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

• ClinicalTrials.gov Identifier: NCT03608397
• Recruitment Status: Completed
• First Posted: August 1, 2018
• Last Update Posted: December 5, 2022
• Sponsor: Revance Therapeutics, Inc.
• Collaborator: Syneos Health
• Information provided by (Responsible Party): Revance Therapeutics, Inc.

Tracking Information

• First Submitted Date: July 11, 2018
• First Posted Date: August 1, 2018
• Last Update Posted Date: December 5, 2022
• Actual Study Start Date: June 20, 2018
• Actual Primary Completion Date: December 3, 2019 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 23, 2018)

Change from Baseline Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-total score [ Time Frame: Week 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).

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: July 31, 2018)

• Change from Baseline TWSTRS-total score [ Time Frame: Up to 36 Weeks ]
  Change from baseline in TWSTRS-total score (all post-treatment time points)
• Duration of effect [ Time Frame: Up to 36 Weeks ]
  Duration of effect based on target TWSTRS score
• Patient Global Impression of Change (PGIC) Improvement [ Time Frame: Week 4 or Week 6 ]
  Percentage responders at Week 4 or 6
• Incidence of treatment-emergent adverse events (Safety) [ Time Frame: Up to 36 Weeks ]
  Evaluation of adverse events and serious adverse events over the course of the study.

Original Secondary Outcome Measures (submitted: July 23, 2018)

• Change from Baseline TWSTRS-total score [ Time Frame: Up to 36 Weeks ]
  Change from baseline in TWSTRS-total score (all post-treatment time points)
• Duration of effect Duration of effect [ Time Frame: Up to 36 Weeks ]
  Duration of effect based on target TWSTRS score
• Patient Global Impression of Change (PGIC) Improvement [ Time Frame: Week 4 or Week 6 ]
  Percentage responders at Week 4 or 6
• Incidence of treatment-emergent adverse events (Safety) [ Time Frame: Up to 36 Weeks ]
  Evaluation of adverse events and serious adverse events over the course of the study.

Current Other Pre-specified Outcome Measures (submitted: July 23, 2018)

• Change in TWSTRS subscale scores [ Time Frame: Up to 36 Weeks ]
  Change from baseline in TWSTRS subscale scores (TWSTRS-Severity, TWSTRS-Disability, and TWSTRS-Pain) (all post-treatment time points)
• Clinical Global Impression of Change (CGIC) [ Time Frame: Up to 36 Weeks ]
  CGIC at all post-treatment time points
• Patient Global Impression of Change (PGIC) [ Time Frame: Up to 36 Weeks ]
  PGIC at all post-treatment time points

• Original Other Pre-specified Outcome Measures: Same as current

Descriptive Information

• Brief Title: Single Treatment of DaxibotulinumtoxinA for Injection in Adults With Isolated Cervical Dystonia (ASPEN-1)
• Official Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multi-Center Trial to Evaluate the Efficacy and Safety of a Single Treatment of DaxibotulinumtoxinA for Injection in Adults With Isolated Cervical Dystonia (ASPEN-1)
• 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).
• Study Type: Interventional
• Study Phase: Phase 3

Study Design

Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:

Approximately 300 subjects, recruited from study centers in the United States (US), Canada, and Europe will be randomized to DAXI high dose, DAXI low dose or placebo group.

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:

Double Blinded

Primary Purpose: Treatment

• Condition: Cervical Dystonia

Intervention

• Biological: DaxibotulinumtoxinA for injection
  DaxibotulinumtoxinA for injection is a sterile, white to off-white lyophilized product containing the active ingredient, daxibotulinumtoxinA, and inactive ingredients to be reconstituted with sterile, non-preserved, 0.9% sodium chloride solution saline.
• Biological: Placebo
  Placebo is a sterile lyophilized product consisting of inactive ingredients without the neurotoxin to be reconstituted with sterile, non-preserved 0.9% sodium chloride solution.

Study Arms

• Experimental: DAXI for injection low dose
  Low Dose Group
  Intervention: Biological: DaxibotulinumtoxinA for injection
• Experimental: DAXI for injection high dose
  High Dose Group
  Intervention: Biological: DaxibotulinumtoxinA for injection
• Placebo Comparator: Placebo
  Placebo Group
  Intervention: Biological: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: July 23, 2018): 301
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: June 16, 2020
• Actual Primary Completion Date: December 3, 2019 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 80 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Austria, Canada, Czechia, France, Germany, Poland, Spain, United Kingdom, United States

Administrative Information

• NCT Number: NCT03608397
• Other Study ID Numbers: 1720302
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Revance Therapeutics, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Revance Therapeutics, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Syneos Health
• Investigators: Not Provided
• PRS Account: Revance Therapeutics, Inc.
• Verification Date: November 2022