search
Back to results

Dysport for the Treatment of OMD

Primary Purpose

Oral Dystonia, Tardive Dystonia

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Low Dose - AbobotulinumtoxinA
Sponsored by
Emory University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Oral Dystonia focused on measuring Oromandibular Dystonia, Oral Dystonia, Tardive Dystonia, botulinum toxin, abobotulinumtoxinA

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • a diagnosis of primary or tardive OMD
  • moderate or severe severity, defined as GDS score ≥4 in either "lower face" or "jaw and tongue" section
  • capability of attending the scheduled visits
  • only those who have been previously injected with onabotulinumtoxinA and responded to that treatment, and are at least 12 weeks post last injection
  • Women of childbearing age need to use contraception in order to be included.

Exclusion Criteria:

  • Existence of a systemic disease that could confound the evaluation
  • previous placement of Deep Brain Stimulation electrodes to treat dystonia
  • concomitant oral medications that could interfere with the action of botulinum toxin Type A (e.g., aminoglycosides)
  • on an unstable dosage of any medication prescribed to treat dystonia (e.g., benzodiazepines, baclofen or anticholinergics)
  • any known hypersensitivity to any botulinum toxin preparation and allergy to cow's milk protein
  • immunoresistance to other forms of botulinum toxin type A
  • existence of a concomitant neuromuscular disorder (e.g., Myasthenia Gravis or Lambert-Eaton syndrome, etc)
  • infection at the proposed injection sites
  • pregnant women
  • women of childbearing age NOT on contraception
  • breastfeeding women
  • inability to comply with scheduled visits
  • patients who had been previously injected with botulinum toxin type A but who did not respond

Sites / Locations

  • Wesley Woods Health Center; Emory University Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Dysport Injections

Arm Description

Participants with OMD who have been previously treated with any botulinum toxin Type A will be injected with Dysport®.

Outcomes

Primary Outcome Measures

Mean Global Dystonia Rating Scale Score as Measured by Blinded Rater
This scale measures the severity of dystonia for the jaw and tongue by a blinded rater. Dystonia is rated from 0 to 10: 0=No dystonia present, 1=Minimal dystonia, 5=Moderate dystonia,10=Most severe dystonia

