Dysport for the Treatment of OMD

NCT ID: NCT01921270

Last Updated: 2017-11-17

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

18 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-08-31

Study Completion Date

2017-02-08

Brief Summary

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The purpose of this study is to study the efficacy and safety of AbobotulinumtoxinA (Dysport) for use in Oromandibular Dystonia (OMD).

Detailed Description

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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.

Conditions

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Oral Dystonia Tardive Dystonia

Keywords

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Oromandibular Dystonia Oral Dystonia Tardive Dystonia botulinum toxin abobotulinumtoxinA

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

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.

Study Groups

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Dysport Injections

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

Group Type EXPERIMENTAL

Low Dose - AbobotulinumtoxinA

Intervention Type DRUG

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

Interventions

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Low Dose - AbobotulinumtoxinA

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

Intervention Type DRUG

Other Intervention Names

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Dysport

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ipsen

INDUSTRY

Sponsor Role collaborator

Emory University

OTHER

Sponsor Role lead

Responsible Party

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Stewart Factor

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Stewart A Factor, DO

Role: PRINCIPAL_INVESTIGATOR

Emory University

Locations

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Wesley Woods Health Center; Emory University Hospital

Atlanta, Georgia, United States

Site Status

Countries

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United States

References

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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.

Reference Type BACKGROUND
PMID: 10599789 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 21348790 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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IRB00064292

Identifier Type: -

Identifier Source: org_study_id