Dose-escalating Safety and Preliminary Efficacy of DaxibotulinumtoxinA for Injection in Cervical Dystonia
Primary Purpose
Torticollis (Spasmodic)
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
DaxibotulinumtoxinA
Sponsored by
About this trial
This is an interventional treatment trial for Torticollis (Spasmodic) focused on measuring Cervical dystonia, Spasmodic torticollis
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
Sites / Locations
- The Parkinson's & Movement Disorder Institute
- Colorado Springs Neurological Associates
- University of Florida Center for Movement Disorders & Neurorestoration
- Precision Research Organization
- University of South Florida
- Emory University
- Rush University Medical Center
- Kansas City Bone & Joint Clinic
- The NeuroMedical Center Clinic
- Wake forest Baptist Health
- Riverhills Healthcare, Inc.
- Coastal Neurology
- Parkinson's Disease Center and Movement Disorder Clinic
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
daxibotulinumtoxinA (DAXI) for injection
Arm Description
DAXI for injection
Outcomes
Primary Outcome Measures
Improvement of dystonia as measured by change from baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) - Total Score
Secondary Outcome Measures
Change from baseline in Toronto Western Spasmodic Torticollis Rating Scale(TWSTRS) - Total Score
Change from baseline in TWSTRS subscale scores (Severity, Disability and Pain)
Duration of effect based on number of weeks from treatment until return of symptoms that warrant treatment
Percentage of treatment responders with at least a 20% reduction in the TWSTRS - Total score post-treatment
Patient-rated quality of life measurement based upon change from baseline of the Cervical Dystonia Impact Profile Scale (CDIP-58)
Percentage of responders showing improvement on Clinical Global Impression of Change (CGIC)
Full Information
NCT ID
NCT02706795
First Posted
March 8, 2016
Last Updated
October 30, 2019
Sponsor
Revance Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT02706795
Brief Title
Dose-escalating Safety and Preliminary Efficacy of DaxibotulinumtoxinA for Injection in Cervical Dystonia
Study Type
Interventional
2. Study Status
Record Verification Date
October 2019
Overall Recruitment Status
Completed
Study Start Date
September 2015 (undefined)
Primary Completion Date
December 7, 2016 (Actual)
Study Completion Date
July 17, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Revance Therapeutics, Inc.
4. Oversight
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Torticollis (Spasmodic)
Keywords
Cervical dystonia, Spasmodic torticollis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Enrollment
37 (Actual)
8. Arms, Groups, and Interventions
Arm Title
daxibotulinumtoxinA (DAXI) for injection
Arm Type
Experimental
Arm Description
DAXI for injection
Intervention Type
Biological
Intervention Name(s)
DaxibotulinumtoxinA
Intervention Description
Low, mid, and high dose sequential treatments of daxibotulinumtoxinA in dose-escalation safety and efficacy study
Primary Outcome Measure Information:
Title
Improvement of dystonia as measured by change from baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) - Total Score
Time Frame
Week 4
Secondary Outcome Measure Information:
Title
Change from baseline in Toronto Western Spasmodic Torticollis Rating Scale(TWSTRS) - Total Score
Time Frame
Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24
Title
Change from baseline in TWSTRS subscale scores (Severity, Disability and Pain)
Time Frame
Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24
Title
Duration of effect based on number of weeks from treatment until return of symptoms that warrant treatment
Time Frame
Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24
Title
Percentage of treatment responders with at least a 20% reduction in the TWSTRS - Total score post-treatment
Time Frame
Weeks 2, 4, 6, 9, 12, 16, 20, 24
Title
Patient-rated quality of life measurement based upon change from baseline of the Cervical Dystonia Impact Profile Scale (CDIP-58)
Time Frame
Post-treatment: Weeks 4, 6, 12, 16, 20, 24
Title
Percentage of responders showing improvement on Clinical Global Impression of Change (CGIC)
Time Frame
Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24
10. Eligibility
Sex
All
Minimum Age & Unit of Time
30 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Nubia Kaba
Organizational Affiliation
Sponsor GmbH
Official's Role
Study Director
Facility Information:
Facility Name
The Parkinson's & Movement Disorder Institute
City
Fountain Valley
State/Province
California
ZIP/Postal Code
92708
Country
United States
Facility Name
Colorado Springs Neurological Associates
City
Colorado Springs
State/Province
Colorado
ZIP/Postal Code
80907
Country
United States
Facility Name
University of Florida Center for Movement Disorders & Neurorestoration
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32607
Country
United States
Facility Name
Precision Research Organization
City
Miami Lakes
State/Province
Florida
ZIP/Postal Code
33016
Country
United States
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33613
Country
United States
Facility Name
Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Kansas City Bone & Joint Clinic
City
Overland Park
State/Province
Kansas
ZIP/Postal Code
66211
Country
United States
Facility Name
The NeuroMedical Center Clinic
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70810
Country
United States
Facility Name
Wake forest Baptist Health
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Riverhills Healthcare, Inc.
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45212
Country
United States
Facility Name
Coastal Neurology
City
Port Royal
State/Province
South Carolina
ZIP/Postal Code
29935
Country
United States
Facility Name
Parkinson's Disease Center and Movement Disorder Clinic
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
30009213
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.
Results Reference
derived
Learn more about this trial
Dose-escalating Safety and Preliminary Efficacy of DaxibotulinumtoxinA for Injection in Cervical Dystonia
We'll reach out to this number within 24 hrs