Back to resultsEfficacy and Safety of DYSPORT® Using 2mL Dilution in Adults With Cervical Dystonia.
Primary Purpose
Cervical Dystonia
Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Botulinum toxin type A
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Cervical Dystonia
Eligibility Criteria
Inclusion Criteria:
- Primary diagnosis of Cervical Dystonia at least 9 months since onset and either previously untreated with botulinum toxin or currently treated with Botox at a total dosing range of 100-200 U and ≤60 U in the sternocleidomastoid muscle at the last injection cycle, and having had a satisfactory treatment response in the principal investigator's judgment during the last two sequential Botox treatment cycles.
- TWSTRS total score≥ 20; TWSTRS-severity subscale score> 10;
Exclusion Criteria:
- In apparent remission from Cervical Dystonia
- Diagnosis of pure retrocollis or pure anterocollis
- For non-naïve subjects, previous poor response to either of the last two Botox treatments
- Known requirement of <100U or >200U of Botox injected into the neck muscles
Sites / Locations
- University of Alabama at Birmingham
- Movement Disorders Center of Arizona, LLC
- University of Arizona
- East Bay Physician's Group
- Parkinson's and Movement Disorder Institute
- Loma Linda University Healthcare, Department of Neurology
- USC Keck School of Medicine
- UC Davis Medical Center
- University of Colorado at Denver Health Sciences
- Advanced Neurosciences Research
- Associated Neurologists of Southern Connecticut
- Georgetown University Hospital
- Parkinson's & Movement Disorders Center of Boca Raton
- University of Florida Center for Movement Disorders and Neurorestoration
- Emerald Coast Center for Neurological Disorders
- PD Treatment Center of SW FL
- University of South Florida
- Guilford Neurologic Associates
- Premiere Research Institute at Palm Beach Neurology
- Emory University
- NeuroTrials Research Inc.
- Rush University Medical Center
- Kansas City Bone & Joint Clinic
- International Clinical Research Institute
- Tufts Medical Center
- Rehabilitation Consultants PA
- University of Medicine and Dentistry of New Jersey
- Atlantic Neuroscience Institute
- Kingston Neurological Associates
- Fazzini Parkinson's Disease & Dystonia Center
- The Ichan School of Medicine at Mount Sinai
- Island Neurological Associates
- Guilford Neurologic Associates; Cone Health Medical Group
- Wake Forest School of Medicine
- University of Cincinnati Physicians Company, LLC
- OHSU Center for Health and Healing
- Penn State Hershey Neurology
- Coastal Neurology
- North Texas Movement Disorders Institute
- Baylor College of Medicine
- University of Texas Health Science Center at Houston
- Puget Sound Neurology
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Placebo Comparator
Arm Label
Dysport®
Placebo
Arm Description
Dysport® (intramuscular injection), between 250 and 500 units (U)/vial using 2mL dilution, 1 cycle only
Placebo, up to 2mL
Outcomes
Primary Outcome Measures
Change From Baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score at Week 4.
The change from baseline in the TWSTRS total score at Week 4 was determined for the subjects who received a single dose of Dysport® or placebo by intramuscular injection at the baseline visit (Day 1), and is expressed as weighted overall treatment difference. The TWSTRS is an assessment scale used to measure the impact of CD on subjects, and comprises 3 subscales: severity, disability and pain, each of which is scored independently. The total score from the 3 subscales gives the TWSTRS total score with a value from 0 to 85 (best to worst). The score was assessed by the investigator prior to study treatment at baseline and at all post-treatment visits.
Secondary Outcome Measures
Change From Baseline in TWSTRS Total Score at Week 2.
The change from baseline in the TWSTRS total score at Week 2 was determined for the subjects who received a single dose of Dysport® or placebo by intramuscular injection at the baseline visit (Day 1), and is expressed as weighted overall treatment difference. The TWSTRS is an assessment scale used to measure the impact of CD on subjects, and comprises 3 subscales: severity, disability and pain, each of which is scored independently. The total score from the 3 subscales gives the TWSTRS total score with a value from 0 to 85 (best to worst). The score was assessed by the investigator prior to study treatment at baseline and at all post-treatment visits.
Change From Baseline in Clinical Global Impression of Change (CGIC) in CD at Week 2.
The CGIC is an investigator-reported assessment of the global clinical change in CD since study treatment administration. The CGIC uses a seven-point Likert scale ranging from +3 (very much improved) to -3 (very much worse), and was assessed by the investigator at the Week 2 and Week 4 visits.
