Prospective, Open-label, Single-arm, Single-center Trial to Investigate the Tolerability of NT 201 and Quality of Life of Patients in the Treatment of Blepharospasm With Shortened Injection Intervals
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- The difference between the actual Blepharospasm Disability Index [BSDI] value at each follow up visit and the BSDI value at baseline.
研究概览
简要总结
The objective of this Phase II study is to show that pre-treated patients with BEB with the need of shortened injection intervals achieve an improved and stable quality of life level if they receive repeated NT 201 injections at short injection intervals. In addition, the tolerability of repeated shortened NT 201 administrations in patients with BEB including the development of neutralizing antibodies will be investigated.
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
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A clinical diagnosis of bilateral BEB characterized by spontaneous, spasmodic, intermittent or persistent involuntary contractions of the orbicular oculi muscles
- •Medical need for treatment with shortened injection intervals (< 12 weeks) confirmed by the patient and investigator
- •Blepharospasm Disability Index (BSDI) at baseline visit before injection of > / = 1,6
排除标准
- •Atypical variant of blepharospasm caused by inhibition of levator palpebrae muscle (apraxia of the eyelid)
- •Generalized disorders of muscle activity (e.g. myasthenia gravis, Lambert-Eaton syndrome), as well as amyotrophic lateral sclerosis or any other significant neuromuscular disease which might interfere with the trial
- •The previous three injections with Botulinum toxin Type A with more than 50 Units [U] per eye per injection session
- •Treatment with Botulinum toxins for any indication other than BEB within 4 months prior to baseline and during the trial
研究组 & 干预措施
NT 201 (IncobotulinumtoxinA/Xeomin®)
干预措施: NT 201 (Drug)
结局指标
主要结局
The difference between the actual Blepharospasm Disability Index [BSDI] value at each follow up visit and the BSDI value at baseline.
时间窗: From baseline up to week 62. Follow up visits include 8 injection visits (V2 to V8) with intervals of at least 6 weeks between injections.
The BSDI includes 6 items to be assessed by a 5-point scoring (i.e., 0-4 points per item) ranging from "no impairment, "slight impairment", "moderate impairment", "severe impairment" to "no longer possible due to my illness". The 6 items are "Driving a vehicle", "Reading", "Watching TV", "Shopping", "Walking" and "Doing everyday activities".
次要结局
- Change in Jankovic Rating Scale (JRS) score from baseline over time.(From baseline up to week 62)
- Patient Evaluation of Global Response [PEGR] rated by patient at visits V2 to V8 and at follow-up visit(Week 6 up to week 62)