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Injectable DaxibotulinumtoxinA in Cervical Dystonia: A Phase 2 Dose‐Escalation Multicenter Study
Author(s) -
Jankovic Joseph,
Truong Daniel,
Patel Atul T.,
Brashear Allison,
Evatt Marian,
Rubio Roman G.,
Oh Chad K.,
Snyder Daniel,
Shears Gill,
Comella Cynthia
Publication year - 2018
Publication title -
movement disorders clinical practice
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.754
H-Index - 18
ISSN - 2330-1619
DOI - 10.1002/mdc3.12613
Subject(s) - medicine , cervical dystonia , spasmodic torticollis , adverse effect , dysphagia , botulinum toxin , dosing , incidence (geometry) , anesthesia , surgery , torticollis , physics , optics
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.