TWSTRS Responders at Week 2.
Treatment response was determined as the number of responders at Week 2 relative to the baseline TWSTRS total score. A treatment responder is defined as a subject who had at least a 30% reduction in the TWSTRS total score after treatment. This was calculated as ([Week 2 score - baseline score]/baseline score) * 100.
Change From Baseline in CGIC in CD at Week 4.
The CGIC is an investigator-reported assessment of the global clinical change in CD since study treatment administration. The CGIC uses a seven-point Likert scale ranging from +3 (very much improved) to -3 (very much worse), and was assessed by the investigator at the Week 2 and Week 4 visits.
TWSTRS Responders at Week 4.
Treatment response was determined as the number of responders at Week 4 relative to the baseline TWSTRS total score. A treatment responder is defined as a subject who had at least a 30% reduction in the TWSTRS total score after treatment. This was calculated as ([Week 4 score - baseline score]/baseline score) * 100.
Change From Baseline in Cervical Dystonia Impact Profile-58 (CDIP-58) Total Score at Week 4.
The CDIP-58 scale is a subject-based rating scale measuring the health impact of CD measured in 8 health dimensions including head and neck symptoms, pain and discomfort, upper limb activities, walking, sleep, annoyance, mood and psychosocial functioning. Subscale scores were transformed to a common theoretical range of 0 (no impact) to 100 (most impact). Negative changes from the baseline total score indicate improvement in the impact of CD on health whereas postive changes indicate worsening.
Change From Baseline in CDIP-58 Total Score at Week 2.
The CDIP-58 scale is a subject-based rating scale measuring the health impact of CD measured in 8 health dimensions including head and neck symptoms, pain and discomfort, upper limb activities, walking, sleep, annoyance, mood and psychosocial functioning. Subscale scores were transformed to a common theoretical range of 0 (no impact) to 100 (most impact). Negative changes from the baseline total score indicate improvement in the impact of CD on health whereas postive changes indicate worsening. The hierarchical testing procedure would only be conducted if the previous secondary efficacy endpoint (change from baseline in CDIP-58 total score at Week 4) reached a statistically significant treatment effect. This secondary efficacy endpoint (change from baseline in CDIP-58 total score at Week 2) was performed to characterise the full clinical effect.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01753310
Brief Title
Efficacy and Safety of DYSPORT® Using 2mL Dilution in Adults With Cervical Dystonia.
Official Title
A Phase 3b, Multicentre, Randomised, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy and Safety of DYSPORT® Using 2mL Dilution in Adults With Cervical Dystonia.
Study Type
Interventional
2. Study Status
Record Verification Date
July 2019
Overall Recruitment Status
Completed
Study Start Date
January 2013 (undefined)
Primary Completion Date
October 2014 (Actual)
Study Completion Date
January 2015 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ipsen
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of the protocol is to evaluate the efficacy and safety of Dysport® using 2 mL dilution compared with placebo for the treatment of Cervical Dystonia.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cervical Dystonia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
134 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Dysport®
Arm Type
Active Comparator
Arm Description
Dysport® (intramuscular injection), between 250 and 500 units (U)/vial using 2mL dilution, 1 cycle only
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo, up to 2mL
Intervention Type
Biological
Intervention Name(s)
Botulinum toxin type A
Other Intervention Name(s)
AbobotulinumtoxinA (Dysport®)
Intervention Description
Intramuscular injection, between 250 and 500 units (U)/vial using 2mL dilution, 1 cycle only
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Up to 2mL
Primary Outcome Measure Information:
Title
Change From Baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score at Week 4.
Description
The change from baseline in the TWSTRS total score at Week 4 was determined for the subjects who received a single dose of Dysport® or placebo by intramuscular injection at the baseline visit (Day 1), and is expressed as weighted overall treatment difference. The TWSTRS is an assessment scale used to measure the impact of CD on subjects, and comprises 3 subscales: severity, disability and pain, each of which is scored independently. The total score from the 3 subscales gives the TWSTRS total score with a value from 0 to 85 (best to worst). The score was assessed by the investigator prior to study treatment at baseline and at all post-treatment visits.
Time Frame
4 weeks post-treatment
Secondary Outcome Measure Information:
Title
Change From Baseline in TWSTRS Total Score at Week 2.