Secondary Outcome Measures

Change in Analogue Pain Scale Score
Measure of jaw pain by visual analogue scale (0-100) where 0 represents "no pain" and 100 represents the "most severe pain".
Mean Sialorrhea Clinical Scale for Parkinson's Disease (SCS-PD) Score
The SCS-PD measures drooling. Individual items are scored on a scale from 0-3 where 0 represents "never" and 3 represents "always". The overall maximum score is 21. A higher score indicates greater drooling severity. A lower score indicates lesser severity.
Change in Number of Tongue Bites Per Day
The patient will be asked to estimate how many times they tend to accidentally/involuntarily bite their tongue per day.
Mean Swallowing Disturbance Questionnaire (SDQ-20) Score
Ease of chewing and swallowing will be assessed by the SDQ-20 (modified to exclude question 5 due to redundancy as it relates to drooling and question 15 which is not relevant to the study as it involves prior aspiration pneumonias). Individual items are scored from 0 (never) to 3 (very frequently). The overall score is the total for all items; a higher score indicating more frequent swallowing disturbance; a lower score indicating no or less frequent disturbance, with a possible maximum score of 39.
Mean Fahn-Marsden Part B "Speech" Question (BFM-q21) Rating
The Fahn-Marsden Part B "Speech" Question assesses the ease of producing speech. Responses range from 0=Normal, 1=Slightly involved, easily understood, 2=Some difficulty understanding, 3=Marked difficulty understanding.
Mean Oromandibular Dystonia Quality of Life Questionnaire (OMDQ-25) Score
The OMDQ-25 is a subjective quality of life measurement made for patients with Oromandibular Dystonia. The maximum total score is 100 indicating the highest quality of life. A score of 50 indicates a mediocre quality of life. A lower score indicates perceived lower quality of life.
Mean Global Clinical Impression - Improvement Scale (CGI) Index Score
The Clinical Global Impression - Improvement scale (CGI-I) is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. Responses are scored on a scale from 1 to 7; 1 represents "very much improved" and 7 represents "very much worse".
Mean Global Clinical Impression Scale (CGI-S) With Severity Index Score
The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Responses are scored on a scale from 1 to 7; 1 represents "normal, not at all ill" and 7 represents "among the most extremely ill patients".
Mean Global Clinical Impression- Efficacy Index Score
The Clinical Global Impression - Efficacy Index is a 4×4 rating scale that assesses the therapeutic effect of treatment. Responses range on a scale from 0 to 4 with 4 being the best response.
Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Un-blinded Rater
The UDRS measures dystonia severity. The UDRS is being rated by un-blinded video evaluators regarding severity of subject's dystonia. Scores range from 0 to 10; 0 indicating no dystonia, 5 indicating moderate dystonia, and 10 indicating the worst dystonia.
Mean Global Dystonia Rating Scale Score as Measured by Un-blinded Rater
This scale measures the severity of dystonia for the jaw and tongue by an un-blinded rater. Dystonia is rated from 0 to 10: 0=No dystonia present, 1=Minimal dystonia, 5=Moderate dystonia,10=Most severe dystonia
Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Blinded Rater
The UDRS measures dystonia severity. The UDRS is being rated by blinded video evaluators regarding severity of subject's dystonia. Scores range from 0 to 10; 0 indicating no dystonia, 5 indicating moderate dystonia, and 10 indicating the worst dystonia.

Full Information

First Posted
August 9, 2013
Last Updated
October 19, 2017
Sponsor
Emory University
Collaborators
Ipsen
search

1. Study Identification

Unique Protocol Identification Number
NCT01921270
Brief Title
Dysport for the Treatment of OMD
Official Title
A Pilot Dose Ranging Study of Dysport® (AbobotulinumtoxinA) in the Treatment of Oromandibular Dystonia
Study Type
Interventional

2. Study Status

Record Verification Date
October 2017
Overall Recruitment Status
Completed
Study Start Date
August 2013 (undefined)
Primary Completion Date
February 8, 2017 (Actual)
Study Completion Date
February 8, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Emory University
Collaborators
Ipsen

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
The purpose of this study is to study the efficacy and safety of AbobotulinumtoxinA (Dysport) for use in Oromandibular Dystonia (OMD).
Detailed Description
Oromandibular dystonia (OMD) is an uncommon, disabling form of cranial dystonia, involving involuntary movements of the lower facial, masticatory, and lingual muscles. This can cause jaw movements including opening, closure, protrusion, retraction, or deviation. Common additional facial movements involve grimacing or lip pursing. When there is tongue involvement, it usually presents as tongue protrusion or curling. Such patients are impaired in relation to eating, speaking and swallowing This study aims to evaluate the efficacy and safety of a low dose of Dysport® deemed tolerable during phase 1 in subjects with oromandibular dystonia (OMD). Participants will be injected with Dysport® only, with an unblinded open-label disclosure. The safety and efficacy pf receiving Dysport® will be recorded for all subjects that undergo injection. All subjects will be examined and videotaped at the injection visit, then at 6 and 12 weeks after injection with a standardized protocol. The primary outcome will be blinded examination scores of the videos performed after the study is complete.The evaluators will be three different movement disorders experts, not otherwise involved in the study, who will review the videotaped examinations, presented in a random order, using the Global Dystonia Rating scale (GDS). Evaluators will rate the dystonia at baseline (injection visit) and 6 weeks after injection.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Oral Dystonia, Tardive Dystonia
Keywords
Oromandibular Dystonia, Oral Dystonia, Tardive Dystonia, botulinum toxin, abobotulinumtoxinA

