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| Name | Class |
|---|---|
| Syneos Health | OTHER |
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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).
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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| DAXI for injection low dose | Experimental | Low Dose Group |
|
| DAXI for injection high dose | Experimental | High Dose Group |
|
| Placebo | Placebo Comparator | Placebo Group |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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). | Week 4 and Week 6 |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline TWSTRS-total score | Change from baseline in TWSTRS-total score (all post-treatment time points) | Up to 36 Weeks |
| Duration of effect | Duration of effect based on target TWSTRS score |
| Measure | Description | Time Frame |
|---|---|---|
| Change in TWSTRS subscale scores | Change from baseline in TWSTRS subscale scores (TWSTRS-Severity, TWSTRS-Disability, and TWSTRS-Pain) (all post-treatment time points) | Up to 36 Weeks |
| Clinical Global Impression of Change (CGIC) |
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| HOPE Research Institute | Phoenix | Arizona | 85018 | United States | ||
| Movement Disorders Center of Arizona |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38295339 | Derived | 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. |
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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.
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Double Blinded
|
| Placebo | Biological | 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. |
|
| Up to 36 Weeks |
| Patient Global Impression of Change (PGIC) Improvement | Percentage responders at Week 4 or 6 | Week 4 or Week 6 |
| Incidence of treatment-emergent adverse events (Safety) | Evaluation of adverse events and serious adverse events over the course of the study. | Up to 36 Weeks |
CGIC at all post-treatment time points
| Up to 36 Weeks |
| Patient Global Impression of Change (PGIC) | PGIC at all post-treatment time points | Up to 36 Weeks |
| Scottsdale |
| Arizona |
| 85258-4581 |
| United States |
| The Parkinsons and Movement Disorder Institute | Fountain Valley | California | 92708 | United States |
| University of California, Irvine | Irvine | California | 92697 | United States |
| Loma Linda University | Loma Linda | California | 92354 | United States |
| USC Keck School of Medicine | Los Angeles | California | 90033 | United States |
| Care Access Research | Pasadena | California | 91101 | United States |
| Sutter Institute for Medical Research | Sacramento | California | 95816 | United States |
| Rocky Mountain Movement Disorders Center | Englewood | Colorado | 80113 | United States |
| Associated Neurologist, P.C. | Danbury | Connecticut | 06810 | United States |
| New England Institute for Clinical Research | Stamford | Connecticut | 06905 | United States |
| MedStar Georgetown University Hospital | Washington D.C. | District of Columbia | 20007 | United States |
| Parkinson's Disease and Movement Disorders Center of Boca Raton | Boca Raton | Florida | 33486 | United States |
| Design Neuroscience Center | Doral | Florida | 33172 | United States |
| University of Florida Center for Movement Disorders and Neurorestoration | Gainesville | Florida | 32607 | United States |
| Infinity Clinical Research | Hollywood | Florida | 33024 | United States |
| University of Florida Health Science Center Jacksonville | Jacksonville | Florida | 32209 | United States |
| University of Miami | Miami | Florida | 33136 | United States |
| Suncoast Neuroscience Associates | St. Petersburg | Florida | 33713-8844 | United States |
| USF Parkinson's Disease and Movement Disorders Center | Tampa | Florida | 33613 | United States |
| Emory University | Atlanta | Georgia | 30329 | United States |
| Rush University Medical Center | Chicago | Illinois | 60612 | United States |
| Kansas Institute of Reseach | Overland Park | Kansas | 66211-1358 | United States |
| Michigan State University | East Lansing | Michigan | 48824 | United States |
| QUEST Research Institute | Farmington | Michigan | 48334 | United States |
| Henry Ford West Bloomfield Hospital | West Bloomfield | Michigan | 48322 | United States |
