Clinical trial NCT02706795: Dose-escalating Safety and Preliminary Efficacy of DaxibotulinumtoxinA for Injection in Cervical Dystonia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Torticollis (Spasmodic)
Keywords: Cervical dystonia
Spasmodic torticollis
Lead sponsor: Revance Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2016-03-11
Enrollment: 37
Sex: ALL
Minimum age (years): 30
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: This is an open-label, dose-escalation study to assess the safety and preliminary efficacy of daxibotulinumtoxinA in subjects with isolated cervical dystonia (CD).


Eligibility criteria: Inclusion Criteria:

* Meets diagnostic criteria for isolated cervical dystonia
* Has moderate severity with a baseline Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) - Total score of at least 20 and a TWSTRS-Severity subscale score or at least 15
* Has been using stable doses of focal dystonia medications for at least 3 months and willing to continue through end of study

Exclusion Criteria:

* Cervical dystonia attributable to an underlying etiology, such as traumatic torticollis or tardive torticollis; predominant retrocollis or anterocollis cervical dystonia
* Significant dystonia in other body areas, or is currently being treated with botulinum toxin for dystonia in other areas than isolated cervical dystonia
* Neurological abnormalities other than cervical dystonia
* History of severe dysphagia or aspiration, or current clinically significant swallowing disorder
* Previous neck surgery, phenol injection to the neck muscles, myotomy or denervation surgery in the neck/shoulder region, or intrathecal baclofen
* Marked limitation on passive range of motion that suggests contractures or other structural abnormality, e.g. cervical contractures or cervical spinal deformity
* Profound atrophy of cervical musculature
* Any neurological condition that may place the subject at increased risk with exposure to botulinum toxin type A, including peripheral motor neuropathic diseases such as amyotrophic lateral sclerosis and motor neuropathy, and neuromuscular junctional disorders such as Lambert-Eaton syndrome and myasthenia gravis
* Suboptimal efficacy response to any prior botulinum toxin type A product, when a previous treatment produced more optimal therapeutic response, as judged subjectively by the Investigator
* Previous treatment with any botulinum toxin product for any condition within the 6 months prior to Screening and during the study; presence of any blood coagulation disorder; or on anticoagulation treatment with international normalized ratio (INR) \> 3.5

Primary outcomes:
measure: Improvement of dystonia as measured by change from baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) - Total Score
timeFrame: Week 4
Secondary outcomes:
measure: Change from baseline in Toronto Western Spasmodic Torticollis Rating Scale(TWSTRS) - Total Score
timeFrame: Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24
measure: Change from baseline in TWSTRS subscale scores (Severity, Disability and Pain)
timeFrame: Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24
measure: Duration of effect based on number of weeks from treatment until return of symptoms that warrant treatment
timeFrame: Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24
measure: Percentage of treatment responders with at least a 20% reduction in the TWSTRS - Total score post-treatment
timeFrame: Weeks 2, 4, 6, 9, 12, 16, 20, 24
measure: Patient-rated quality of life measurement based upon change from baseline of the Cervical Dystonia Impact Profile Scale (CDIP-58)
timeFrame: Post-treatment: Weeks 4, 6, 12, 16, 20, 24
measure: Percentage of responders showing improvement on Clinical Global Impression of Change (CGIC)
timeFrame: Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24
Other outcomes:
Locations:
city: Fountain Valley
country: United States
facility: The Parkinson's & Movement Disorder Institute
state: California
city: Colorado Springs
country: United States
facility: Colorado Springs Neurological Associates
state: Colorado
city: Gainesville
country: United States
facility: University of Florida Center for Movement Disorders & Neurorestoration
state: Florida
city: Miami Lakes
country: United States
facility: Precision Research Organization
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Overland Park
country: United States
facility: Kansas City Bone & Joint Clinic
state: Kansas
city: Baton Rouge
country: United States
facility: The NeuroMedical Center Clinic
state: Louisiana
city: Winston-Salem
country: United States
facility: Wake forest Baptist Health
state: North Carolina
city: Cincinnati
country: United States
facility: Riverhills Healthcare, Inc.
state: Ohio
city: Port Royal
country: United States
facility: Coastal Neurology
state: South Carolina
city: Houston
country: United States
facility: Parkinson's Disease Center and Movement Disorder Clinic
state: Texas
Study officials:
affiliation: Sponsor GmbH
name: Nubia Kaba
role: STUDY_DIRECTOR
References:
citation: Jankovic J, Truong D, Patel AT, Brashear A, Evatt M, Rubio RG, Oh CK, Snyder D, Shears G, Comella C. Injectable DaxibotulinumtoxinA in Cervical Dystonia: A Phase 2 Dose-Escalation Multicenter Study. Mov Disord Clin Pract. 2018 Apr 26;5(3):273-282. doi: 10.1002/mdc3.12613. eCollection 2018 May-Jun.