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

NCT ID: NCT03617367

Last Updated: 2022-01-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 357 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-09-05
• Study Completion Date: 2021-05-25

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.

Detailed Description

Approximately 350 adult subjects will be recruited from approximately 80 study centers in the United States, Canada, and Europe who were enrolled in the ASPEN-1 Study Protocol 1720302 and de novo subjects (not previously enrolled in ASPEN-1 Study Protocol 1720302) will be treated with up to 4 different doses of daxibotulinumtoxinA for injection.

Conditions

Cervical Dystonia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

daxibotulinumtoxinA (DAXI) for injection

DAXI for injection

Group Type: EXPERIMENTAL

daxibotulinumtoxinA for injection

Intervention Type: 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.

Interventions

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.

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• 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

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 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)

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Syneos Health

OTHER

Sponsor Role: collaborator

Revance Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

HOPE Research Institute

Phoenix, Arizona, United States

Movement Disorders Center of Arizona

Scottsdale, Arizona, United States

The Parkinsons and Movement Disorder Institute

Fountain Valley, California, United States

Loma Linda University

Loma Linda, California, United States

USC Keck School of Medicine

Los Angeles, California, United States

Care Access Research LLC

Pasadena, California, United States

Rocky Mountain Movement Disorders Center

Englewood, Colorado, United States

Ki Health Partners LLC DBA New England Institute for Clinical Research

Stamford, Connecticut, United States

Parkinson's Disease and Movement Disorders Center of Boca Raton

Boca Raton, Florida, United States

University of Florida Center for Movement Disorders and Neurorestoration

Gainesville, Florida, United States

Infinity Clinical research

Hollywood, Florida, United States

University of Miami

Miami, Florida, United States

Suncoast Neuroscience Associates

St. Petersburg, Florida, United States

USF Parkinson's Disease and Movement Disorders Center

Tampa, Florida, United States

Emory University

Atlanta, Georgia, United States

Rush University Medical Center

Chicago, Illinois, United States

Kansas Institute of Research

Overland Park, Kansas, United States

Michigan State University

East Lansing, Michigan, United States

QUEST Research Institute

Farmington, Michigan, United States

Henry Ford West Bloomfield Hospital

West Bloomfield, Michigan, United States

St Louis University

St Louis, Missouri, United States

Washington University

St Louis, Missouri, United States

University of Nebraska Medical Center

Omaha, Nebraska, United States

Mount Sinai Movement Disorders Center

New York, New York, United States

University of Rochester

Rochester, New York, United States

Duke University

Durham, North Carolina, United States

Wake Forest Health Sciences

Winston-Salem, North Carolina, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Coastal Neurology

Port Royal, South Carolina, United States

Intrafusion Research Network - Wesley Neurology Clinic

Cordova, Tennessee, United States

Veracity Neuroscience LLC

Memphis, Tennessee, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Texas Neurology. P.A.

Dallas, Texas, United States

Baylor College of Medicine

Houston, Texas, United States

Houston Methodist Neurological Institute

Houston, Texas, United States

Central Texas Neurology Consultants

Round Rock, Texas, United States

The University of Vermont Medical Center

Burlington, Vermont, United States

Neurologisches Studienzentrum Universitätsklinik für Neurologie Innsbruck

Innsbruck, , Austria

University Health Network, Toronto Western Hospital

Toronto, Ontario, Canada

Fakultní nemocnice Ostrava

Ostrava-Poruba, , Czechia

Nemocnice Pardubickeho kraje, a.s.; Pardubicka nemocnice

Pardubice, , Czechia

Neurologicka klinika 1. LF UK a VFN v Praze

Prague, , Czechia

Vestra Clinics s.r.o.

Rychnov nad Kněžnou, , Czechia

Hôpital Neurologique Pierre Wertheimer

Bron, , France

CHU de Grenoble

Grenoble, , France

Hôpital Roger Salengro

Lille, , France

CHU Caremeau

Nîmes, , France

Universitaetsklinikum Duesseldorf

Düsseldorf, , Germany

Medizinische Hochschule Hannover

Hanover, , Germany

Klinikum rechts der Isar der TUM

München, , Germany

GFO Kliniken Troisdorf, Betriebsstätte St. Johannes Sieglar

Troisdorf, , Germany

Universitätsklinikum Tübingen

Tübingen, , Germany

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

Gdansk, , Poland

Marta Dagmara BANACH Marta Banach Specjalistyczny Gabinet Neurologiczny

Krakow, , Poland

Krakowska Akademia Neurologii Sp. z o.o.

Krakow, , Poland

Wojewodzki Szpital Specjalistyczny w Olsztynie

Olsztyn, , Poland

Centrum Medyczne Pratia Warszawa

Warsaw, , Poland

Mazovian Brodno Hospital

Warsaw, , Poland

Hospital Universitari Vall d'Hebron

Barcelona, , Spain

Hospital de la Santa Creu i Sant Pau

Barcelona, , Spain

Hospital Universitario Burgos

Burgos, , Spain

Hospital Universitario de La Princesa

Madrid, , Spain

Royal Devon and Exeter Foundation Trust Hospital

Exeter, , United Kingdom

The Walton Centre NHS Foundation Trust, Neuroscience Research Centre

Liverpool, , United Kingdom

Salford Royal NHS Foundation Trust

Salford, , United Kingdom

Countries

United States; Austria; Canada; Czechia; France; Germany; Poland; Spain; United Kingdom

References

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 May 29. doi: 10.1002/mdc3.70104. Online ahead of print.

Reference Type: DERIVED

PMID: 40439027 (View on PubMed)

Other Identifiers

1720304

Identifier Type: -

Identifier Source: org_study_id