| St Louis University | St Louis | Missouri | 63105 | United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| University of Nebraska Medical Center | Omaha | Nebraska | 68198 | United States |
| Mount Sinai Movement Disorders Center | New York | New York | 10029 | United States |
| University of Rochester | Rochester | New York | 14618 | United States |
| Duke University | Durham | North Carolina | 27710 | United States |
| Wake Forest Health Sciences | Winston-Salem | North Carolina | 27157 | United States |
| Thomas Jefferson University | Philadelphia | Pennsylvania | 19107 | United States |
| University of Pennsylvania, Department of Neurology | Philadelphia | Pennsylvania | 19107 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Coastal Neurology | Port Royal | South Carolina | 29935 | United States |
| Wesley Neurology Clinic | Cordova | Tennessee | 38018 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| Texas Neurology. P.A. | Dallas | Texas | 37232 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| Houston Methodist Neurological Institute | Houston | Texas | 77030 | United States |
| Central Texas Neurology Consultants | Round Rock | Texas | 78681 | United States |
| University of Vermont Medical Center | Burlington | Vermont | 05401 | United States |
| Medical University Innsbruck | Innsbruck | 6020 | Austria |
| Universitaetsklinik fuer Neurologie | Vienna | 1090 | Austria |
| University Health Network, Toronto Western Hospital | Toronto | Ontario | M5T2S8 | Canada |
| Fakultni nemocnice Olomouc, Neurologicka klinika | Olomouc | 779 00 | Czechia |
| Fakultní nemocnice Ostrava, Neurologicka klinika | Ostrava-Poruba | 70852 | Czechia |
| Lekarna Pardubicke nemocnice | Pardubice | 53203 | Czechia |
| Neurologicka klinika 1. LF UK a VFN v Praze | Prague | 12821 | Czechia |
| Vestra Clinics s.r.o. | Rychnov nad Kněžnou | 51601 | Czechia |
| Hôpital Neurologique Pierre Wertheimer | Bron | 69500 | France |
| CHU Grenoble Alpes | Grenoble | 38043 | France |
| Hôpital Roger Salengro - CHRU de Lille | Lille | 59037 | France |
| CHU Caremeau, Service de Neurologie | Nîmes | 30029 | France |
| Praxis fuer Neurologie im Bismark Karrée | Berlin | 10627 | Germany |
| Universitaetsklinikum Duesseldorf | Düsseldorf | 40225 | Germany |
| Medizinische Hochschule Hannover | Hanover | 30625 | Germany |
| Klinikum rechts der Isar der TUM | München | 81675 | Germany |
| GFO Kliniken Troisdorf, Betriebsstätte St. Johannes Sieglar | Troisdorf | 53844 | Germany |
| Universitätsklinikum Tübingen | Tübingen | 72076 | Germany |
| Szpital sw. Wojciecha Podmiot Leczniczy Copernicus Sp. Z o.o. | Gdansk | 80462 | Poland |
| Marta Dagmara BANACH Marta Banach Specjalistyczny Gabinet Neurologiczny | Krakow | 30539 | Poland |
| Krakowska Akademia Neurologii | Krakow | 31505 | Poland |
| Wojewodzki Szpital Specjalistyczny w Olsztynie | Olsztyn | 10-561 | Poland |
| Centrum Medyczne Pratia Warszawa | Warsaw | 01868 | Poland |
| Mazowiecki Szpital Brodnowski Sp. z o.o. | Warsaw | 03-242 | Poland |
| Hospital Universitari Vall d'Hebron | Barcelona | 08035 | Spain |
| Hospital de la Santa Creu i Sant Pau | Barcelona | 08041 | Spain |
| Hospital Universitario Burgos | Burgos | 09006 | Spain |
| Hospital Universitario de La Princesa | Madrid | 28006 | Spain |
| Royal Devon and Exeter Foundation Trust Hospital | Exeter | EX25DW | United Kingdom |
| The Walton Centre NHS Foundation Trust | Liverpool | L97LJ | United Kingdom |
| Salford Royal NHS Foundation Trust | Salford | M55AP | United Kingdom |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Apr 13, 2023 | May 5, 2023 | 10 | ||
| May 30, 2023 | May 31, 2023 | 11 | ||
| Jun 21, 2023 | Jun 22, 2023 | 12 |
| ID | Term |
|---|---|
| D014103 | Torticollis |
| ID | Term |
|---|---|
| D004421 | Dystonia |
| D020820 | Dyskinesias |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| D019274 | Botulinum Toxins, Type A |
| D007267 | Injections |
| ID | Term |
|---|---|
| D001905 | Botulinum Toxins |
| D008666 | Metalloendopeptidases |
| D010450 | Endopeptidases |
| D010447 | Peptide Hydrolases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
| D045762 | Enzymes and Coenzymes |
| D045726 | Metalloproteases |
| D001426 | Bacterial Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D001427 | Bacterial Toxins |
| D014118 | Toxins, Biological |
| D001685 | Biological Factors |
| D004333 | Drug Administration Routes |
| D004358 | Drug Therapy |
| D013812 | Therapeutics |
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