Study to Evaluate Safety, Efficacy of Botulinum Toxin Type A in Patients With Cervical Dystonia

Study Overview
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Brief Summary
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Study is to investigate the use of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) scale in a cervical dystonia population treated with botulinum toxin type A, and placebo.

Conditions
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Cervical Dystonia

Intervention / Treatment
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* Biological: botulinum toxin type A
* Biological: botulinum toxin type A Formulation 2
* Drug: Normal Saline


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of cervical dystonia Exclusion Criteria: Current or previous botulinum toxin treatment of any type for any condition

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: botulinum toxin Type A<br>Intramuscular injections into the affected muscles. Maximum dose of 360 units. Subjects may receive up to three treatments. | Biological: botulinum toxin type A<br>* Intramuscular injections into the affected muscles. Maximum dose of 360 units. Subjects may receive up to three treatments.<br>* Other names: BOTOX®;|
| Active Comparator: botulinum toxin Type A Formulation 2<br>Intramuscular injections into the affected muscles. Maximum dose of 360 units. Subjects may receive up to three treatments. | Biological: botulinum toxin type A Formulation 2<br>* Intramuscular injections into the affected muscles. Maximum dose of 360 units. Subjects may receive up to three treatments.<br>|
| Other: Placebo (Normal Saline) / botulinum toxin Type A<br>Intramuscular injections of the assigned study medication into the affected muscles (placebo for treatment cycle 1 and botulinum toxin Type A for subsequent treatments). Maximum dose of 360 units. Subjects may receive up to three treatments. | Biological: botulinum toxin type A<br>* Intramuscular injections into the affected muscles. Maximum dose of 360 units. Subjects may receive up to three treatments.<br>* Other names: BOTOX®;Drug: Normal Saline<br>* Intramuscular injections of placebo (normal saline) into the affected muscles for treatment cycle 1.<br>|
| Other: Placebo (Normal Saline) / botulinum toxin Type A Formulation 2<br>Intramuscular injections of the assigned study medication into the affected muscles (placebo for treatment cycle 1 and botulinum toxin Type A Formulation 2 for subsequent treatments). Maximum dose of 360 units. Subjects may receive up to three treatments. | Biological: botulinum toxin type A Formulation 2<br>* Intramuscular injections into the affected muscles. Maximum dose of 360 units. Subjects may receive up to three treatments.<br>Drug: Normal Saline<br>* Intramuscular injections of placebo (normal saline) into the affected muscles for treatment cycle 1.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in Observed Total Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Score at Week 4 of Treatment Cycle 1 | Change from baseline in observed TWSTRS score at Week 4 of Treatment Cycle 1. The TWSTRS is an assessment scale used to measure the impact of cervical dystonia on patients. The score is comprised of 3 subscales: Severity, Disability, and Pain, each of which is scored independently. The total of these 3 comprises the TWSTRS total score which is scored from 0 (least symptoms) to 85 (worst symptoms). Higher scores indicate a greater degree of symptom severity. A negative change from baseline represents improvement and a positive change from baseline indicates worsening. | Baseline, Week 4 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Physician's Global Assessment of Response to Treatment at Week 4 of Treatment Cycle 1 | Physician's global assessment of response to treatment at Week 4 of Treatment Cycle 1. Responses were measured on a 9-point scale of +4 to -4, with higher scores denoting improvement in cervical dystonia: +4 was 'Complete abolishment of signs and symptoms (100% improvement)', 0 represented 'No change', and -4 represented 'Very marked worsening (about 100% worse or greater)'. | Week 4 | 
| Patient's Global Assessment of Response to Treatment at Week 4 of Treatment Cycle 1 | Patient's global assessment of response to treatment at Week 4 of Treatment Cycle 1. Responses were measured on a 9-point scale of +4 to -4, with higher scores denoting improvement in cervical dystonia: +4 was 'Complete abolishment of signs and symptoms (100% improvement)', 0 represented 'No change', and -4 represented 'Very marked worsening (about 100% worse or greater)'. | Week 4 | 
| Change From Baseline in Pain as Evaluated With the TWSTRS Pain Subscale at Week 4 of Treatment Cycle 1 | Change from baseline in pain as evaluated with the TWSTRS pain subscale at Week 4 of Treatment Cycle 1. The TWSTRS pain subscale scores range from 0 to 20 (0=no pain and 20=worst pain), based on severity of neck pain (0=no pain and 10=worst pain), the duration of pain (0=none and 5=most), and the degree of disability (0=none and 5=most). A negative number change from Baseline represents a decrease in pain (improvement). | Baseline, Week 4 | 
| Duration of Treatment Effect for Treatment Responders | Duration of Treatment Effect for Treatment Responders is defined as the number of days from the date of first treatment to the first visit after Week 4 of Treatment Cycle 1, at which the Total TWSTRS score reaches at least 90% of the baseline score. A treatment responder is defined as a patient who has at least a 30% reduction in Total TWSTRS score at Week 4 after the first treatment. The TWSTRS score measures the impact of cervical dystonia on patients (0=least symptoms and 85= worst symptoms). | Up to 6 Months |