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Single Treatment of DaxibotulinumtoxinA for Injection in Adults With Isolated Cervical Dystonia (ASPEN-1)

Phase 3
Completed
Conditions
Cervical Dystonia
Registration Number
NCT03608397
Lead Sponsor
Revance Therapeutics, Inc.
Brief Summary

This is a randomized, double-blind, placebo-controlled, parallel group, multi-center trial of two doses of daxibotulinumtoxinA (DAXI) for injection (high-dose; low-dose in adult subjects with isolated (primary) cervical dystonia (CD).

Detailed Description

Approximately 300 subjects, recruited from approximately 80 study centers in the United States (US), Canada, and Europe will be randomized to DAXI for injection high dose, DAXI for injection low dose, or placebo group, respectively. Subjects will be stratified by treatment center and history of prior treatment with botulinum neurotoxin (BoNT).

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
301
Inclusion Criteria
  • Adults, 18 to 80 years of age
  • Meets diagnostic criteria for isolated CD (idiopathic; dystonic symptoms localized to the head, neck, shoulder areas) with at least moderate severity at Baseline (Day 1), defined as a TWSTRS-total score of at least 20, with at least 15 on the TWSTRS-Severity subscale, at least 3 on the TWSTRS-Disability subscale, and at least 1 on the TWSTRS-Pain subscale
Exclusion Criteria
  • Cervical dystonia attributable to an underlying etiology, (e.g., traumatic torticollis or tardive torticollis)
  • Predominant retrocollis or anterocollis CD
  • Significant dystonia in other body areas, or is currently being treated with BoNT for dystonia in areas other than those associated with isolated CD
  • Severe dysphagia (Grade 3 or 4 on the Dysphagia Severity Scale) at Screening or Baseline (prior to study treatment)
  • Any neuromuscular neurological conditions that may place the subject at increased risk of morbidity with exposure to BoNT, including peripheral motor neuropathic diseases (e.g., amyotrophic lateral sclerosis and motor neuropathy, and neuromuscular junctional disorders such as Lambert-Eaton syndrome and myasthenia gravis)
  • Previous treatment with any BoNT product for any condition within the 14 weeks prior to Screening
  • Botulinum Neurotoxin Type A (BoNTA), except the investigational daxibotulinumtoxinA, treatment-experienced subjects who had suboptimal or no treatment response to the most recent BoNTA injection for CD, as determined by the investigator, or history of primary or secondary non-response to BoNTA injections, known to have neutralizing antibodies to BoNTA; or have a history of botulinum toxin type B (rimabotulinumtoxinB [Myobloc/Neurobloc]) injection for CD due to non-response or suboptimal response to BoNTA

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Primary Outcome Measures
NameTimeMethod
Change from Baseline Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-total scoreWeek 4 and Week 6

TWSTRS is used to assess the severity of cervical Dystonia and the success of its treatment. The average of the change from baseline in TWSTRS-total score at Weeks 4 and 6 will be determined. TWSTRS-total score has a minimum score of 0 and a maximum score of 85, where higher scores represent worse outcomes. It is made up of the summation of 3 subscales: the Torticollis Severity Scale (minimum score of 0, maximum score of 35), the Disability Scale (minimum score of 0, maximum score of 30), and the Pain Scale (minimum score of 0, maximum score of 20).

Secondary Outcome Measures
NameTimeMethod
Duration of effectUp to 36 Weeks

Duration of effect based on target TWSTRS score

Patient Global Impression of Change (PGIC) ImprovementWeek 4 or Week 6

Percentage responders at Week 4 or 6

Incidence of treatment-emergent adverse events (Safety)Up to 36 Weeks

Evaluation of adverse events and serious adverse events over the course of the study.

Change from Baseline TWSTRS-total scoreUp to 36 Weeks

Change from baseline in TWSTRS-total score (all post-treatment time points)

Trial Locations

Locations (75)

HOPE Research Institute

🇺🇸

Phoenix, Arizona, United States

Movement Disorders Center of Arizona

🇺🇸

Scottsdale, Arizona, United States

The Parkinsons and Movement Disorder Institute

🇺🇸

Fountain Valley, California, United States

University of California, Irvine

🇺🇸

Irvine, California, United States

Loma Linda University

🇺🇸

Loma Linda, California, United States

USC Keck School of Medicine

🇺🇸

Los Angeles, California, United States

Care Access Research

🇺🇸

Pasadena, California, United States

Sutter Institute for Medical Research

🇺🇸

Sacramento, California, United States

Rocky Mountain Movement Disorders Center

🇺🇸

Englewood, Colorado, United States

Associated Neurologist, P.C.

🇺🇸

Danbury, Connecticut, United States

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HOPE Research Institute
🇺🇸Phoenix, Arizona, United States
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