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Masking Description
Evaluators will review the videotaped examinations, presented in a random order, using the Global Dystonia Rating scale (GDS). The evaluators will rate the dystonia at baseline (injection visit) and 6 weeks after injection.
Allocation
N/A
Enrollment
18 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Dysport Injections
Arm Type
Experimental
Arm Description
Participants with OMD who have been previously treated with any botulinum toxin Type A will be injected with Dysport®.
Intervention Type
Drug
Intervention Name(s)
Low Dose - AbobotulinumtoxinA
Other Intervention Name(s)
Dysport
Intervention Description
Participants will receive low dose AbobotulinumtoxinA injections. Location of injections will be determined by the clinician to treat the individual's dystonic symptom. Muscles that may be included for in injection for OMD with Jaw Closing: Medial Pterygoid 50 units, Masseter 25 units Muscles that may be included in injection for OMD with Jaw Opening: Lateral Pterygoid 50 units, Anterior digastrics 10 units Muscle that will be included in injection for OMD with Tongue Protrusion: Genioglossus 7.5 units
Primary Outcome Measure Information:
Title
Mean Global Dystonia Rating Scale Score as Measured by Blinded Rater
Description
This scale measures the severity of dystonia for the jaw and tongue by a blinded rater. Dystonia is rated from 0 to 10: 0=No dystonia present, 1=Minimal dystonia, 5=Moderate dystonia,10=Most severe dystonia
Time Frame
Baseline, Week 6, Week 12
Secondary Outcome Measure Information:
Title
Change in Analogue Pain Scale Score
Description
Measure of jaw pain by visual analogue scale (0-100) where 0 represents "no pain" and 100 represents the "most severe pain".
Time Frame
Baseline, Week 12
Title
Mean Sialorrhea Clinical Scale for Parkinson's Disease (SCS-PD) Score
Description
The SCS-PD measures drooling. Individual items are scored on a scale from 0-3 where 0 represents "never" and 3 represents "always". The overall maximum score is 21. A higher score indicates greater drooling severity. A lower score indicates lesser severity.
Time Frame
Baseline, Week 6, Week 12
Title
Change in Number of Tongue Bites Per Day
Description
The patient will be asked to estimate how many times they tend to accidentally/involuntarily bite their tongue per day.
Time Frame
Baseline, Week 12
Title
Mean Swallowing Disturbance Questionnaire (SDQ-20) Score
Description
Ease of chewing and swallowing will be assessed by the SDQ-20 (modified to exclude question 5 due to redundancy as it relates to drooling and question 15 which is not relevant to the study as it involves prior aspiration pneumonias). Individual items are scored from 0 (never) to 3 (very frequently). The overall score is the total for all items; a higher score indicating more frequent swallowing disturbance; a lower score indicating no or less frequent disturbance, with a possible maximum score of 39.
Time Frame
Baseline, Week 6, Week 12
Title
Mean Fahn-Marsden Part B "Speech" Question (BFM-q21) Rating
Description
The Fahn-Marsden Part B "Speech" Question assesses the ease of producing speech. Responses range from 0=Normal, 1=Slightly involved, easily understood, 2=Some difficulty understanding, 3=Marked difficulty understanding.
Time Frame
Baseline, Week 6, Week 12
Title
Mean Oromandibular Dystonia Quality of Life Questionnaire (OMDQ-25) Score
Description
The OMDQ-25 is a subjective quality of life measurement made for patients with Oromandibular Dystonia. The maximum total score is 100 indicating the highest quality of life. A score of 50 indicates a mediocre quality of life. A lower score indicates perceived lower quality of life.
Time Frame
Baseline, Week 6, Week 12
Title
Mean Global Clinical Impression - Improvement Scale (CGI) Index Score
Description
The Clinical Global Impression - Improvement scale (CGI-I) is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. Responses are scored on a scale from 1 to 7; 1 represents "very much improved" and 7 represents "very much worse".
Time Frame
Week 6, Week 12
