minimal, 5 moderate and 10 severe dystonia). There are no modifying ratings or weighting factors in the GDS. The total score is the sum of the scores for all the body areas. The maximal total score of the GDS is 140.
Measured: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Hospital Anxiety and Depression Scale (HADS)
It comprises 14 items (7 items each for anxiety and depression), with a score ranging between 0 and 21 for the anxiety and depression subscales. For each subscale the total score is at most 21. A score of ≥11 is considered a clinically significant disorder, whereas a score between 8 and 10 suggests a mild disorder.
Measured: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Pittsburgh Sleep Quality Index (PSQI)
The Pittsburgh Sleep Quality Index (PSQI) is a self-rated questionnaire which assesses sleep quality and disturbances over a 1-month time interval. Scores for each question range from 0 to 3, with higher scores indicating more acute sleep disturbances. Developers have suggested a cut- off score of 5 for the global scale as it correctly identified 88.5% of the patient group in their validation study.
Measured: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Epworth Sleepiness Scale (ESS)
The ESS is a short, self-administered questionnaire designed to measure sleep propensity in a simple, standardized way (only 8 questions on a total scale of 0 to 24).
Measured: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Cervical Dystonia Impact Profile - 58 item (CDIP-58)
A Cervical Dystonia Impact Profile-58 (CDIP-58) score represents a patient's self-reported assessment of how much their cervical dystonia impacts their quality of life, with a higher score indicating a greater negative impact across various aspects like pain, movement limitations, sleep disturbances, mood, and social functioning; essentially, a higher score means a more significant impact from their cervical dystonia. The score is calculated from 58 questions, each rated on a scale, resulting in a total score between 0 (no impact) and 100 (severe impact).
Measured: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Clinical Global Impression of Severity scale (CGI-S)
The Clinical Global Impression Scale-Severity (CGI-S) is a 7-point categorical scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis.The CGI-Severity (CGI-S) asks the clinician one question: "Considering your total clinical experience with this particular population, how mentally ill is the patient at this time?" which is rated on the following seven-point scale: 1=normal, not at all ill; 2=borderline mentally ill; 3=mildly ill; 4: Moderately ill 5: Markedly ill 6: Severely ill 7: Among the most extremely ill patients
Measured: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Patient Global Impression of Severity scale (PGI-S)
The Patient Global Impression of Severity (PGI-S) scale is a single-item, 6-point, self-administered tool and is used to assess health severity in both health economics evaluation and as an outcome measure in clinical trials in a range of diseases.The highest achievable stage in PGI is Level I, which is for scores 951-1000. In between, an equal width of 50 points has been kept for each Level. In the PGI, Level II means PGI score 901-950, Level III: 851-900, Level IV: 801-850, and so on up to Level IX: 551-600.
Measured: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Columbia Suicide Severity Rating Scale (C-SSRS)
The Columbia-Suicide Severity Rating Scale (C-SSRS) is an assessment tool that. evaluates suicidal ideation and behavior. The scale includes 4 dimensions: hopelessness, suicidal ideation, negative self-evaluation, and hostility. Each of the sub dimensions receives a total score and the sum of all scores gives the overall suicide probability score. Total scores that can be obtained from the scale range from 36 to 144
Measured: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Pain in Dystonia Scale (PIDS)
PIDS offers practicality featuring self-rating of pain intensity using a visual analogue scale, evaluation of pain across body regions, assessment of pain's impact on daily activities, and consideration of external factors that trigger or alleviate pain.The Scale is a 5-point, criterion-based, ordinal scale designed to assess dystonia in eight body regions: eyes, mouth, neck, trunk, and the four extremities (see Appendix I). Raters score dystonia as none (0), slight (1), mild (2), moderate (3), or severe (4).
Measured: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2025
- Study start17 Dec 2024
- Primary completionJun 2027 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cervical DystoniaStudies of the same condition, open and under way.
