Dose-escalating Safety and Preliminary Efficacy of DaxibotulinumtoxinA for Injection in Cervical Dystonia

October 30, 2019 updated by: Revance Therapeutics, Inc.
This is an open-label, dose-escalation study to assess the safety and preliminary efficacy of daxibotulinumtoxinA in subjects with isolated cervical dystonia (CD).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
37

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Fountain Valley, California, United States, 92708
        • The Parkinson's & Movement Disorder Institute
    • Colorado
      • Colorado Springs, Colorado, United States, 80907
        • Colorado Springs Neurological Associates
    • Florida
      • Gainesville, Florida, United States, 32607
        • University of Florida Center for Movement Disorders & Neurorestoration
      • Miami Lakes, Florida, United States, 33016
        • Precision Research Organization
      • Tampa, Florida, United States, 33613
        • University of South Florida
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Emory University
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Rush University Medical Center
    • Kansas
      • Overland Park, Kansas, United States, 66211
        • Kansas City Bone & Joint Clinic
    • Louisiana
      • Baton Rouge, Louisiana, United States, 70810
        • The NeuroMedical Center Clinic
    • North Carolina
      • Winston-Salem, North Carolina, United States, 27157
        • Wake Forest Baptist Health
    • Ohio
      • Cincinnati, Ohio, United States, 45212
        • Riverhills Healthcare, Inc.
    • South Carolina
      • Port Royal, South Carolina, United States, 29935
        • Coastal Neurology
    • Texas
      • Houston, Texas, United States, 77030
        • Parkinson's Disease Center and Movement Disorder Clinic

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

26 years to 71 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: daxibotulinumtoxinA (DAXI) for injection
DAXI for injection
Biological: DaxibotulinumtoxinA
Low, mid, and high dose sequential treatments of daxibotulinumtoxinA in dose-escalation safety and efficacy study

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Improvement of dystonia as measured by change from baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) - Total Score
Time Frame: Week 4
Week 4

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Change from baseline in Toronto Western Spasmodic Torticollis Rating Scale(TWSTRS) - Total Score
Time Frame: Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24
Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24
Change from baseline in TWSTRS subscale scores (Severity, Disability and Pain)
Time Frame: Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24
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
Time Frame: Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24
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
Time Frame: Weeks 2, 4, 6, 9, 12, 16, 20, 24
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)
Time Frame: Post-treatment: Weeks 4, 6, 12, 16, 20, 24
Post-treatment: Weeks 4, 6, 12, 16, 20, 24
Percentage of responders showing improvement on Clinical Global Impression of Change (CGIC)
Time Frame: Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24
Post-treatment: Weeks 2, 4, 6, 9, 12, 16, 20, 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Revance Therapeutics, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Nubia Kaba, Sponsor GmbH

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 7, 2016

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 17, 2017

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 8, 2016

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 8, 2016

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 11, 2016

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 6, 2019

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 30, 2019

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
October 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • RT002-CL005

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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