Title
Mean Global Clinical Impression Scale (CGI-S) With Severity Index Score
Description
The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Responses are scored on a scale from 1 to 7; 1 represents "normal, not at all ill" and 7 represents "among the most extremely ill patients".
Time Frame
Baseline, Week 6, Week 12
Title
Mean Global Clinical Impression- Efficacy Index Score
Description
The Clinical Global Impression - Efficacy Index is a 4×4 rating scale that assesses the therapeutic effect of treatment. Responses range on a scale from 0 to 4 with 4 being the best response.
Time Frame
Week 6, Week 12
Title
Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Un-blinded Rater
Description
The UDRS measures dystonia severity. The UDRS is being rated by un-blinded video evaluators regarding severity of subject's dystonia. Scores range from 0 to 10; 0 indicating no dystonia, 5 indicating moderate dystonia, and 10 indicating the worst dystonia.
Time Frame
Baseline, Week 6, Week 12
Title
Mean Global Dystonia Rating Scale Score as Measured by Un-blinded Rater
Description
This scale measures the severity of dystonia for the jaw and tongue by an un-blinded rater. Dystonia is rated from 0 to 10: 0=No dystonia present, 1=Minimal dystonia, 5=Moderate dystonia,10=Most severe dystonia
Time Frame
Baseline, Week 6, Week 12
Title
Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Blinded Rater
Description
The UDRS measures dystonia severity. The UDRS is being rated by blinded video evaluators regarding severity of subject's dystonia. Scores range from 0 to 10; 0 indicating no dystonia, 5 indicating moderate dystonia, and 10 indicating the worst dystonia.
Time Frame
Baseline, Week 6, Week 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: a diagnosis of primary or tardive OMD moderate or severe severity, defined as GDS score ≥4 in either "lower face" or "jaw and tongue" section capability of attending the scheduled visits only those who have been previously injected with onabotulinumtoxinA and responded to that treatment, and are at least 12 weeks post last injection Women of childbearing age need to use contraception in order to be included. Exclusion Criteria: Existence of a systemic disease that could confound the evaluation previous placement of Deep Brain Stimulation electrodes to treat dystonia concomitant oral medications that could interfere with the action of botulinum toxin Type A (e.g., aminoglycosides) on an unstable dosage of any medication prescribed to treat dystonia (e.g., benzodiazepines, baclofen or anticholinergics) any known hypersensitivity to any botulinum toxin preparation and allergy to cow's milk protein immunoresistance to other forms of botulinum toxin type A existence of a concomitant neuromuscular disorder (e.g., Myasthenia Gravis or Lambert-Eaton syndrome, etc) infection at the proposed injection sites pregnant women women of childbearing age NOT on contraception breastfeeding women inability to comply with scheduled visits patients who had been previously injected with botulinum toxin type A but who did not respond
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Stewart A Factor, DO
Organizational Affiliation
Emory University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Wesley Woods Health Center; Emory University Hospital
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
10599789
Citation
Tan EK, Jankovic J. Botulinum toxin A in patients with oromandibular dystonia: long-term follow-up. Neurology. 1999 Dec 10;53(9):2102-7. doi: 10.1212/wnl.53.9.2102.
Results Reference
background
PubMed Identifier
21348790
Citation
Rosales RL, Ng AR, Santos MM, Fernandez HH. The broadening application of chemodenervation in X-linked dystonia-parkinsonism (Part II): an open-label experience with botulinum toxin-A (Dysport(R)) injections for oromandibular, lingual, and truncal-axial dystonias. Int J Neurosci. 2011;121 Suppl 1:44-56. doi: 10.3109/00207454.2011.558260.
Results Reference
background

Learn more about this trial

Dysport for the Treatment of OMD

We'll reach out to this number within 24 hrs