Efficacy and Safety of DYSPORT® Using 2mL Dilution in Adults With Cervical Dystonia.
- Conditions
- Cervical Dystonia
- Interventions
- Biological: Botulinum toxin type ADrug: Placebo
- Registration Number
- NCT01753310
- Lead Sponsor
- Ipsen
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 134
- 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;
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Dysport® Botulinum toxin type A Dysport® (intramuscular injection), between 250 and 500 units (U)/vial using 2mL dilution, 1 cycle only Placebo Placebo Placebo, up to 2mL
- Primary Outcome Measures
Name Time Method Change From Baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score at Week 4. 4 weeks post-treatment 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
Name Time Method Change From Baseline in Cervical Dystonia Impact Profile-58 (CDIP-58) Total Score at Week 4. 4 weeks post-treatment 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. 2 weeks post-treatment 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.
Change From Baseline in TWSTRS Total Score at Week 2. 2 weeks post-treatment 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. 2 weeks post-treatment 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. 2 weeks post-treatment 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. 4 weeks post-treatment 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. 4 weeks post-treatment 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.
Trial Locations
- Locations (42)
Loma Linda University Healthcare, Department of Neurology
🇺🇸Loma Linda, California, United States
Guilford Neurologic Associates
🇺🇸West Palm Beach, Florida, United States
NeuroTrials Research Inc.
🇺🇸Atlanta, Georgia, United States
Rush University Medical Center
🇺🇸Chicago, Illinois, United States
Tufts Medical Center
🇺🇸Boston, Massachusetts, United States
University of Cincinnati Physicians Company, LLC
🇺🇸Cincinnati, Ohio, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
University of Texas Health Science Center at Houston
🇺🇸Houston, Texas, United States
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
Island Neurological Associates
🇺🇸Plainview, New York, United States
USC Keck School of Medicine
🇺🇸Los Angeles, California, United States
Parkinson's & Movement Disorders Center of Boca Raton
🇺🇸Boca Raton, Florida, United States
Guilford Neurologic Associates; Cone Health Medical Group
🇺🇸Greensboro, North Carolina, United States
Penn State Hershey Neurology
🇺🇸Hershey, Pennsylvania, United States
UC Davis Medical Center
🇺🇸Sacramento, California, United States
University of Colorado at Denver Health Sciences
🇺🇸Aurora, Colorado, United States
University of South Florida
🇺🇸Tampa, Florida, United States
OHSU Center for Health and Healing
🇺🇸Portland, Oregon, United States
Movement Disorders Center of Arizona, LLC
🇺🇸Scottsdale, Arizona, United States
University of Arizona
🇺🇸Tucson, Arizona, United States
East Bay Physician's Group
🇺🇸Berkeley, California, United States
Advanced Neurosciences Research
🇺🇸Fort Collins, Colorado, United States
Associated Neurologists of Southern Connecticut
🇺🇸Fairfield, Connecticut, United States
University of Florida Center for Movement Disorders and Neurorestoration
🇺🇸Gainesville, Florida, United States
Georgetown University Hospital
🇺🇸Washington, District of Columbia, United States
Premiere Research Institute at Palm Beach Neurology
🇺🇸West Palm Beach, Florida, United States
Emory University
🇺🇸Atlanta, Georgia, United States
Emerald Coast Center for Neurological Disorders
🇺🇸Pensacola, Florida, United States
Kansas City Bone & Joint Clinic
🇺🇸Kansas City, Kansas, United States
International Clinical Research Institute
🇺🇸Overland Park, Kansas, United States
Rehabilitation Consultants PA
🇺🇸Eagan, Minnesota, United States
University of Medicine and Dentistry of New Jersey
🇺🇸Stratford, New Jersey, United States
Atlantic Neuroscience Institute
🇺🇸Summit, New Jersey, United States
Kingston Neurological Associates
🇺🇸Kingston, New York, United States
Fazzini Parkinson's Disease & Dystonia Center
🇺🇸New York, New York, United States
The Ichan School of Medicine at Mount Sinai
🇺🇸New York, New York, United States
Wake Forest School of Medicine
🇺🇸Winston-Salem, North Carolina, United States
Puget Sound Neurology
🇺🇸Tacoma, Washington, United States
Parkinson's and Movement Disorder Institute
🇺🇸Fountain Valley, California, United States
PD Treatment Center of SW FL
🇺🇸Port Charlotte, Florida, United States
Coastal Neurology
🇺🇸Port Royal, South Carolina, United States
North Texas Movement Disorders Institute
🇺🇸Bedford, Texas, United States