Clinical trial NCT00541905: 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
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Cervical Dystonia
Lead sponsor: Merz Pharmaceuticals GmbH
Sponsor class: INDUSTRY
First posted: 2007-10-10
Enrollment: 82
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS), total score: Change from study baseline (initial injection session) to week 4
timeFrame: From baseline to week 4
Secondary outcomes:
measure: TWSTRS, Single interventional effect
timeFrame: 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
measure: TWSTRS, overall interventional effect
timeFrame: From study baseline to week 14-28, 24-52, 34-76, and 44-100
measure: Global Assessment of Efficacy by Investigator (GAEI)
timeFrame: Week 10-24, 20-48, 30-72, and 40-96
measure: Subject Evaluation of Global Response (PEGR)
timeFrame: Week 10-24, 20-48, 30-72, and 40-96
measure: Time from last injection session to onset of treatment effect as given by subjective subject assessment
timeFrame: Up to 4 weeks from last injection session
measure: Time from last injection session to waning of treatment effect as rated by subjective subject assessment
timeFrame: Up to 24 weeks from last injection session
measure: Duration of treatment effect
timeFrame: Up to 24 weeks from last injection session
measure: Dystonia Discomfort Scale (DDS) score (subject diary), change from baseline
timeFrame: From study baseline up to week 100
Other outcomes:
Locations:
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover (LKP)
Study officials:
affiliation: Hannover Medical School
name: Prof. Dirk Dressler, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Dressler D, Kupsch A, Paus S, Seitzinger A, Gebhardt B. Sustained efficacy of Incobotulinumtoxin-A (Xeomin®; Botulinum Neurotoxin Type-A, free from Complexing Proteins) in Long-Term Treatment of Cervical Dystonia. European Journal of Neurology 18 (Suppl. 2): 482, 2011.
citation: Dressler D, Paus S, Seitzinger A, Gebhardt B, Kupsch A. Long-term efficacy and safety of incobotulinumtoxinA injections in patients with cervical dystonia. J Neurol Neurosurg Psychiatry. 2013 Sep;84(9):1014-9. doi: 10.1136/jnnp-2012-303608. Epub 2013 May 18.