Clinical trial NCT01486264: Open-Label Non-Inferiority Study Evaluating the Efficacy and Safety of Xeomin® in Subjects With Cervical Dystonia Flex
Official title: An Open-Label, Non-Inferiority Study Evaluating the Efficacy and Safety of Two Injection Schedules of Xeomin® (incobotulinumtoxinA) [Short Flex Versus Long Flex] in Subjects With Cervical Dystonia With < 10 Weeks of Benefit From OnabotulinumtoxinA Treatment
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Cervical Dystonia
Lead sponsor: Merz Pharmaceuticals GmbH
Sponsor class: INDUSTRY
Collaborators: name: Merz North America, Inc.
First posted: 2011-12-06
Enrollment: 283
Sex: ALL
Minimum age (years): 18
Maximum age (years): 81
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will compare Xeomin®, a botulinum toxin medication, in shorter treatment intervals (Short Flex dosing) to the standard interval dosing (Long Flex dosing) to determine if the response to treatment is comparable in both how it works and any side effects. Xeomin® is approved by the United States Food and Drug Administration (FDA) for the treatment of cervical dystonia (CD). The use of Xeomin® is investigational in regards to shorter treatment intervals. An investigational use is one that is not approved by the FDA.

Detailed description: Dystonia is a movement disorder which is characterized by sustained, involuntary muscle contractions which frequently causes twisting and repetitive movements or abnormal postures of the trunk, neck, face, or arms and legs. In focal dystonia, the abnormal movements involve a single area of the body. A commonly described form of focal dystonia is cervical dystonia (CD). Botulinum toxin treatment can be offered as a treatment option for the treatment of CD.

The current practice for botulinum toxin injection treatment is to inject patients every 3 months. However, not all patients receive continuing benefit from botulinum toxin injections for an entire 3 months. In a recent survey, approximately 45% of patients report that they would prefer a treatment cycle of less than 10 weeks.This study will compare Xeomin®, a botulinum toxin treatment, in shorter treatment intervals (Short Flex dosing) to the standard interval dosing (Long Flex dosing) to determine if the response to treatment is comparable in both how it works and any side effects. Xeomin® is approved by the United States Food and Drug Administration (FDA) for the treatment of CD. The use of Xeomin® is investigational in regards to shorter treatment intervals. An investigational use is one that is not approved by the FDA.

The purpose of this research study is to evaluate the efficacy of the Short Flex dosing of Xeomin® compared to the Long Flex dosing regimen of Xeomin®, using a standard scale completed by the doctors and subjects as well as questionnaires that ask subjects to rate symptoms of CD.

Eligibility criteria: Inclusion Criteria:

* Documented clinical diagnosis of idiopathic or genetic Cervical Dystonia

Exclusion Criteria:

* Current treatment with botulinum toxin of any type for any other indication (including aesthetic indications) and for any body region during the study.

