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

Primary Purpose

Cervical Dystonia

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
DaxibotulinumtoxinA for injection
Placebo
Sponsored by
Revance Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cervical Dystonia focused on measuring DAXI, Cervical Dystonia, TWSTRS, Toxin

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • HOPE Research Institute
  • Movement Disorders Center of Arizona
  • The Parkinsons and Movement Disorder Institute
  • University of California, Irvine
  • Loma Linda University
  • USC Keck School of Medicine
  • Care Access Research
  • Sutter Institute for Medical Research
  • Rocky Mountain Movement Disorders Center
  • Associated Neurologist, P.C.
  • New England Institute for Clinical Research
  • MedStar Georgetown University Hospital
  • Parkinson's Disease and Movement Disorders Center of Boca Raton
  • Design Neuroscience Center
  • University of Florida Center for Movement Disorders and Neurorestoration
  • Infinity Clinical Research
  • University of Florida Health Science Center Jacksonville
  • University of Miami
  • Suncoast Neuroscience Associates
  • USF Parkinson's Disease and Movement Disorders Center
  • Emory University
  • Rush University Medical Center
  • Kansas Institute of Reseach
  • Michigan State University
  • QUEST Research Institute
  • Henry Ford West Bloomfield Hospital
  • St Louis University
  • Washington University
  • University of Nebraska Medical Center
  • Mount Sinai Movement Disorders Center
  • University of Rochester
  • Duke University
  • Wake Forest Health Sciences
  • Thomas Jefferson University
  • University of Pennsylvania, Department of Neurology
  • Medical University of South Carolina
  • Coastal Neurology
  • Wesley Neurology Clinic
  • Vanderbilt University Medical Center
  • Texas Neurology. P.A.
  • Baylor College of Medicine
  • Houston Methodist Neurological Institute
  • Central Texas Neurology Consultants
  • University of Vermont Medical Center
  • Medical University Innsbruck
  • Universitaetsklinik fuer Neurologie
  • University Health Network, Toronto Western Hospital
  • Fakultni nemocnice Olomouc, Neurologicka klinika
  • Fakultní nemocnice Ostrava, Neurologicka klinika
  • Lekarna Pardubicke nemocnice
  • Neurologicka klinika 1. LF UK a VFN v Praze
  • Vestra Clinics s.r.o.
  • Hôpital Neurologique Pierre Wertheimer
  • CHU Grenoble Alpes
  • Hôpital Roger Salengro - CHRU de Lille
  • CHU Caremeau, Service de Neurologie
  • Praxis fuer Neurologie im Bismark Karrée
  • Universitaetsklinikum Duesseldorf
  • Medizinische Hochschule Hannover
  • Klinikum rechts der Isar der TUM
  • GFO Kliniken Troisdorf, Betriebsstätte St. Johannes Sieglar
  • Universitätsklinikum Tübingen
  • Szpital sw. Wojciecha Podmiot Leczniczy Copernicus Sp. Z o.o.
  • Marta Dagmara BANACH Marta Banach Specjalistyczny Gabinet Neurologiczny
  • Krakowska Akademia Neurologii
  • Wojewodzki Szpital Specjalistyczny w Olsztynie
  • Centrum Medyczne Pratia Warszawa
  • Mazowiecki Szpital Brodnowski Sp. z o.o.
  • Hospital Universitari Vall d'Hebron
  • Hospital de la Santa Creu i Sant Pau
  • Hospital Universitario Burgos
  • Hospital Universitario de La Princesa
  • Royal Devon and Exeter Foundation Trust Hospital
  • The Walton Centre NHS Foundation Trust
  • Salford Royal NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

DAXI for injection low dose

DAXI for injection high dose

Placebo

Arm Description

Low Dose Group

High Dose Group

Placebo Group

Outcomes

Primary Outcome Measures

Change from Baseline Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-total score
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

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

Full Information

First Posted
July 11, 2018
Last Updated
November 29, 2022
Sponsor
Revance Therapeutics, Inc.
Collaborators
Syneos Health
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1. Study Identification

Unique Protocol Identification Number
NCT03608397
Brief Title
Single Treatment of DaxibotulinumtoxinA for Injection in Adults With Isolated Cervical Dystonia (ASPEN-1)
Acronym
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

2. Study Status

Record Verification Date
November 2022
Overall Recruitment Status
Completed
Study Start Date
June 20, 2018 (Actual)
Primary Completion Date
December 3, 2019 (Actual)
Study Completion Date
June 16, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Revance Therapeutics, Inc.
Collaborators
Syneos Health

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cervical Dystonia
Keywords
DAXI, Cervical Dystonia, TWSTRS, Toxin

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
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
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
Double Blinded
Allocation
Randomized
Enrollment
301 (Actual)

8. Arms, Groups, and Interventions

Arm Title
DAXI for injection low dose
Arm Type
Experimental
Arm Description
Low Dose Group
Arm Title
DAXI for injection high dose
Arm Type
Experimental
Arm Description
High Dose Group
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo Group
Intervention Type
Biological
Intervention Name(s)
DaxibotulinumtoxinA for injection
Intervention Description
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.
Intervention Type
Biological
Intervention Name(s)
Placebo
Intervention Description
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.
Primary Outcome Measure Information:
Title
Change from Baseline Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-total score
Description
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).
Time Frame
Week 4 and Week 6
Secondary Outcome Measure Information:
Title
Change from Baseline TWSTRS-total score
Description
Change from baseline in TWSTRS-total score (all post-treatment time points)
Time Frame
Up to 36 Weeks
Title
Duration of effect
Description
Duration of effect based on target TWSTRS score
Time Frame
Up to 36 Weeks
Title
Patient Global Impression of Change (PGIC) Improvement
Description
Percentage responders at Week 4 or 6
Time Frame
Week 4 or Week 6
Title
Incidence of treatment-emergent adverse events (Safety)
Description
Evaluation of adverse events and serious adverse events over the course of the study.
Time Frame
Up to 36 Weeks
Other Pre-specified Outcome Measures:
Title
Change in TWSTRS subscale scores
Description
Change from baseline in TWSTRS subscale scores (TWSTRS-Severity, TWSTRS-Disability, and TWSTRS-Pain) (all post-treatment time points)
Time Frame
Up to 36 Weeks
Title
Clinical Global Impression of Change (CGIC)
Description
CGIC at all post-treatment time points
Time Frame
Up to 36 Weeks
Title
Patient Global Impression of Change (PGIC)
Description
PGIC at all post-treatment time points
Time Frame
Up to 36 Weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
HOPE Research Institute
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85018
Country
United States
Facility Name
Movement Disorders Center of Arizona
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85258-4581
Country
United States
Facility Name
The Parkinsons and Movement Disorder Institute
City
Fountain Valley
State/Province
California
ZIP/Postal Code
92708
Country
United States
Facility Name
University of California, Irvine
City
Irvine
State/Province
California
ZIP/Postal Code
92697
Country
United States
Facility Name
Loma Linda University
City
Loma Linda
State/Province
California
ZIP/Postal Code
92354
Country
United States
Facility Name
USC Keck School of Medicine
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Care Access Research
City
Pasadena
State/Province
California
ZIP/Postal Code
91101
Country
United States
Facility Name
Sutter Institute for Medical Research
City
Sacramento
State/Province
California
ZIP/Postal Code
95816
Country
United States
Facility Name
Rocky Mountain Movement Disorders Center
City
Englewood
State/Province
Colorado
ZIP/Postal Code
80113
Country
United States
Facility Name
Associated Neurologist, P.C.
City
Danbury
State/Province
Connecticut
ZIP/Postal Code
06810
Country
United States
Facility Name
New England Institute for Clinical Research
City
Stamford
State/Province
Connecticut
ZIP/Postal Code
06905
Country
United States
Facility Name
MedStar Georgetown University Hospital
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
Facility Name
Parkinson's Disease and Movement Disorders Center of Boca Raton
City
Boca Raton
State/Province
Florida
ZIP/Postal Code
33486
Country
United States
Facility Name
Design Neuroscience Center
City
Doral
State/Province
Florida
ZIP/Postal Code
33172
Country
United States
Facility Name
University of Florida Center for Movement Disorders and Neurorestoration
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32607
Country
United States
Facility Name
Infinity Clinical Research
City
Hollywood
State/Province
Florida
ZIP/Postal Code
33024
Country
United States
Facility Name
University of Florida Health Science Center Jacksonville
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32209
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Suncoast Neuroscience Associates
City
Saint Petersburg
State/Province
Florida
ZIP/Postal Code
33713-8844
Country
United States
Facility Name
USF Parkinson's Disease and Movement Disorders Center
City
Tampa
State/Province
Florida
ZIP/Postal Code
33613
Country
United States
Facility Name
Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Kansas Institute of Reseach
City
Overland Park
State/Province
Kansas
ZIP/Postal Code
66211-1358
Country
United States
Facility Name
Michigan State University
City
East Lansing
State/Province
Michigan
ZIP/Postal Code
48824
Country
United States
Facility Name
QUEST Research Institute
City
Farmington
State/Province
Michigan
ZIP/Postal Code
48334
Country
United States
Facility Name
Henry Ford West Bloomfield Hospital
City
West Bloomfield
State/Province
Michigan
ZIP/Postal Code
48322