Description
The change from baseline in the TWSTRS total score at Week 2 was determined for the subjects who received a single dose of Dysport® or placebo by intramuscular injection at the baseline visit (Day 1), and is expressed as weighted overall treatment difference. The TWSTRS is an assessment scale used to measure the impact of CD on subjects, and comprises 3 subscales: severity, disability and pain, each of which is scored independently. The total score from the 3 subscales gives the TWSTRS total score with a value from 0 to 85 (best to worst). The score was assessed by the investigator prior to study treatment at baseline and at all post-treatment visits.
Time Frame
2 weeks post-treatment
Title
Change From Baseline in Clinical Global Impression of Change (CGIC) in CD at Week 2.
Description
The CGIC is an investigator-reported assessment of the global clinical change in CD since study treatment administration. The CGIC uses a seven-point Likert scale ranging from +3 (very much improved) to -3 (very much worse), and was assessed by the investigator at the Week 2 and Week 4 visits.
Time Frame
2 weeks post-treatment
Title
TWSTRS Responders at Week 2.
Description
Treatment response was determined as the number of responders at Week 2 relative to the baseline TWSTRS total score. A treatment responder is defined as a subject who had at least a 30% reduction in the TWSTRS total score after treatment. This was calculated as ([Week 2 score - baseline score]/baseline score) * 100.
Time Frame
2 weeks post-treatment
Title
Change From Baseline in CGIC in CD at Week 4.
Description
The CGIC is an investigator-reported assessment of the global clinical change in CD since study treatment administration. The CGIC uses a seven-point Likert scale ranging from +3 (very much improved) to -3 (very much worse), and was assessed by the investigator at the Week 2 and Week 4 visits.
Time Frame
4 weeks post-treatment
Title
TWSTRS Responders at Week 4.
Description
Treatment response was determined as the number of responders at Week 4 relative to the baseline TWSTRS total score. A treatment responder is defined as a subject who had at least a 30% reduction in the TWSTRS total score after treatment. This was calculated as ([Week 4 score - baseline score]/baseline score) * 100.
Time Frame
4 weeks post-treatment
Title
Change From Baseline in Cervical Dystonia Impact Profile-58 (CDIP-58) Total Score at Week 4.
Description
The CDIP-58 scale is a subject-based rating scale measuring the health impact of CD measured in 8 health dimensions including head and neck symptoms, pain and discomfort, upper limb activities, walking, sleep, annoyance, mood and psychosocial functioning. Subscale scores were transformed to a common theoretical range of 0 (no impact) to 100 (most impact). Negative changes from the baseline total score indicate improvement in the impact of CD on health whereas postive changes indicate worsening.
Time Frame
4 weeks post-treatment
Title
Change From Baseline in CDIP-58 Total Score at Week 2.
Description
The CDIP-58 scale is a subject-based rating scale measuring the health impact of CD measured in 8 health dimensions including head and neck symptoms, pain and discomfort, upper limb activities, walking, sleep, annoyance, mood and psychosocial functioning. Subscale scores were transformed to a common theoretical range of 0 (no impact) to 100 (most impact). Negative changes from the baseline total score indicate improvement in the impact of CD on health whereas postive changes indicate worsening. The hierarchical testing procedure would only be conducted if the previous secondary efficacy endpoint (change from baseline in CDIP-58 total score at Week 4) reached a statistically significant treatment effect. This secondary efficacy endpoint (change from baseline in CDIP-58 total score at Week 2) was performed to characterise the full clinical effect.
Time Frame
2 weeks post-treatment
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Primary diagnosis of Cervical Dystonia at least 9 months since onset and either previously untreated with botulinum toxin or currently treated with Botox at a total dosing range of 100-200 U and ≤60 U in the sternocleidomastoid muscle at the last injection cycle, and having had a satisfactory treatment response in the principal investigator's judgment during the last two sequential Botox treatment cycles.
TWSTRS total score≥ 20; TWSTRS-severity subscale score> 10;
Exclusion Criteria:
In apparent remission from Cervical Dystonia
Diagnosis of pure retrocollis or pure anterocollis
For non-naïve subjects, previous poor response to either of the last two Botox treatments
Known requirement of <100U or >200U of Botox injected into the neck muscles
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director, Neurology, M.D.
Organizational Affiliation
Ipsen
Official's Role
Study Director
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Facility Name
Movement Disorders Center of Arizona, LLC
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85258
Country
United States
Facility Name
University of Arizona
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85724
Country
United States
Facility Name
East Bay Physician's Group
City
Berkeley
State/Province
California
ZIP/Postal Code
94705
Country
United States
Facility Name
Parkinson's and Movement Disorder Institute
City
Fountain Valley
State/Province
California
ZIP/Postal Code
92708
Country
United States
Facility Name
Loma Linda University Healthcare, Department of Neurology
City
Loma Linda
State/Province
California
ZIP/Postal Code
92354
Country
United States
Facility Name
USC Keck School of Medicine
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
UC Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
University of Colorado at Denver Health Sciences
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Advanced Neurosciences Research
City
Fort Collins
State/Province
Colorado
ZIP/Postal Code
80528
Country
United States
Facility Name
Associated Neurologists of Southern Connecticut
City
Fairfield
State/Province
Connecticut
ZIP/Postal Code
06824
Country
United States
Facility Name
Georgetown University Hospital
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
Facility Name
Parkinson's & Movement Disorders Center of Boca Raton
City
Boca Raton
State/Province
Florida
ZIP/Postal Code
33486
Country
United States
Facility Name
University of Florida Center for Movement Disorders and Neurorestoration
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32607
Country
United States
Facility Name
Emerald Coast Center for Neurological Disorders
City
Pensacola
State/Province
Florida
ZIP/Postal Code
32514
Country
United States
Facility Name
PD Treatment Center of SW FL
City
Port Charlotte
State/Province
Florida
ZIP/Postal Code
33980
Country
United States
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33606
Country
United States
Facility Name
Guilford Neurologic Associates
City
West Palm Beach
State/Province
Florida
ZIP/Postal Code
33407
Country
United States
Facility Name
Premiere Research Institute at Palm Beach Neurology
City
West Palm Beach
State/Province
Florida
ZIP/Postal Code
33407
Country
United States
Facility Name
Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
NeuroTrials Research Inc.
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
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
Kansas City
State/Province
Kansas
ZIP/Postal Code
66211
Country
United States
Facility Name
International Clinical Research Institute
City
Overland Park
State/Province
Kansas
ZIP/Postal Code
66210
Country
United States
Facility Name
Tufts Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02111
Country
United States
Facility Name
Rehabilitation Consultants PA
City
Eagan
State/Province
Minnesota
ZIP/Postal Code
55122
Country
United States
Facility Name
University of Medicine and Dentistry of New Jersey
City
Stratford
State/Province
New Jersey
ZIP/Postal Code
08084
Country
United States
Facility Name
Atlantic Neuroscience Institute
City
Summit
State/Province
New Jersey
ZIP/Postal Code
07901
Country
United States
Facility Name
Kingston Neurological Associates
City
Kingston
State/Province
New York
ZIP/Postal Code
12401
Country
United States
Facility Name
Fazzini Parkinson's Disease & Dystonia Center
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
The Ichan School of Medicine at Mount Sinai
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Island Neurological Associates
City
Plainview
State/Province
New York
ZIP/Postal Code
11803
Country
United States
Facility Name
Guilford Neurologic Associates; Cone Health Medical Group
City
Greensboro
State/Province
North Carolina
ZIP/Postal Code
27405
Country
United States
Facility Name
Wake Forest School of Medicine
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
University of Cincinnati Physicians Company, LLC
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45267
Country
United States
Facility Name
OHSU Center for Health and Healing
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Penn State Hershey Neurology
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Coastal Neurology
City
Port Royal
State/Province
South Carolina
ZIP/Postal Code
29935
Country
United States
Facility Name
North Texas Movement Disorders Institute
City
Bedford
State/Province
Texas
ZIP/Postal Code
76201
Country
United States
Facility Name
Baylor College of Medicine
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Texas Health Science Center at Houston
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Puget Sound Neurology
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98409
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
33524060
Citation
Patel AT, Lew MF, Dashtipour K, Isaacson S, Hauser RA, Ondo W, Maisonobe P, Wietek S, Rubin B, Brashear A. Sustained functional benefits after a single set of injections with abobotulinumtoxinA using a 2-mL injection volume in adults with cervical dystonia: 12-week results from a randomized, double-blind, placebo-controlled phase 3b study. PLoS One. 2021 Feb 1;16(2):e0245827. doi: 10.1371/journal.pone.0245827. eCollection 2021. Erratum In: PLoS One. 2021 Apr 14;16(4):e0250475.
Results Reference
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Efficacy and Safety of DYSPORT® Using 2mL Dilution in Adults With Cervical Dystonia.
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