Long-Term Safety and Efficacy of Repeat Treatments of DaxibotulinumtoxinA for Injection in Adults With Isolated Cervical Dystonia (ASPEN-OLS)

NCT03617367 | Completed Phase 3 DMC FDA Drug

• 1 other identifier: 1720304
• Study Type: interventional
• Target: 357
• Locations: 9 countries
• Sites: 65

Brief Summary

Phase 3, open-label, multi-center trial to evaluate the long-term safety, efficacy, and immunogenicity of up to four continuous treatment cycles of daxibotulinumtoxinA (DAXI) for injection.

Conditions

Cervical Dystonia

Keywords

DAXI; Cervical Dystonia; TWSTRS; Toxin; multiple treatment

Outcome Measures

Primary Outcomes (1)

• Long Term Safety of patients determined by the incidence of treatment-emergent adverse events

  Evaluation of adverse events and serious adverse events, from multiple continuous treatments of DAXI for injection, over the course of the study.

  Up to 52 Weeks

Secondary Outcomes (2)

• The proportion of subjects with at least moderate improvement on the Clinician Global Impression of Change (CGIC) at Weeks 4 or 6 for Treatment Cycles 1, 2, 3, and 4

  Weeks 4 and 6 of treatment cycles 1, 2, 3, and 4 (12 - 52 weeks duration each)

• The average of the change from Baseline in the TWSTRS Total Score at Weeks 4 and 6 for Treatment Cycles 1, 2, 3, and 4 [Time Frame: Weeks 4 and 6 of treatment cycles 1, 2, 3, and 4 (12 - 52 weeks duration each)]

  Weeks 4 and 6 of treatment cycles 1, 2, 3, and 4 (12 - 52 weeks duration each)

Study Arms (1)

daxibotulinumtoxinA (DAXI) for injection

EXPERIMENTAL

DAXI for injection

Biological: daxibotulinumtoxinA for injection

Interventions

daxibotulinumtoxinA for injection BIOLOGICAL

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.

daxibotulinumtoxinA (DAXI) for injection

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 (minimum TWSTRS subscale criteria applicable only to subjects not previously enrolled in Study Protocol 1720302)
• Subjects who were previously enrolled in Study Protocol 1720302, and completed the study, including:
• Those with no reduction or have an increase from baseline in the average TWSTRS-total score at Weeks 4 and 6 (i.e., no improvement or worsened disease status), and the investigator agreed that there was a need for retreatment based on the subject's symptoms and neurologic exam findings
• Those who benefited from study treatment and completed follow-up study visits up to the time point of when their TWSTRS - total score reached/exceeded their target TWSTRS score
• Those who benefited from study treatment but subsequently experienced significant recurrence of CD symptoms (e.g. pain) during the study before their TWSTRS-total score reached their target TWSTRS score and requested retreatment, which the investigator determined was warranted based on the subject's symptoms and neurologic exam findings
• Those who completed study visits up to Week 36 and their TWSTRS-total score never reached their target TWSTRS score and they never requested another treatment. The investigator determined that these subjects can be followed in the OLS until their TWSTRS-total score is the same or higher than their target TWSTRS score or until they request retreatment, which the investigator determined is clinically indicated
• De novo subjects (not previously enrolled in Study Protocol 1720302):
• Naïve to BoNT treatment
• BoNT treatment-experienced; if previously treated with BoNTA, the subject must have demonstrated a clinically meaningful response to the last BoNTA treatment based on the clinical judgment of the investigator

You may not qualify if:

• 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 botulinum toxin (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 (applicable only to de novo subjects)
• Botulinum neurotoxin 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 (rimabotulinumtoxinA \[Myobloc/Neurobloc\]) injection for CD due to non-response or suboptimal response to BoNTA (applicable only to de novo subjects)

Sponsors & Collaborators

• Revance Therapeutics, Inc.: lead
• Syneos Health: collaborator

Study Sites (65)

HOPE Research Institute

Phoenix, Arizona, 85018, United States

Location

Movement Disorders Center of Arizona

Scottsdale, Arizona, 85258, United States

Location

The Parkinsons and Movement Disorder Institute

Fountain Valley, California, 92708, United States

Location

Loma Linda University

Loma Linda, California, 92354, United States

Location

USC Keck School of Medicine

Los Angeles, California, 90033, United States

Location

Care Access Research LLC

Pasadena, California, 91101, United States

Location

Rocky Mountain Movement Disorders Center

Englewood, Colorado, 80113, United States

Location

Ki Health Partners LLC DBA New England Institute for Clinical Research

Stamford, Connecticut, 06905, United States

Location

Parkinson's Disease and Movement Disorders Center of Boca Raton

Boca Raton, Florida, 33486, United States

Location

University of Florida Center for Movement Disorders and Neurorestoration

Gainesville, Florida, 32609, United States

Location

Infinity Clinical research

Hollywood, Florida, 33024, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Suncoast Neuroscience Associates

St. Petersburg, Florida, 33713, United States

Location

USF Parkinson's Disease and Movement Disorders Center

Tampa, Florida, 33613, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Kansas Institute of Research

Overland Park, Kansas, 66211-1358, United States

Location

Michigan State University

East Lansing, Michigan, 48824, United States

Location

QUEST Research Institute

Farmington, Michigan, 48334, United States

Location

Henry Ford West Bloomfield Hospital

West Bloomfield, Michigan, 48322, United States

Location

St Louis University

St Louis, Missouri, 63104, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68105, United States

Location

Mount Sinai Movement Disorders Center

New York, New York, 10029, United States

Location

University of Rochester

Rochester, New York, 14618, United States

Location

Duke University

Durham, North Carolina, 27710, United States

Location

Wake Forest Health Sciences

Winston-Salem, North Carolina, 27157, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19107, United States

Location

Coastal Neurology

Port Royal, South Carolina, 29935, United States

Location

Intrafusion Research Network - Wesley Neurology Clinic

Cordova, Tennessee, 38018, United States

Location

Veracity Neuroscience LLC

Memphis, Tennessee, 38157, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Texas Neurology. P.A.

