Study to Assess the Efficacy and Safety of Dysport® in Cervical Dystonia

Study Overview
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Brief Summary
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The aim is to demonstrate equivalent efficacy and safety in the treatment of the two most frequent forms of cervical dystonia (predominantly rotational torticollis and predominantly laterocollis) with the standard initial dose of 500 units Dysport®. The patients will be assigned to one of the two basic types of cervical dystonia, either predominantly rotational torticollis or predominantly laterocollis on the basis of clinical examination. This will determine which therapy is to be administered, using the clearly defined, structured injection protocols.

Official Title
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Multicentre Open Study on the Efficacy and Safety of Botulinum Toxin A (500 Units Dysport®) in the Treatment of Heterogeneous Forms of Cervical Dystonia

Conditions
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Cervical Dystonia

Intervention / Treatment
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* Biological: Botulinum toxin type A


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: De novo patients with cervical dystonia Outpatient Patients to be of age 18 years or older Written informed consent to participate in the study Exclusion Criteria: Pre-treatment of cervical dystonia with botulinum toxin Pre-treatment with botulinum toxin for any indication other than cervical dystonia within the past 12 months Pure retro- or antecollis Neurological or other diseases which may affect head and neck motor function or neuromuscular transmission, e.g. polyneuropathy with pareses, myasthenia, myopathy, motor neurone diseases, Bekhterev's disease

