Injectable DaxibotulinumtoxinA in Cervical Dystonia: A Phase 2 Dose-Escalation Multicenter Study

Joseph Jankovic, Daniel Truong, Atul T. Patel, Allison Brashear, Marian Evatt, Roman G. Rubio, Chad K. Oh, Daniel Snyder, Gill Shears, Cynthia Comella

Research output: Contribution to journalArticle

5 Scopus citations

Abstract

Background: Injectable daxibotulinumtoxinA (an investigational botulinum toxin, RT002) may offer a more prolonged duration of response—and therefore less frequent dosing—than onabotulinumtoxinA. Objectives: To perform a phase 2, open-label, dose-escalation study to assess the efficacy and safety of daxibotulinumtoxinA in cervical dystonia. Methods: Subjects with moderate-to-severe isolated cervical dystonia were enrolled in sequential cohorts to receive a single open-label, intramuscular dose of injectable daxibotulinumtoxinA of up to 200 U (n = 12), 200–300 U (n = 12), or 300–450 U (n = 13; clinicaltrials.gov identifier NCT02706795). Results: Overall, 33/37 enrollees completed the trial. DaxibotulinumtoxinA was associated with mean reductions in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-Total score of 16.8 (38%) at week 4, 21.3 (50%) at week 6, and 12.8 (30%) at week 24. The proportion of subjects who were responders (achieved ≥ 20% reduction in TWSTRS-Total score) was 94% at week 6 and 68% at week 24. The median duration of response (time until > 20% of the improvement in TWSTRS-Total score achieved at week 4 was no longer retained or re-treatment was needed) was 25.3 weeks (95% CI, 20.14–26.14 weeks). There were no serious adverse events and there was no apparent dose-related increase in the incidence of adverse events. The most common treatment-related adverse events were dysphagia (14%) and injection site erythema (8%). Conclusions: Preliminary assessments suggest that injectable daxibotulinumtoxinA at doses up to 450 U is well tolerated and may offer prolonged efficacy in the treatment of cervical dystonia. Further studies involving larger numbers of patients are now warranted.

Original languageEnglish (US)
Pages (from-to)273-282
Number of pages10
JournalMovement Disorders Clinical Practice
Volume5
Issue number3
DOIs
StatePublished - May 1 2018
Externally publishedYes

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Keywords

  • botulinum toxin
  • CDIP
  • cervical dystonia
  • daxibotulinumtoxinA
  • TWSTRS

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

Cite this

Jankovic, J., Truong, D., Patel, A. T., Brashear, A., Evatt, M., Rubio, R. G., Oh, C. K., Snyder, D., Shears, G., & Comella, C. (2018). Injectable DaxibotulinumtoxinA in Cervical Dystonia: A Phase 2 Dose-Escalation Multicenter Study. Movement Disorders Clinical Practice, 5(3), 273-282. https://doi.org/10.1002/mdc3.12613