Efficacy and Safety of DYSPORT® Using 2mL Dilution in Adults With Cervical Dystonia.

Cervical Dystonia Clinical Trial

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01753310
• Other study ID #: A-TL-52120-169
• Status: Completed
• Phase: Phase 3
• Start date: January 2013
• Est. completion date: January 2015

Study information

• Verified date: July 2019
• Source: Ipsen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 134
• Est. completion date: January 2015
• Est. primary completion date: October 2014
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cervical Dystonia
• Dystonia
• Dystonic Disorders
• Torticollis

Intervention

Biological:
• Botulinum toxin type A
Intramuscular injection, between 250 and 500 units (U)/vial using 2mL dilution, 1 cycle only

Drug:
• Placebo
Up to 2mL

Locations

Country	Name	City	State
United States	Emory University	Atlanta	Georgia
United States	NeuroTrials Research Inc.	Atlanta	Georgia
United States	University of Colorado at Denver Health Sciences	Aurora	Colorado
United States	North Texas Movement Disorders Institute	Bedford	Texas
United States	East Bay Physician's Group	Berkeley	California
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	Parkinson's & Movement Disorders Center of Boca Raton	Boca Raton	Florida
United States	Tufts Medical Center	Boston	Massachusetts
United States	Rush University Medical Center	Chicago	Illinois
United States	University of Cincinnati Physicians Company, LLC	Cincinnati	Ohio
United States	Rehabilitation Consultants PA	Eagan	Minnesota
United States	Associated Neurologists of Southern Connecticut	Fairfield	Connecticut
United States	Advanced Neurosciences Research	Fort Collins	Colorado
United States	Parkinson's and Movement Disorder Institute	Fountain Valley	California
United States	University of Florida Center for Movement Disorders and Neurorestoration	Gainesville	Florida
United States	Guilford Neurologic Associates; Cone Health Medical Group	Greensboro	North Carolina
United States	Penn State Hershey Neurology	Hershey	Pennsylvania
United States	Baylor College of Medicine	Houston	Texas
United States	University of Texas Health Science Center at Houston	Houston	Texas
United States	Kansas City Bone & Joint Clinic	Kansas City	Kansas
United States	Kingston Neurological Associates	Kingston	New York
United States	Loma Linda University Healthcare, Department of Neurology	Loma Linda	California
United States	USC Keck School of Medicine	Los Angeles	California
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	International Clinical Research Institute	Overland Park	Kansas
United States	Emerald Coast Center for Neurological Disorders	Pensacola	Florida
United States	Island Neurological Associates	Plainview	New York
United States	PD Treatment Center of SW FL	Port Charlotte	Florida
United States	Coastal Neurology	Port Royal	South Carolina
United States	OHSU Center for Health and Healing	Portland	Oregon
United States	UC Davis Medical Center	Sacramento	California
United States	Movement Disorders Center of Arizona, LLC	Scottsdale	Arizona
United States	University of Medicine and Dentistry of New Jersey	Stratford	New Jersey
United States	Atlantic Neuroscience Institute	Summit	New Jersey
United States	Puget Sound Neurology	Tacoma	Washington
United States	University of South Florida	Tampa	Florida
United States	University of Arizona	Tucson	Arizona
United States	Georgetown University Hospital	Washington	District of Columbia
United States	Guilford Neurologic Associates	West Palm Beach	Florida
United States	Premiere Research Institute at Palm Beach Neurology	West Palm Beach	Florida
United States	Wake Forest School of Medicine	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Ipsen

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	4 weeks post-treatment
Secondary	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.	2 weeks post-treatment
Secondary	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.	2 weeks post-treatment
Secondary	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.	2 weeks post-treatment
Secondary	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.	4 weeks post-treatment
Secondary	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.	4 weeks post-treatment
Secondary	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.	4 weeks post-treatment
Secondary	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.	2 weeks post-treatment