Primary outcomes:
measure: Change From Baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Severity Subscale Score Based on Blinded Rater Assessment at Week 4 After the 8th Injection
timeFrame: Baseline and Week 4 after the 8th injection (Week 44 to 68 for Short Flex and Week 84 to 104 for Long Flex)
Secondary outcomes:
measure: Change From Baseline in TWSTRS Total Score Based on Blinded Rater Assessment at Week 4 After the 8th Injection
timeFrame: Baseline and Week 4 after the 8th injection (Week 44 to 68 for Short Flex and Week 84 to 104 for Long Flex)
measure: Change From Baseline in TWSTRS Total Score Based on Unblinded Rater Assessment at Week 4 After the 8th Injection
timeFrame: Baseline and Week 4 after the 8th injection (Week 44 to 68 for Short Flex and Week 84 to 104 for Long Flex)
measure: Change From Baseline in TWSTRS Severity Subscale Based on Unblinded Rater Assessment at Week 4 After the 8th Injection
timeFrame: Baseline and Week 4 after the 8th injection (Week 44 to 68 for Short Flex and Week 84 to 104 for Long Flex)
measure: Change From Baseline in TWSTRS Disability Subscale Based on Unblinded Rater Assessment at Week 4 After the 8th Injection
timeFrame: Baseline and Week 4 after the 8th injection (Week 44 to 68 for Short Flex and Week 84 to 104 for Long Flex)
measure: Change From Baseline in TWSTRS Pain Subscale Based on Unblinded Rater Assessment at Week 4 After the 8th Injection
timeFrame: Baseline and Week 4 after the 8th injection (Week 44 to 68 for Short Flex and Week 84 to 104 for Long Flex)
measure: Change From Control Visit Week 4 After First Injection in Investigator-Rated Global Response at Week 4 After the 8th Injection
timeFrame: Week 4 and Week 4 after the 8th injection (Week 44 to 68 for Short Flex and Week 84 to 104 for Long Flex)
measure: Change From Control Visit Week 4 After First Injection in Subject-Rated Global Response at Week 4 After the 8th Injection
timeFrame: Week 4 and Week 4 after the 8th injection (Week 44 to 68 for Short Flex and Week 84 to 104 for Long Flex)
measure: Change From Control Visit Week 4 After First Injection in Subject Satisfaction Score at Week 4 After the 8th Injection
timeFrame: Week 4 and Week 4 after the 8th injection (Week 44 to 68 for Short Flex and Week 84 to 104 for Long Flex)
measure: Change From Baseline in Clinical Global Impression-Severity
timeFrame: Baseline and 8th injection (Week 44 for Short Flex and Week 84 for Long Flex)
measure: Change From Baseline in Cervical Dystonia Impact Profile-58 (CDIP-58) Total Score at Week 4 After the 8th Injection
timeFrame: Baseline and Week 4 after the 8th injection (Week 44 to 68 for Short Flex and Week 84 to 104 for Long Flex)
measure: Change From Baseline in CDIP-58 Subscales' Scores at Week 4 After the 8th Injection
timeFrame: Baseline and Week 4 after the 8th injection (Week 44 to 68 for Short Flex and Week 84 to 104 for Long Flex)
measure: Time to Offset of Xeomin Effects by Injection Cycle
timeFrame: Week 4 up to Week 112
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Merz Investigative Site #001234
state: Alabama
city: Fountain Valley
country: United States
facility: Merz Investigative Site 001017
state: California
city: Loma Linda
country: United States
facility: Merz Investigative Site #001225
state: California
city: Los Angeles
country: United States
facility: Merz Investigative Site #001219
state: California
city: Manchester
country: United States
facility: Merz Investigative Site # 001276
state: Connecticut
city: Washington
country: United States
facility: Merz Investigative Site #001231
state: District of Columbia
city: Boca Raton
country: United States
facility: Merz Investigative Site #001076
state: Florida
city: Gainesville
country: United States
facility: Merz Investigative Site #001019
state: Florida
city: Jacksonville
country: United States
facility: Merz Investigative Site #001046
state: Florida
city: Melbourne
country: United States
facility: Merz Investigative Site #001075
state: Florida
city: Port Charlotte
country: United States
facility: Merz Investigative Site #001217
state: Florida
city: Tampa
country: United States
facility: Merz Investigative Site #1253
state: Florida
city: Atlanta
country: United States
facility: Merz Investigative Site #001055
state: Georgia
city: Chicago
country: United States
facility: Merz Investigative Site# 01255
state: Illinois
city: Chicago
country: United States
facility: Merz Investigative Site #001215
state: Illinois
city: Des Moines
country: United States
facility: Merz Investigative Site # 01069
state: Iowa
city: Overland Park
country: United States
facility: Merz Investigative Site #001110
state: Kansas
city: Elkridge
country: United States
facility: Merz Investigative Site # 001071
state: Maryland
city: Detroit
country: United States
facility: Merz Investigative Site # 001018
state: Michigan
city: Farmington Hills
country: United States
facility: Merz Investigative Site #001030
state: Michigan
city: Eagan
country: United States
facility: Merz Investigative Site # 0001275
state: Minnesota
city: Saint Louis
country: United States
facility: Merz Investigative Site #1250
state: Missouri
city: Saint Louis
country: United States
facility: Merz Investigative Site #001210
state: Missouri
city: Albany
country: United States
facility: Merz Investigative Site #001221
state: New York
city: New York
country: United States
facility: Merz Investigative Site #001233
state: New York
city: New York
country: United States
facility: Merz Investigative Site #1256
state: New York
city: Charlotte
country: United States
facility: Merz Investigative Site# 01252
state: North Carolina
city: Durham
country: United States
facility: Merz Investigative Site #001005
state: North Carolina
city: Raleigh
country: United States
facility: Merz Investigative Site# 01260
state: North Carolina
city: Winston-Salem
country: United States
facility: Merz Investigative Site #001009
state: North Carolina
city: Cincinnati
country: United States
facility: Merz Investigative Site #1265
state: Ohio
city: Tulsa
country: United States
facility: Merz Investigative Site #001220
state: Oklahoma
city: Portland
country: United States
facility: Merz Investigative Site #1033
state: Oregon
city: Portland
country: United States
facility: Merz Investigative Site #1251
state: Oregon
city: Hershey
country: United States
facility: Merz Investigative Site # 0001271
state: Pennsylvania
city: Philadelphia
country: United States
facility: Merz Investigative Site #1249
state: Pennsylvania
city: Nashville
country: United States
facility: Merz Investigative Site #001206
state: Tennessee
city: Dallas
country: United States
facility: Merz Investigative Site #1074
state: Texas
city: Dallas
country: United States
facility: Merz Investigative Site #001223
state: Texas
city: Houston
country: United States
facility: Merz Investigative Site # 001216
state: Texas
city: Houston
country: United States
facility: Merz Investigative Site# 001266
state: Texas
city: Kirkland
country: United States
facility: Merz Investigative Site #001224
state: Washington
city: Seattle
country: United States
facility: Merz Investigative Site #1270
state: Washington
Study officials:
affiliation: Merz North America, Inc.
name: Michael Kulagowski, MD
role: STUDY_DIRECTOR
References:
citation: Comella C, Hauser RA, Isaacson SH, Truong D, Oguh O, Hui J, Molho ES, Brodsky M, Furr-Stimming E, Comes G, Hast MA, Charles D. Efficacy and safety of two incobotulinumtoxinA injection intervals in cervical dystonia patients with inadequate benefit from standard injection intervals of botulinum toxin: Phase 4, open-label, randomized, noninferiority study. Clin Park Relat Disord. 2022 Mar 14;6:100142. doi: 10.1016/j.prdoa.2022.100142. eCollection 2022.