A Pilot Dose Ranging Study of Dysport® (AbobotulinumtoxinA) in the Treatment of Oromandibular Dystonia
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Mean Global Dystonia Rating Scale Score as Measured by Blinded Rater
研究概览
简要总结
The purpose of this study is to study the efficacy and safety of AbobotulinumtoxinA (Dysport) for use in Oromandibular Dystonia (OMD).
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
盲法说明
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.
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
研究组 & 干预措施
Dysport Injections
Participants with OMD who have been previously treated with any botulinum toxin Type A will be injected with Dysport®.
干预措施: Low Dose - AbobotulinumtoxinA (Drug)
结局指标
主要结局
Mean Global Dystonia Rating Scale Score as Measured by Blinded Rater
时间窗: 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
次要结局
- Mean Global Clinical Impression Scale (CGI-S) With Severity Index Score(Baseline, Week 6, Week 12)
- Mean Oromandibular Dystonia Quality of Life Questionnaire (OMDQ-25) Score(Baseline, Week 6, Week 12)
- Mean Global Clinical Impression - Improvement Scale (CGI) Index Score(Week 6, Week 12)
- Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Blinded Rater(Baseline, Week 6, Week 12)
- Change in Analogue Pain Scale Score(Baseline, Week 12)
- Mean Sialorrhea Clinical Scale for Parkinson's Disease (SCS-PD) Score(Baseline, Week 6, Week 12)
- Change in Number of Tongue Bites Per Day(Baseline, Week 12)
- Mean Swallowing Disturbance Questionnaire (SDQ-20) Score(Baseline, Week 6, Week 12)
- Mean Fahn-Marsden Part B "Speech" Question (BFM-q21) Rating(Baseline, Week 6, Week 12)
- Mean Global Dystonia Rating Scale Score as Measured by Un-blinded Rater(Baseline, Week 6, Week 12)
- Mean Global Clinical Impression- Efficacy Index Score(Week 6, Week 12)
- Mean Unified Dystonia Rating Scale (UDRS) Score as Measured by Un-blinded Rater(Baseline, Week 6, Week 12)