Dysport for the Treatment of OMD

Phase 1

Completed

• Conditions: Oral Dystonia; Tardive Dystonia
• Interventions: Drug: Low Dose - AbobotulinumtoxinA

• Registration Number: NCT01921270
• Lead Sponsor: Emory University

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.

Study Timeline

• Study Start: 2013-08
• Study First Submitted: 2013-08-09
• Study First Submitted QC: 2013-08-12
• Study First Posted: 2013-08-13
• Primary Completion: 2017-02-08
• Study Completion: 2017-02-08
• Results First Submitted: 2017-07-24
• Status Verified: 2017-10
• Results First Submitted QC: 2017-10-19
• Last Update Submitted: 2017-10-19
• Results First Posted: 2017-11-17
• Last Update Posted: 2017-11-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 18

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.

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Dysport Injections	Low Dose - AbobotulinumtoxinA	Participants with OMD who have been previously treated with any botulinum toxin Type A will be injected with Dysport®.

Primary Outcome Measures

Name	Time	Method
Mean Global Dystonia Rating Scale Score as Measured by Blinded Rater	Baseline, Week 6, Week 12	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

Name	Time	Method
Mean Global Clinical Impression Scale (CGI-S) With Severity Index Score	Baseline, Week 6, Week 12	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 Oromandibular Dystonia Quality of Life Questionnaire (OMDQ-25) Score	Baseline, Week 6, Week 12	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	Week 6, Week 12	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 Unified Dystonia Rating Scale (UDRS) Score as Measured by Blinded Rater	Baseline, Week 6, Week 12	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.
Change in Analogue Pain Scale Score	Baseline, Week 12	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	Baseline, Week 6, Week 12	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	Baseline, Week 12	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	Baseline, Week 6, Week 12	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	Baseline, Week 6, Week 12	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 Global Dystonia Rating Scale Score as Measured by Un-blinded Rater	Baseline, Week 6, Week 12	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 Global Clinical Impression- Efficacy Index Score	Week 6, Week 12	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	Baseline, Week 6, Week 12	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.

Trial Locations

Locations (1)

Wesley Woods Health Center; Emory University Hospital; 🇺🇸 Atlanta, Georgia, United States