A PHASE IIIb, MULTICENTRE, RANDOMIZED, DOUBLE BLIND, PLACEBO CONTROLLED STUDY EVALUATING THE EFFICACY AND SAFETY OF DYSPORT USING 2 mL DILUTION IN ADULTS WITH CERVICAL DYSTONIA A-TL-52120-169
Description
Detailed Description
A multicentre, randomised, double blind, placebo controlled study where at study entry, subjects will be randomised in a ratio of 2:1 to receive either Dysport or placebo. The primary objective of the study is to evaluate the efficacy and safety of Dysport 500 units (U)/vial using 2 mL dilution compared with placebo for the treatment of cervical dystonia (CD). All subjects are planned to have a single treatment in this study. Following study treatment, follow up visits will be performed at Week 2, Week 4 and Week 12 (+28 days/4 weeks) or early withdrawal due to any reason. All subjects who complete the Week 12 visit will be considered to have completed the study and will be offered entry into an open label extension (OLE) study, which consists of up to three treatment cycles of Dysport using the 2 mL dilution scheme. Between Weeks 4 and 8, subjects may be deemed eligible for early entry into the OLE study, i.e. before they reach the planned Week 12 study visit if rescue treatment is needed. Approximately 132 male and female subjects with CD will be enrolled. The primary efficacy endpoint is the change from baseline in TWSTRS total score at Week 4. Clinical Trial Rationale:The current USPI allows for only one dilution of Dysport: 500 U in 1 mL volume. Feedback obtained from scientific experts and Investigators at medical advisory boards and in market research data has pointed to the lack of scientific data supporting a 2 mL dilution as an obstacle to providing appropriate, safe and effective Dysport utilisation in the USA for subjects suffering from CD. Despite the lack of labelled information, the 2 mL dilution with Dysport reflects real world clinical practice in the USA.The addition of data in the USPI supporting the safety and efficacy of a 2 mL dilution with Dysport will provide the clinician more flexibility in injection volume range to better equip them to meet the needs of a broader spectrum of subjects with CD. Therefore, in this clinical study the majority of enrolled subjects will be previously treated with Botox to reflect the real world clinical scenario in the USA.Statistical analyses will be performed by an external CRO. Statistical evaluation will be performed by using SAS. Exploratory analysis will be performed for each of the tertiary secondary efficacy endpoints using appropriate methods.
Phase
Phase 3 - a treatment has shown activity against a particular disease, where it is either added to existing treatment or compared to the standard treatment.Inclusion and Exclusion 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;
- 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
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California
- USC Keck School of Medicine, Los Angeles, California, 90033
- Parkinson's and Movement Disorder Institute, Fountain Valley, California, 92708
- Loma Linda University Healthcare, Department of Neurology, Loma Linda, California, 92354
- East Bay Physician's Group, Berkeley, California, 94705
- UC Davis Medical Center, Sacramento, California, 95817
- Sutter Cancer Center, Sacramento, California, 95816
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Arizona
- Movement Disorders Center of Arizona, LLC, Scottsdale, Arizona, 85258
- University of Arizona, Tucson, Arizona, 85724
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Oregon
- OHSU Center for Health and Healing, Portland, Oregon, 97239
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Colorado
- University of Colorado at Denver Health Sciences, Aurora, Colorado, 80045
- University of Colorado at Denver Health Sciences, Aurora, Colorado, 80045
- Advanced Neurosciences Research, Fort Collins, Colorado, 80528
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Washington
- Puget Sound Neurology, Tacoma, Washington, 98409
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Texas
- North Texas Movement Disorders Institute, Bedford, Texas, 76201
- Baylor College of Medicine, Houston, Texas, 77030
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Kansas
- International Clinical Research Institute, Overland Park, Kansas, 66210
- Kansas City Bone & Joint Clinic, Kansas City, Kansas, 66211
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Minnesota
- Rehabilitation Consultants PA, Eagan, Minnesota, 55122
- Rehabilitation Consultants PA, Eagan, Minnesota, 55122
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Illinois
- Rush University Medical Center, Chicago, Illinois, 60612
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Florida
- Emerald Coast Center for Neurological Disorders, Pensacola, Florida, 32514
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Ohio
- University of Cincinnati Physicians Company, LLC, Cincinnati, Ohio, 45267
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Georgia
- NeuroTrials Research Inc., Atlanta, Georgia, 30342
- Emory University, Atlanta, Georgia, 30329
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