Daily Dystonia Practice - A Trial to Investigate NT 201, the Duration of Treatment Effect After One Injection Session and in Long-term Treatment in Cervical Dystonia

Study Overview
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Brief Summary
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The aim of this study is to confirm efficacy and safety of NT 201 (Xeomin®, also known as IncobotulinumtoxinA) after one injection session and to determine the efficacy and safety profile and the duration of treatment effect of NT 201 in long-term treatment with repeated injection sessions.

Official Title
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Prospective, Single-arm, Multicenter Trial to Investigate the Efficacy and Safety of NT 201 and the Duration of Treatment Effect After One Injection Session and in Long-term Treatment in Patients With Cervical Dystonia

Conditions
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Cervical Dystonia

Intervention / Treatment
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* Drug: NT 201


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) - total score at baseline > / = 25 with TWSTRS - severity score > / = 10 and TWSTRS - disability score > / = 3 Patients must be on a stable dose of other medications For pre-treated patients only: Source documentation of the last two consecutive injection sessions with botulinum neurotoxin type A and stable treatment response directly prior to trial entry For pre-treated patients only: At least 10 weeks must have passed between the last injection session of botulinum neurotoxin type A for cervical dystonia and the time of the baseline visit. For pre-treated patients only: The most recent injection session with botulinum neurotoxin for cervical dystonia must have been at a dose of ≤ 300 units of Botox® or Xeomin®, or ≤ 1,200 units Dysport®. Age > / = 18 and < 76 years

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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: NT 201 (50-300 Units)<br>NT 201 (Xeomin®, also know as IncobotulinumtoxinA or Botulinum toxin type A (150 kiloDalton), free from complexing proteins) (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection. | Drug: NT 201<br>* Subjects to receive up to 5 injection sessions, with a total dose of up to 300 Units each. No more than 50 units should have be given at any one injection site during one session.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS), total score: Change from study baseline (initial injection session) to week 4 |  | From baseline to week 4 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| TWSTRS, Single interventional effect | Single interventional effect of one injection session by the change of the TWSTRS total, severity and disability scores from each cycle baseline to 4 weeks thereafter. | From week 10-24 to week 14-28, from week 20-48 to week 24-52, from week 30-72 to week 34-76, and from week 40-96 to week 44-100 | 
| TWSTRS, overall interventional effect | Measured by the change in the TWSTRS total, severity and disability scores, from study baseline to each examination 4 weeks after the injection sessions | From study baseline to week 14-28, 24-52, 34-76, and 44-100 | 
| Global Assessment of Efficacy by Investigator (GAEI) | At the end of each injection cycle | Week 10-24, 20-48, 30-72, and 40-96 | 
| Subject Evaluation of Global Response (PEGR) | At the end of each injection cycle. | Week 10-24, 20-48, 30-72, and 40-96 | 
| Time from last injection session to onset of treatment effect as given by subjective subject assessment | Measured in days. | Up to 4 weeks from last injection session | 
| Time from last injection session to waning of treatment effect as rated by subjective subject assessment | Measured in weeks. | Up to 24 weeks from last injection session | 
| Duration of treatment effect | Defined as time period within an injection cycle from the day of the injection session until the day where the need for reinjection was indicated by the subject (if subject experienced an onset of treatment effect). | Up to 24 weeks from last injection session | 
| Dystonia Discomfort Scale (DDS) score (subject diary), change from baseline |  | From study baseline up to week 100 |