Ages Eligible for Study
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Minimum Age: 18 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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br> | Biological: Botulinum toxin type A<br>* Active Drug: Botulinum type A toxin (Dysport®) 500 Units / 2.5 ml injected in muscles involved in cervical dystonia<br>* Other names: AbobotulinumtoxinA (Dysport®);|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in the Total Score of the Tsui Rating Scale (Patient in Sitting Position) at the First On-treatment Visit (Week 4 or Week 12) | The Tsui rating scale measures severity and duration of head deviation, shoulder elevation and head tremor. This instrument is based on 4 subscores: Subscore A: amplitude of rotation, deflection (tilt) and ante- / retrocollis (range: 0-9 points) Subscore B: duration of movement (values 1 or 2) Subscore C: severity and duration of shoulder elevation (range: 0-3 points) Subscore D: severity and duration of tremor (range: 0-4 points). The total score was calculated as follows: total score = subscores (A x B) + C + D. The total score ranges between 0 and 25 points. A high total score represents severe CD. The mean change in the total score of the Tsui rating scale (patient in the sitting position) between baseline (Week 0 visit) and the first on-treatment visit (Week 4 or Week 12 visit) is presented. | Baseline to Week 4 or Week 12 (up to 12 weeks) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in the Total Score of the Tsui Rating Scale (Patient in Sitting Position) Between Visit 1 (Week 0) and Visit 3 (Week 12) | The Tsui rating scale measures severity and duration of head deviation, shoulder elevation and head tremor. This instrument is based on 4 subscores: Subscore A: amplitude of rotation, deflection (tilt) and ante- / retrocollis (range: 0-9 points) Subscore B: duration of movement (values 1 or 2) Subscore C: severity and duration of shoulder elevation (range: 0-3 points) Subscore D: severity and duration of tremor (range: 0-4 points). The total score was calculated as follows: total score = subscores (A x B) + C + D. The total score ranges between 0 and 25 points. A high total score represents severe CD. The mean change in the total score of the Tsui rating scale (patient in the sitting position) between baseline (Week 0 visit) and the Week 12 visit is presented. | Baseline to Week 12 | 
| Change in the Total Score of the Tsui Rating Scale (Patient Walking) Between Baseline (Week 0) and Visit 2 (Week 4) and Visit 3 (Week 12) | The Tsui rating scale measures severity and duration of head deviation, shoulder elevation and head tremor. This instrument is based on 4 subscores: Subscore A: amplitude of rotation, deflection (tilt) and ante- / retrocollis (range: 0-9 points) Subscore B: duration of movement (values 1 or 2) Subscore C: severity and duration of shoulder elevation (range: 0-3 points) Subscore D: severity and duration of tremor (range: 0-4 points). The total score was calculated as follows: total score = subscores (A x B) + C + D. The total score ranges between 0 and 25 points. A high total score represents severe CD. The mean changes in the total score of the Tsui rating scale (patient walking) between baseline (Week 0 visit) and the Week 4 and Week 12 visits are presented. | Baseline to Week 4 and Week 12 | 
| Change in the 4 Subscores of the Tsui Rating Scale (Patient in the Sitting Position) Between Visit 1 (Week 0) and Visit 2 (Week 4) and Visit 3 (Week 12) | The Tsui rating scale measures severity and duration of head deviation, shoulder elevation and head tremor. This instrument is based on 4 subscores: Subscore A: amplitude of rotation, deflection (tilt) and ante- / retrocollis (range: 0-9 points) Subscore B: duration of movement (values 1 or 2) Subscore C: severity and duration of shoulder elevation (range: 0-3 points) Subscore D: severity and duration of tremor (range: 0-4 points). A higher score for each subscale represents severe CD symptoms. The total score was calculated as follows: total score = subscores (A x B) + C + D. The total score ranges between 0 and 25 points. A high total score represents severe CD. The mean changes in the subscores A to D of the Tsui rating scale (patient in the sitting position) between baseline (Week 0 visit) and the Week 4 and Week 12 visits are presented. | Baseline to Week 4 and Week 12 | 
| Change in the Craniocervical Dystonia Questionnaire (CDQ-24) Total Score and Subscores Between Visit 1 (Week 0) and Visit 2 (Week 4) and Visit 3 (Week 12) | The CDQ-24 is a disease-specific quality of life (QoL) instrument and was assessed at Visits 1 to 3. It consists of 24 items investigating problems in daily living skills related to CD. This instrument is based on 5 subscales: Stigma, Emotional well-being, Pain, Activities of daily living (ADL), Social/family life to which a number of the 24 items are assigned. There are five possible answers to each item representing increasing severity of impairment (scores 0 to 4). The total scores ranged from 0 to 96 (best to worst QoL). In order to obtain scores of the individual subscales, the total score of each subscale (sum of the individual item scores) was transformed linearly to a 0 to 100 scale (best to worst QoL). The mean changes in the CDQ-24 total score between baseline (Week 0 visit) and the Week 4 and Week 12 visits are presented. | Baseline to Week 4 and Week 12 | 
| Changes in the Items of the Patient Diary Based on Day-to-day Function and Activities, Pain and Duration of Pain Between Visit 1 (Week 0) and Visit 2 (Week 4) and Visit 3 (Week 12) | The weekly recorded patient diary consists of the three items: Day-to-Day Capacities and Activities, Pain and Duration of Pain. Each item was rated by the patient on an 11-point scale ranging from 0 = no problems at all to 10 = most severe problems (the actual wording is adapted to each item in question). The mean changes between the baseline (Week 0 visit) and the Week 4 and Week 12 visits are presented. | Baseline to Week 4 and Week 12 | 
| Categorical Changes in the Items of the Patient Diary Based on Day-to-day Function and Activities, Pain and Duration of Pain Between Visit 1 (Week 0) and Visit 2 (Week 4) and Visit 3 (Week 12) | The weekly recorded patient diary consists of the three items: Day-to-Day Capacities and Activities, Pain and Duration of Pain. Each item was rated by the patient on an 11-point scale ranging from 0 = no problems at all to 10 = most severe problems (the actual wording is adapted to each item in question). The following categorical changes between the baseline (Week 0 visit) and the Week 4 and Week 12 visits are presented: Improvement, No change and Deterioration. | Baseline to Week 4 and Week 12 | 
| Number of Patients Without Pain and/or With a Reduction in Pain Based on a Global Assessment of Pain by the Investigator and by the Patient at Visit 2 (Week 4) and Visit 3 (Week 12) | Global pain was assessed at Visit 2 (Week 4) and Visit 3 (Week 12); investigators and patients assessed change in global pain according to the following response categories: = no pain (anymore) = less pain = no change = more pain The numbers of patients falling under each of these categories as assessed by the investigator and patient at Weeks 4 and 12 are presented. | Week 4 visit and Week 12 visit | 
| Global Assessment of Efficacy by the Investigator and by the Patient at Visit 2 (Week 4) and Visit 3 (Week 12) | At Visit 2 (Week 4) and Visit 3 (Week 12) investigators and patients assessed global efficacy of injection of 500 U Dysport® according to the following response categories: = very good = good = moderate = insufficient The numbers of patients falling under each of these categories as assessed by the investigator and patient at Weeks 4 and 12 are presented. | Week 4 and Week 12 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Dysport, Cervical Dystonia