NCT02706795

Brief Summary

This is an open-label, dose-escalation study to assess the safety and preliminary efficacy of daxibotulinumtoxinA in subjects with isolated cervical dystonia (CD).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
37

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Sep 2015

Geographic Reach
1 country

13 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2015

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

March 8, 2016

Completed
3 days until next milestone

First Posted

Study publicly available on registry

March 11, 2016

Completed
9 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 7, 2016

Completed
7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

July 17, 2017

Completed
Last Updated

November 6, 2019

Status Verified

October 1, 2019

Enrollment Period

1.3 years

First QC Date

March 8, 2016

Last Update Submit

October 30, 2019

Conditions

Torticollis (Spasmodic)

Keywords

Cervical dystoniaSpasmodic torticollis

Outcome Measures

Primary Outcomes (1)

  • Improvement of dystonia as measured by change from baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) - Total Score

    Week 4

Secondary Outcomes (6)

  • Change from baseline in Toronto Western Spasmodic Torticollis Rating Scale(TWSTRS) - Total Score

    Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24

  • Change from baseline in TWSTRS subscale scores (Severity, Disability and Pain)

    Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24

  • Duration of effect based on number of weeks from treatment until return of symptoms that warrant treatment

    Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24

  • Percentage of treatment responders with at least a 20% reduction in the TWSTRS - Total score post-treatment

    Weeks 2, 4, 6, 9, 12, 16, 20, 24

  • Patient-rated quality of life measurement based upon change from baseline of the Cervical Dystonia Impact Profile Scale (CDIP-58)

    Post-treatment: Weeks 4, 6, 12, 16, 20, 24

  • +1 more secondary outcomes

Study Arms (1)

daxibotulinumtoxinA (DAXI) for injection

EXPERIMENTAL

DAXI for injection

Biological: DaxibotulinumtoxinA

Interventions

DaxibotulinumtoxinABIOLOGICAL

Low, mid, and high dose sequential treatments of daxibotulinumtoxinA in dose-escalation safety and efficacy study

daxibotulinumtoxinA (DAXI) for injection

Eligibility Criteria

Age30 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Meets diagnostic criteria for isolated cervical dystonia
  • Has moderate severity with a baseline Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) - Total score of at least 20 and a TWSTRS-Severity subscale score or at least 15
  • Has been using stable doses of focal dystonia medications for at least 3 months and willing to continue through end of study

You may not qualify if:

  • Cervical dystonia attributable to an underlying etiology, such as traumatic torticollis or tardive torticollis; predominant retrocollis or anterocollis cervical dystonia
  • Significant dystonia in other body areas, or is currently being treated with botulinum toxin for dystonia in other areas than isolated cervical dystonia
  • Neurological abnormalities other than cervical dystonia
  • History of severe dysphagia or aspiration, or current clinically significant swallowing disorder
  • Previous neck surgery, phenol injection to the neck muscles, myotomy or denervation surgery in the neck/shoulder region, or intrathecal baclofen
  • Marked limitation on passive range of motion that suggests contractures or other structural abnormality, e.g. cervical contractures or cervical spinal deformity
  • Profound atrophy of cervical musculature
  • Any neurological condition that may place the subject at increased risk with exposure to botulinum toxin type A, including peripheral motor neuropathic diseases such as amyotrophic lateral sclerosis and motor neuropathy, and neuromuscular junctional disorders such as Lambert-Eaton syndrome and myasthenia gravis
  • Suboptimal efficacy response to any prior botulinum toxin type A product, when a previous treatment produced more optimal therapeutic response, as judged subjectively by the Investigator
  • Previous treatment with any botulinum toxin product for any condition within the 6 months prior to Screening and during the study; presence of any blood coagulation disorder; or on anticoagulation treatment with international normalized ratio (INR) \> 3.5

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (13)

The Parkinson's & Movement Disorder Institute

Fountain Valley, California, 92708, United States

Location

Colorado Springs Neurological Associates

Colorado Springs, Colorado, 80907, United States

Location

University of Florida Center for Movement Disorders & Neurorestoration

Gainesville, Florida, 32607, United States

Location

Precision Research Organization

Miami Lakes, Florida, 33016, United States

Location

University of South Florida

Tampa, Florida, 33613, United States

Location

Emory University

Atlanta, Georgia, 30329, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Kansas City Bone & Joint Clinic

Overland Park, Kansas, 66211, United States

Location

The NeuroMedical Center Clinic

Baton Rouge, Louisiana, 70810, United States

Location

Wake forest Baptist Health

Winston-Salem, North Carolina, 27157, United States

Location

Riverhills Healthcare, Inc.

Cincinnati, Ohio, 45212, United States

Location

Coastal Neurology

Port Royal, South Carolina, 29935, United States

Location

Parkinson's Disease Center and Movement Disorder Clinic

Houston, Texas, 77030, United States

Location

Related Publications (1)

  • Jankovic J, Truong D, Patel AT, Brashear A, Evatt M, Rubio RG, Oh CK, Snyder D, Shears G, Comella C. Injectable DaxibotulinumtoxinA in Cervical Dystonia: A Phase 2 Dose-Escalation Multicenter Study. Mov Disord Clin Pract. 2018 Apr 26;5(3):273-282. doi: 10.1002/mdc3.12613. eCollection 2018 May-Jun.

    PMID: 30009213DERIVED

MeSH Terms

Conditions

Torticollis

Interventions

Botulinum Toxins, Type A

Condition Hierarchy (Ancestors)

DystoniaDyskinesiasNeurologic ManifestationsSigns and SymptomsPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Botulinum ToxinsMetalloendopeptidasesEndopeptidasesPeptide HydrolasesHydrolasesEnzymesEnzymes and CoenzymesMetalloproteasesBacterial ProteinsProteinsAmino Acids, Peptides, and ProteinsBacterial ToxinsToxins, BiologicalBiological Factors

Study Officials

  • Nubia Kaba

    Sponsor GmbH

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 8, 2016

First Posted

March 11, 2016

Study Start

September 1, 2015

Primary Completion

December 7, 2016

Study Completion

July 17, 2017

Last Updated

November 6, 2019

Record last verified: 2019-10

Locations

US(13)