Dallas, Texas, 75214, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Houston Methodist Neurological Institute

Houston, Texas, 77030, United States

Location

Central Texas Neurology Consultants

Round Rock, Texas, 78681, United States

Location

The University of Vermont Medical Center

Burlington, Vermont, 05401, United States

Location

Neurologisches Studienzentrum Universitätsklinik für Neurologie Innsbruck

Innsbruck, 30539, Austria

Location

University Health Network, Toronto Western Hospital

Toronto, Ontario, M5T 2S8, Canada

Location

Fakultní nemocnice Ostrava

Ostrava-Poruba, 70852, Czechia

Location

Nemocnice Pardubickeho kraje, a.s.; Pardubicka nemocnice

Pardubice, 53203, Czechia

Location

Neurologicka klinika 1. LF UK a VFN v Praze

Prague, 12821, Czechia

Location

Vestra Clinics s.r.o.

Rychnov nad Kněžnou, 51601, Czechia

Location

Hôpital Neurologique Pierre Wertheimer

Bron, 69500, France

Location

CHU de Grenoble

Grenoble, 38043, France

Location

Hôpital Roger Salengro

Lille, 59037, France

Location

CHU Caremeau

Nîmes, 30029, France

Location

Universitaetsklinikum Duesseldorf

Düsseldorf, 40225, Germany

Location

Medizinische Hochschule Hannover

Hanover, 30625, Germany

Location

Klinikum rechts der Isar der TUM

München, 81675, Germany

Location

GFO Kliniken Troisdorf, Betriebsstätte St. Johannes Sieglar

Troisdorf, 53844, Germany

Location

Universitätsklinikum Tübingen

Tübingen, 72076, Germany

Location

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

Gdansk, 80462, Poland

Location

Marta Dagmara BANACH Marta Banach Specjalistyczny Gabinet Neurologiczny

Krakow, 30539, Poland

Location

Krakowska Akademia Neurologii Sp. z o.o.

Krakow, 31505, Poland

Location

Wojewodzki Szpital Specjalistyczny w Olsztynie

Olsztyn, 10561, Poland

Location

Centrum Medyczne Pratia Warszawa

Warsaw, 01868, Poland

Location

Mazovian Brodno Hospital

Warsaw, 03242, Poland

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital de la Santa Creu i Sant Pau

Barcelona, 08041, Spain

Location

Hospital Universitario Burgos

Burgos, 09006, Spain

Location

Hospital Universitario de La Princesa

Madrid, 28006, Spain

Location

Royal Devon and Exeter Foundation Trust Hospital

Exeter, EX2 5DW, United Kingdom

Location

The Walton Centre NHS Foundation Trust, Neuroscience Research Centre

Liverpool, L97LJ, United Kingdom

Location

Salford Royal NHS Foundation Trust

Salford, M55AP, United Kingdom

Location

Related Publications (1)

• McAllister P, Patel AT, Banach M, Ellenbogen A, Slawek J, Paus S, Jinnah HA, Evidente V, Gross TM, Kazerooni R, Gallagher CJ, Hollander DA. Long-Term Safety and Efficacy of Repeat Treatments with DaxibotulinumtoxinA in Cervical Dystonia: Results from the ASPEN-Open-Label Study. Mov Disord Clin Pract. 2025 Nov;12(11):1764-1773. doi: 10.1002/mdc3.70104. Epub 2025 May 29.

  PMID: 40439027 DERIVED

MeSH Terms

Conditions

Torticollis

Interventions

Botulinum Toxins, Type A; Injections

Condition Hierarchy (Ancestors)

Dystonia; Dyskinesias; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Botulinum Toxins; Metalloendopeptidases; Endopeptidases; Peptide Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Metalloproteases; Bacterial Proteins; Proteins; Amino Acids, Peptides, and Proteins; Bacterial Toxins; Toxins, Biological; Biological Factors; Drug Administration Routes; Drug Therapy; Therapeutics

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Masking Details: not applicable (open label)
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a Phase 3, open-label, multi-center trial to evaluate the long-term safety, efficacy, and immunogenicity of up to four continuous treatment cycles of daxibotulinumtoxinA (DAXI) for injection with up to four different doses in adults with isolated cervical dystonia (CD).

Study Record Dates

• First Submitted: July 12, 2018
• First Posted: August 6, 2018
• Study Start: September 5, 2018
• Primary Completion: May 25, 2021
• Study Completion: May 25, 2021
• Last Updated: January 28, 2022

Record last verified: 2022-01

Data Sharing

• IPD Sharing: Will not share

Locations

CZ (4); ES (4); CA (1); DE (5); US (37); GB (3); FR (4); PL (6); AU (1)