Clinical trial NCT03608397: 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cervical Dystonia
Keywords: DAXI
Cervical Dystonia
TWSTRS
Toxin
Lead sponsor: Revance Therapeutics, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Syneos Health
First posted: 2018-08-01
Enrollment: 301
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
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.
Primary purpose: TREATMENT

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).

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

Primary outcomes:
measure: Change from Baseline Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-total score
timeFrame: Week 4 and Week 6
Secondary outcomes:
measure: Change from Baseline TWSTRS-total score
timeFrame: Up to 36 Weeks
measure: Duration of effect
timeFrame: Up to 36 Weeks
measure: Patient Global Impression of Change (PGIC) Improvement
timeFrame: Week 4 or Week 6
measure: Incidence of treatment-emergent adverse events (Safety)
timeFrame: Up to 36 Weeks
Other outcomes:
measure: Change in TWSTRS subscale scores
timeFrame: Up to 36 Weeks
measure: Clinical Global Impression of Change (CGIC)
timeFrame: Up to 36 Weeks
measure: Patient Global Impression of Change (PGIC)
timeFrame: Up to 36 Weeks
Locations:
city: Phoenix
country: United States
facility: HOPE Research Institute
state: Arizona
city: Scottsdale
country: United States
facility: Movement Disorders Center of Arizona
state: Arizona
city: Fountain Valley
country: United States
facility: The Parkinsons and Movement Disorder Institute
state: California
city: Irvine
country: United States
facility: University of California, Irvine
state: California
city: Loma Linda
country: United States
facility: Loma Linda University
state: California
city: Los Angeles
country: United States
facility: USC Keck School of Medicine
state: California
city: Pasadena
country: United States
facility: Care Access Research
state: California
city: Sacramento
country: United States
facility: Sutter Institute for Medical Research
state: California
city: Englewood
country: United States
facility: Rocky Mountain Movement Disorders Center
state: Colorado
city: Danbury
country: United States
facility: Associated Neurologist, P.C.
state: Connecticut
city: Stamford
country: United States
facility: New England Institute for Clinical Research
state: Connecticut
city: Washington
country: United States
facility: MedStar Georgetown University Hospital
state: District of Columbia
city: Boca Raton
country: United States
facility: Parkinson's Disease and Movement Disorders Center of Boca Raton
state: Florida
city: Doral
country: United States
facility: Design Neuroscience Center
state: Florida
city: Gainesville
country: United States
facility: University of Florida Center for Movement Disorders and Neurorestoration
state: Florida
city: Hollywood
country: United States
facility: Infinity Clinical Research
state: Florida
city: Jacksonville
country: United States
facility: University of Florida Health Science Center Jacksonville
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Saint Petersburg
country: United States
facility: Suncoast Neuroscience Associates
state: Florida
city: Tampa
country: United States
facility: USF Parkinson's Disease and Movement Disorders Center
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Overland Park
country: United States
facility: Kansas Institute of Reseach
state: Kansas
city: East Lansing
country: United States
facility: Michigan State University
state: Michigan
city: Farmington
country: United States
facility: QUEST Research Institute
state: Michigan
city: West Bloomfield
country: United States
facility: Henry Ford West Bloomfield Hospital
state: Michigan
city: Saint Louis
country: United States
facility: St Louis University
state: Missouri
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: New York
country: United States
facility: Mount Sinai Movement Disorders Center
state: New York
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Winston-Salem
country: United States
facility: Wake Forest Health Sciences
state: North Carolina
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
city: Philadelphia
country: United States
facility: University of Pennsylvania, Department of Neurology
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Port Royal
country: United States
facility: Coastal Neurology
state: South Carolina
city: Cordova
country: United States
facility: Wesley Neurology Clinic
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: Texas Neurology. P.A.
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: Houston Methodist Neurological Institute
state: Texas
city: Round Rock
country: United States
facility: Central Texas Neurology Consultants
state: Texas
city: Burlington
country: United States
facility: University of Vermont Medical Center
state: Vermont
city: Innsbruck
country: Austria
facility: Medical University Innsbruck
city: Wien
country: Austria
facility: Universitaetsklinik fuer Neurologie
city: Toronto
country: Canada
facility: University Health Network, Toronto Western Hospital
state: Ontario
city: Olomouc
country: Czechia
facility: Fakultni nemocnice Olomouc, Neurologicka klinika
city: Ostrava-Poruba
country: Czechia
facility: Fakultní nemocnice Ostrava, Neurologicka klinika
city: Pardubice
country: Czechia
facility: Lekarna Pardubicke nemocnice
city: Praha
country: Czechia
facility: Neurologicka klinika 1. LF UK a VFN v Praze
city: Rychnov Nad Kněžnou
country: Czechia
facility: Vestra Clinics s.r.o.
city: Bron
country: France
facility: Hôpital Neurologique Pierre Wertheimer
city: Grenoble cedex 09
country: France
facility: CHU Grenoble Alpes
city: Lille
country: France
facility: Hôpital Roger Salengro - CHRU de Lille
city: Nimes cedex 09
country: France
facility: CHU Caremeau, Service de Neurologie
city: Berlin
country: Germany
facility: Praxis fuer Neurologie im Bismark Karrée
city: Duesseldorf
country: Germany
facility: Universitaetsklinikum Duesseldorf
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Muenchen
country: Germany
facility: Klinikum rechts der Isar der TUM
city: Troisdorf
country: Germany
facility: GFO Kliniken Troisdorf, Betriebsstätte St. Johannes Sieglar
city: Tübingen
country: Germany
facility: Universitätsklinikum Tübingen
city: Gdansk
country: Poland
facility: Szpital sw. Wojciecha Podmiot Leczniczy Copernicus Sp. Z o.o.
city: Krakow
country: Poland
facility: Marta Dagmara BANACH Marta Banach Specjalistyczny Gabinet Neurologiczny
city: Kraków
country: Poland
facility: Krakowska Akademia Neurologii
city: Olsztyn
country: Poland
facility: Wojewodzki Szpital Specjalistyczny w Olsztynie
city: Warsaw
country: Poland
facility: Centrum Medyczne Pratia Warszawa
city: Warszaw
country: Poland
facility: Mazowiecki Szpital Brodnowski Sp. z o.o.
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Barcelona
country: Spain
facility: Hospital de la Santa Creu i Sant Pau
city: Burgos
country: Spain
facility: Hospital Universitario Burgos
city: Madrid
country: Spain
facility: Hospital Universitario de La Princesa
city: Exeter
country: United Kingdom
facility: Royal Devon and Exeter Foundation Trust Hospital
city: Liverpool
country: United Kingdom
facility: The Walton Centre NHS Foundation Trust
city: Salford
country: United Kingdom
facility: Salford Royal NHS Foundation Trust
Study officials:
References: