AzTrials
AzTrials

Cervical DystoniaPhase 3ASPEN-1NCT03608397

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

A Phase 3 study testing DaxibotulinumtoxinA for injection in people with Cervical Dystonia, run by Revance Therapeutics, Inc. at 75 sites in 9 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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). Read more about this study

Who is it for?
Ages 18 to 80 years
Conditions the study is for
Cervical Dystonia
What is being tested?
DaxibotulinumtoxinA for injection
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
301 participants
Where
75 sitesin 9 countries
Study dates
20 Jun 2018to 16 Jun 2020
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

75 sites in 9 countries.

75 sites in 9 countries

United States44 sites

  • Emory University

    Atlanta, Georgia, 30329

  • Parkinson's Disease and Movement Disorders Center of Boca Raton

    Boca Raton, Florida, 33486

  • University of Vermont Medical Center

    Burlington, Vermont, 05401

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

  • Rush University Medical Center

    Chicago, Illinois, 60612

  • Wesley Neurology Clinic

    Cordova, Tennessee, 38018

  • Texas Neurology. P.A.

    Dallas, Texas, 37232

  • Associated Neurologist, P.C.

    Danbury, Connecticut, 06810

  • Design Neuroscience Center

    Doral, Florida, 33172

  • Duke University

    Durham, North Carolina, 27710

  • Michigan State University

    East Lansing, Michigan, 48824

  • Rocky Mountain Movement Disorders Center

    Englewood, Colorado, 80113

  • QUEST Research Institute

    Farmington, Michigan, 48334

  • The Parkinsons and Movement Disorder Institute

    Fountain Valley, California, 92708

  • University of Florida Center for Movement Disorders and Neurorestoration

    Gainesville, Florida, 32607

  • Infinity Clinical Research

    Hollywood, Florida, 33024

  • Baylor College of Medicine

    Houston, Texas, 77030

  • Houston Methodist Neurological Institute

    Houston, Texas, 77030

  • University of California, Irvine

    Irvine, California, 92697

  • University of Florida Health Science Center Jacksonville

    Jacksonville, Florida, 32209

  • Loma Linda University

    Loma Linda, California, 92354

  • USC Keck School of Medicine

    Los Angeles, California, 90033

  • University of Miami

    Miami, Florida, 33136

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Mount Sinai Movement Disorders Center

    New York, New York, 10029

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198

  • Kansas Institute of Reseach

    Overland Park, Kansas, 66211-1358

  • Care Access Research

    Pasadena, California, 91101

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19107

  • University of Pennsylvania, Department of Neurology

    Philadelphia, Pennsylvania, 19107

  • HOPE Research Institute

    Phoenix, Arizona, 85018

  • Coastal Neurology

    Port Royal, South Carolina, 29935

  • University of Rochester

    Rochester, New York, 14618

  • Central Texas Neurology Consultants

    Round Rock, Texas, 78681

  • Sutter Institute for Medical Research

    Sacramento, California, 95816

  • Movement Disorders Center of Arizona

    Scottsdale, Arizona, 85258-4581

  • St Louis University

    St Louis, Missouri, 63105

  • Washington University

    St Louis, Missouri, 63110

  • Suncoast Neuroscience Associates

    St. Petersburg, Florida, 33713-8844

  • New England Institute for Clinical Research

    Stamford, Connecticut, 06905

  • USF Parkinson's Disease and Movement Disorders Center

    Tampa, Florida, 33613

  • MedStar Georgetown University Hospital

    Washington D.C., District of Columbia, 20007

  • Henry Ford West Bloomfield Hospital

    West Bloomfield, Michigan, 48322

  • Wake Forest Health Sciences

    Winston-Salem, North Carolina, 27157

Other trials in United States

Germany6 sites

  • Praxis fuer Neurologie im Bismark Karrée

    Berlin, 10627

  • Universitaetsklinikum Duesseldorf

    Düsseldorf, 40225

  • Medizinische Hochschule Hannover

    Hanover, 30625

  • Klinikum rechts der Isar der TUM

    München, 81675

  • GFO Kliniken Troisdorf, Betriebsstätte St. Johannes Sieglar

    Troisdorf, 53844

  • Universitätsklinikum Tübingen

    Tübingen, 72076

Other trials in Germany

Poland6 sites

  • Szpital sw. Wojciecha Podmiot Leczniczy Copernicus Sp. Z o.o.

