Clinical trial NCT01921270: 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
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Oral Dystonia
Tardive Dystonia
Keywords: Oromandibular Dystonia
Oral Dystonia
Tardive Dystonia
botulinum toxin
abobotulinumtoxinA
Lead sponsor: Emory University
Sponsor class: OTHER
Collaborators: name: Ipsen
First posted: 2013-08-13
Enrollment: 18
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Mean Global Dystonia Rating Scale Score as Measured by Blinded Rater
timeFrame: Baseline, Week 6, Week 12
Secondary outcomes:
measure: Change in Analogue Pain Scale Score
timeFrame: Baseline, Week 12
measure: Mean Sialorrhea Clinical Scale for Parkinson's Disease (SCS-PD) Score
timeFrame: Baseline, Week 6, Week 12
measure: Change in Number of Tongue Bites Per Day
timeFrame: Baseline, Week 12
measure: Mean Swallowing Disturbance Questionnaire (SDQ-20) Score
timeFrame: Baseline, Week 6, Week 12
measure: Mean Fahn-Marsden Part B "Speech" Question (BFM-q21) Rating
timeFrame: Baseline, Week 6, Week 12
measure: Mean Oromandibular Dystonia Quality of Life Questionnaire (OMDQ-25) Score
timeFrame: Baseline, Week 6, Week 12
measure: Mean Global Clinical Impression - Improvement Scale (CGI) Index Score
timeFrame: Week 6, Week 12
measure: Mean Global Clinical Impression Scale (CGI-S) With Severity Index Score
timeFrame: Baseline, Week 6, Week 12
measure: Mean Global Clinical Impression- Efficacy Index Score
timeFrame: Week 6, Week 12
measure: Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Un-blinded Rater
timeFrame: Baseline, Week 6, Week 12
measure: Mean Global Dystonia Rating Scale Score as Measured by Un-blinded Rater
timeFrame: Baseline, Week 6, Week 12
measure: Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Blinded Rater
timeFrame: Baseline, Week 6, Week 12
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Wesley Woods Health Center; Emory University Hospital
state: Georgia
Study officials:
affiliation: Emory University
name: Stewart A Factor, DO
role: PRINCIPAL_INVESTIGATOR
References:
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.
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.