Country
United States
Facility Name
St Louis University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63105
Country
United States
Facility Name
Washington University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
University of Nebraska Medical Center
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68198
Country
United States
Facility Name
Mount Sinai Movement Disorders Center
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
University of Rochester
City
Rochester
State/Province
New York
ZIP/Postal Code
14618
Country
United States
Facility Name
Duke University
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Wake Forest Health Sciences
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Thomas Jefferson University
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Facility Name
University of Pennsylvania, Department of Neurology
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Coastal Neurology
City
Port Royal
State/Province
South Carolina
ZIP/Postal Code
29935
Country
United States
Facility Name
Wesley Neurology Clinic
City
Cordova
State/Province
Tennessee
ZIP/Postal Code
38018
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Texas Neurology. P.A.
City
Dallas
State/Province
Texas
ZIP/Postal Code
37232
Country
United States
Facility Name
Baylor College of Medicine
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Houston Methodist Neurological Institute
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Central Texas Neurology Consultants
City
Round Rock
State/Province
Texas
ZIP/Postal Code
78681
Country
United States
Facility Name
University of Vermont Medical Center
City
Burlington
State/Province
Vermont
ZIP/Postal Code
05401
Country
United States
Facility Name
Medical University Innsbruck
City
Innsbruck
ZIP/Postal Code
6020
Country
Austria
Facility Name
Universitaetsklinik fuer Neurologie
City
Wien
ZIP/Postal Code
1090
Country
Austria
Facility Name
University Health Network, Toronto Western Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5T2S8
Country
Canada
Facility Name
Fakultni nemocnice Olomouc, Neurologicka klinika
City
Olomouc
ZIP/Postal Code
779 00
Country
Czechia
Facility Name
Fakultní nemocnice Ostrava, Neurologicka klinika
City
Ostrava-Poruba
ZIP/Postal Code
70852
Country
Czechia
Facility Name
Lekarna Pardubicke nemocnice
City
Pardubice
ZIP/Postal Code
53203
Country
Czechia
Facility Name
Neurologicka klinika 1. LF UK a VFN v Praze
City
Praha
ZIP/Postal Code
12821
Country
Czechia
Facility Name
Vestra Clinics s.r.o.
City
Rychnov Nad Kněžnou
ZIP/Postal Code
51601
Country
Czechia
Facility Name
Hôpital Neurologique Pierre Wertheimer
City
Bron
ZIP/Postal Code
69500
Country
France
Facility Name
CHU Grenoble Alpes
City
Grenoble cedex 09
ZIP/Postal Code
38043
Country
France
Facility Name
Hôpital Roger Salengro - CHRU de Lille
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
CHU Caremeau, Service de Neurologie
City
Nimes cedex 09
ZIP/Postal Code
30029
Country
France
Facility Name
Praxis fuer Neurologie im Bismark Karrée
City
Berlin
ZIP/Postal Code
10627
Country
Germany
Facility Name
Universitaetsklinikum Duesseldorf
City
Duesseldorf
ZIP/Postal Code
40225
Country
Germany
Facility Name
Medizinische Hochschule Hannover
City
Hannover
ZIP/Postal Code
30625
Country
Germany
Facility Name
Klinikum rechts der Isar der TUM
City
Muenchen
ZIP/Postal Code
81675
Country
Germany
Facility Name
GFO Kliniken Troisdorf, Betriebsstätte St. Johannes Sieglar
City
Troisdorf
ZIP/Postal Code
53844
Country
Germany
Facility Name
Universitätsklinikum Tübingen
City
Tübingen
ZIP/Postal Code
72076
Country
Germany
Facility Name
Szpital sw. Wojciecha Podmiot Leczniczy Copernicus Sp. Z o.o.
City
Gdansk
ZIP/Postal Code
80462
Country
Poland
Facility Name
Marta Dagmara BANACH Marta Banach Specjalistyczny Gabinet Neurologiczny
City
Krakow
ZIP/Postal Code
30539
Country
Poland
Facility Name
Krakowska Akademia Neurologii
City
Kraków
ZIP/Postal Code
31505
Country
Poland
Facility Name
Wojewodzki Szpital Specjalistyczny w Olsztynie
City
Olsztyn
ZIP/Postal Code
10-561
Country
Poland
Facility Name
Centrum Medyczne Pratia Warszawa
City
Warsaw
ZIP/Postal Code
01868
Country
Poland
Facility Name
Mazowiecki Szpital Brodnowski Sp. z o.o.
City
Warszaw
ZIP/Postal Code
03-242
Country
Poland
Facility Name
Hospital Universitari Vall d'Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Hospital de la Santa Creu i Sant Pau
City
Barcelona
ZIP/Postal Code
08041
Country
Spain
Facility Name
Hospital Universitario Burgos
City
Burgos
ZIP/Postal Code
09006
Country
Spain
Facility Name
Hospital Universitario de La Princesa
City
Madrid
ZIP/Postal Code
28006
Country
Spain
Facility Name
Royal Devon and Exeter Foundation Trust Hospital
City
Exeter
ZIP/Postal Code
EX25DW
Country
United Kingdom
Facility Name
The Walton Centre NHS Foundation Trust
City
Liverpool
ZIP/Postal Code
L97LJ
Country
United Kingdom
Facility Name
Salford Royal NHS Foundation Trust
City
Salford
ZIP/Postal Code
M55AP
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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