- More trials run by Virginia Commonwealth UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- Investigator-Initiated prospective double-blind crossover clinical trial
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Virginia Commonwealth University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Brian Berman, Principal investigator, Virginia Commonwealth University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06771323
- Sponsor's protocol ID
- HM20029848
- Status last verified
- Jun 2026
- First submitted
- 8 Jan 2025
- Last updated
- 4 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Effectiveness of Valbenazine as Adjunct Therapy to Botulinum Toxin Injections in Cervical Dystonia
- ClinicalTrials.gov ID
- NCT06771323
- Acronym / sponsor ID
- HM20029848
- Conditions
- Cervical Dystonia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Virginia Commonwealth UniversityUniversity, hospital or other
- Study start
- 17 Dec 2024 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 13 Jan 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The dopaminergic system has been implicated in the pathophysiology of hyperkinetic movement disorders including dystonia. For example, it is well established that acute and chronic dystonia syndromes can be caused by dopamine receptor-blocking drugs. Several animal and human imaging studies also support the presence of abnormal dopaminergic function in idiopathic forms of dystonia. Although historically dopamine receptor-blocking medications have been used to treat dystonia, early clinical trials have been limited and the results mixed. The atypical neuroleptic clozapine was found to be moderately effective in treating segmental and generalized dystonia, but its usefulness was limited by potential adverse effects. Another atypical neuroleptic risperidone has been reported to be effective in a 4-week trial of five patients with various forms of dystonia. Dopamine depleting medications such as the VMAT2 inhibitor tetrabenazine has also been found helpful in some patients with dystonia, particularly those with tardive dystonia, which has overlapping phenomenology with idiopathic dystonia. The more recently developed VMAT2 inhibitor valbenazine has demonstrated clear benefit in tardive dyskinesia, but their therapeutic benefit in idiopathic dystonia has not been evaluated in a placebo-controlled clinical trial. Off label uses and an open-label study of valbenazine suggest it could provide some benefit and be well tolerated in idiopathic CD patient populations.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- Investigator-Initiated prospective double-blind crossover clinical trial
| Arm | Type | Receives |
|---|---|---|
| Placebo for their initial injection cycle then switch onto Valbenazine for next injections. | Experimental | Drug: Valbenazine, Drug: PlaceboSubject 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) |
| Albenazine for the first injection cycle then switch to Placebo for next injections | Experimental | Drug: Valbenazine, Drug: PlaceboSubject 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 may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Idiopathic CD (neck musculature first and most prominently affected)
- 18-75 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)
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ValbenazineTo assess if valbenazine 80mg daily improves motor symptoms in idiopathic CD patients with persistent symptoms despite current treated with botulinum toxin injections. | Drug | 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. |
| PlaceboPlacebo | Drug | 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. |
Primary outcome
Toronto Western Spasmodic Torticollis Rating Scale-2 (TWSTRS)-severity score change at 12 weeks of treatment
Time frame: baseline and 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
9 secondary outcomes
Global Dystonia Severity Rating Scale (GDSRS)
Time frame: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Hospital Anxiety and Depression Scale (HADS)
Time frame: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Pittsburgh Sleep Quality Index (PSQI)
Time frame: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Epworth Sleepiness Scale (ESS)
Time frame: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Cervical Dystonia Impact Profile - 58 item (CDIP-58)
Time frame: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Clinical Global Impression of Severity scale (CGI-S)
Time frame: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Patient Global Impression of Severity scale (PGI-S)
Time frame: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Columbia Suicide Severity Rating Scale (C-SSRS)
Time frame: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Pain in Dystonia Scale (PIDS)
Time frame: Visit 2 (Baseline, Week 1) and Visit 3 (Week 6)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2026.
- Individual participant dataSharing planned: No
Related trials for Cervical Dystonia
Enrolling by invitationPhase 2
Accelerating TMS for Cervical Dystonia
Isolated Cervical Dystonia
RecruitingPhase 2
A Study to Evaluate the Efficacy, Safety, and Tolerability of VIM0423 in Individuals With Isolated Dystonia
Dystonia
Not yet recruitingObservational study
Assessment of Cervical Range of Motion Restrictions in Infants With Arthrogenic Torticollis - A Feasibility Study
Torticollis (Arthrogenic)
Recruiting
Effect of Vibro-tactile Stimulation Intensity on Head Posture in Cervical Dystonia
Cervical Dystonia
Active, not recruitingObservational study
An Observational Study of Individuals With Isolated Dystonia
Isolated/Primary Dystonia That Affects More Than One Region of the Body
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Brian Berman principal investigator
Virginia Commonwealth University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Valbenazine in people with Cervical Dystonia, run by Virginia Commonwealth University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ananna Zaman (8043686610, Ananna.Zaman@vcuhealth.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Toronto Western Spasmodic Torticollis Rating Scale-2 (TWSTRS)-severity score change at 12 weeks of treatment (time frame: baseline and 12 weeks of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Jun 2026.