A Phase 3, Open-Label, Multi-Center Trial to Evaluate the Long-Term Safety and Efficacy of Repeat Treatments of DaxibotulinumtoxinA for Injection in Adults with Isolated Cervical Dystonia
Jaroslaw Slawek1, Peter McAllister2, Sebastian Paus3, Daniel Truong4, Todd M. Gross5, Roman G. Rubio5, Pat Kesslak5, Domenico Vitarella5
1Medical University of GdaƄsk and St. Adalbert Hospital, 2New England Institute for Neurology and Headache, 3University of Bonn, 4University of California and The Parkinson and Movement Disorder Institute, 5Revance Therapeutics, Inc.
Objective:
To evaluate the long-term safety of multiple continuous treatments of daxibotulinumtoxinA for injection (DAXI) in subjects with cervical dystonia (CD).
Background:
DAXI is a novel botulinumtoxinA with a proprietary peptide excipient. ASPEN-1, a Phase 3 double blind-placebo controlled study demonstrated efficacy and duration of effect in CD. We report results of open-label safety and efficacy from a large, multicenter, Phase 3 open-label trial of up to 4 continuous treatments of DAXI.
Design/Methods:
Adult with moderate to severe CD were recruited from study centers in the US, Canada, and Europe who were enrolled in ASPEN-1. Additional botulinum toxin (BoNT) treatment-naïve or -experienced adult subjects not enrolled in ASPEN-1 were also enrolled. At baseline, using predefined criteria the investigator selected an open-label dose of DAXI 125 U or 250 U for the subject based on clinical factors, CD severity, and BoNT treatment history. The investigator identified muscles for injection based on clinical presentation (e.g., head, neck, and shoulder position, localization of pain, and muscle hypertrophy). The volume injected per muscle for each dose level was within a predefined dose range per muscle. During each treatment cycle subjects had visits at Weeks 4, 6, 12, and every 4 weeks thereafter up to Week 52 or end of study to assess safety and tolerability. Retreatment was based on loss of 80% of peak treatment effect, or subject request and investigator judgment.  
Results:

Of the 387 subjects screened, 358 subjects were enrolled: 272 (76.0%) rollover from ASPEN-1 and 86 (24.0%) de novo.  Topline safety and efficacy results will be presented.

Conclusions:

Topline safety and efficacy results will be presented.