    Gdansk, 80462

  • Marta Dagmara BANACH Marta Banach Specjalistyczny Gabinet Neurologiczny

    Krakow, 30539

  • Krakowska Akademia Neurologii

    Krakow, 31505

  • Wojewodzki Szpital Specjalistyczny w Olsztynie

    Olsztyn, 10-561

  • Centrum Medyczne Pratia Warszawa

    Warsaw, 01868

  • Mazowiecki Szpital Brodnowski Sp. z o.o.

    Warsaw, 03-242

Other trials in Poland

Czechia5 sites

  • Fakultni nemocnice Olomouc, Neurologicka klinika

    Olomouc, 779 00

  • Fakultní nemocnice Ostrava, Neurologicka klinika

    Ostrava-Poruba, 70852

  • Lekarna Pardubicke nemocnice

    Pardubice, 53203

  • Neurologicka klinika 1. LF UK a VFN v Praze

    Prague, 12821

  • Vestra Clinics s.r.o.

    Rychnov nad Kněžnou, 51601

Other trials in Czechia

France4 sites

  • Hôpital Neurologique Pierre Wertheimer

    Bron, 69500

  • CHU Grenoble Alpes

    Grenoble, 38043

  • Hôpital Roger Salengro - CHRU de Lille

    Lille, 59037

  • CHU Caremeau, Service de Neurologie

    Nîmes, 30029

Other trials in France

Spain4 sites

  • Hospital Universitari Vall d'Hebron

    Barcelona, 08035

  • Hospital de la Santa Creu i Sant Pau

    Barcelona, 08041

  • Hospital Universitario Burgos

    Burgos, 09006

  • Hospital Universitario de La Princesa

    Madrid, 28006

Other trials in Spain

United Kingdom3 sites

  • Royal Devon and Exeter Foundation Trust Hospital

    Exeter, EX25DW

  • The Walton Centre NHS Foundation Trust

    Liverpool, L97LJ

  • Salford Royal NHS Foundation Trust

    Salford, M55AP

Other trials in United Kingdom

Austria2 sites

  • Medical University Innsbruck

    Innsbruck, 6020

  • Universitaetsklinik fuer Neurologie

    Vienna, 1090

Other trials in Austria

Canada1 site

  • University Health Network, Toronto Western Hospital

    Toronto, Ontario, M5T2S8

Other trials in Canada

About this study

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)

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).

Read the 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).

Being tested
DaxibotulinumtoxinA for injection, Placebo
Keywords
DAXI, Cervical Dystonia, TWSTRS, Toxin
Medical subject headings
Torticollis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • DaxibotulinumtoxinA for injectionBiological

    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.

  • PlaceboBiological

    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 groups in NCT03608397
Study groupRoleWhat they receive
DAXI for injection low doseExperimentalLow Dose Group
Receives: Biological: DaxibotulinumtoxinA for injection
DAXI for injection high doseExperimentalHigh Dose Group
Receives: Biological: DaxibotulinumtoxinA for injection
PlaceboPlacebo comparatorPlacebo Group
Receives: Biological: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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).

    Measured: Week 4 and Week 6

Secondary outcomes (4)

  1. Change from Baseline TWSTRS-total score

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

    Measured: Up to 36 Weeks

  2. Duration of effect

    Duration of effect based on target TWSTRS score

    Measured: Up to 36 Weeks

  3. Patient Global Impression of Change (PGIC) Improvement

    Percentage responders at Week 4 or 6

    Measured: Week 4 or Week 6

  4. Incidence of treatment-emergent adverse events (Safety)

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

    Measured: Up to 36 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Aug 2018
  2. Study start20 Jun 2018
  3. Primary completion3 Dec 2019
  4. Study completion16 Jun 2020

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
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 blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
301 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03608397
Sponsor's protocol ID
1720302
Acronym
ASPEN-1
Status last verified
Nov 2022
First submitted
11 Jul 2018
Last updated
5 Dec 2022
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Comella CL, Jankovic J, Hauser RA, Patel AT, Banach MD, Ehler E, Vitarella D, Rubio RG, Gross TM; ASPEN-1 Study Group. Efficacy and Safety of DaxibotulinumtoxinA for Injection in Cervical Dystonia: ASPEN-1 Phase 3 Randomized Controlled Trial. Neurology. 2024 Feb 27;102(4):e208091. doi: 10.1212/WNL.0000000000208091. Epub 2024 Jan 31.

    PubMed 38295339

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing DaxibotulinumtoxinA for injection in people with Cervical Dystonia, run by Revance Therapeutics, Inc. at 75 sites in 9 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 75 sites in United States, Germany, Poland, Czechia, France and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from Baseline Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-total score (time frame: Week 4 and Week 6). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03608397, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Dec 2022.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in