The Safety and Efficacy of DaxibotulinumtoxinA-Lanm for Benign Essential Blepharospasm and Hemifacial Spasm
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 2
- 主要终点
- Therapeutic Response based on the Jankovic Rating Scale
研究概览
简要总结
The goal of this clinical trial is to test the effects of DaxibotulinumtoxinA-Lanm (Daxxify) in patients with benign essential blepharospasms (BEB) and hemifacial spasms (HFS). The main questions to answer:
- Is there clinically significant difference (measured by Jankovic Rating Scale (JRS) score from base to peak efficacy) for patients with BEB and HFS treated with Daxxify?
- What percentage of patients achieve a clinical response?
Participants historically treated with Botox for either BEB or HFS will be crossed over to Daxxify treatment in order to serve as their own control and examine the efficacy of Daxxify.
详细描述
This will be a single-arm, crossover study, in which each patient is their own historical control using a 2:1 conversion ratio of Daxxify to Botox units. Study participants will be selected through the electronic medical records of ophthalmologists at Montefiore Medical Center. Medical documentation on each patient will include information on age, sex, race, Benign Essential Blepharospasm (BEB) diagnosis year and duration, Hemifacial Spasms (HFS) diagnosis year and duration, and past treatments.
The immediate effects (desired or undesired) of Daxxify observed within 48-72 hours, will be defined as (1) complete relief of spasms (2) partial relief to a tolerable level (3) mild to moderate ocular irritation (4) ptosis.
Overall efficacy will be evaluated using the base to peak efficacy (recorded in changes to Jankovic Rating Scale (JRS) scores) and be defined as (1) excellent (resolution of signs and symptoms, only requiring injections > 5-6 months (2) moderate (improvement in signs and symptoms but requiring repeated injections within < 5 months (3) poor (no improvement in signs and symptoms).
Adverse side effects will be documented, and Daxxify will be discontinued in any patients who develop significant side effects that outweigh the benefits.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Benign essential blepharospasm (BEB) or hemifacial spasm (HFS) as diagnosed by an ophthalmologist.
- •No known neurologic or neuromuscular systematic medications.
- •No history or surgical intervention for BEB or HFS.
排除标准
- •Patients will be excluded if age < 18, are pregnant, non-willing, or with contra-indications to botulinum toxin.
研究组 & 干预措施
Benign Essential Blepharospasm or Hemifacial Spasm
Patients with either Benign Essential Blepharospasms or Hemifacial Spasms
干预措施: DaxibotulinumtoxinA (Drug)
结局指标
主要结局
Therapeutic Response based on the Jankovic Rating Scale
时间窗: On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months
Therapeutic response of Daxxify will be measured as the significant difference between the baseline to peak efficacy scores using the modified Jankovic rating scale (JRS). The JRS scale is a validated physician rating scale that consists of two subscales: blepharospasm (or hemifacial spasm) severity and blepharospasm (or hemifacial spasm) frequency. Each subscale is based on a 5-point scale ranging from 0-4, where 0 indicates no symptoms or frequency and 4 indicates the most severe or frequent symptoms. Overall scores will be reported as the sum of the two subscales of severity and frequency of symptoms and will range from 0-8. Basic descriptive statistics will be used to summarize and report therapeutic response. The Wilcoxon signed-ranked test will be used to analyze paired ordinal data gathered from the JRS scores.
Therapeutic Response based on the Jankovic Rating Scale
时间窗: Prior to treatment at beginning of study, 1 month post-treatment, 3 months post-treatment, and on a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months
Therapeutic response of Daxxify will be measured as the significant difference between the baseline to peak efficacy scores using the modified Jankovic rating scale (JRS). The JRS scale is a validated physician rating scale that consists of two subscales: blepharospasm (or hemifacial spasm) severity and blepharospasm (or hemifacial spasm) frequency. Each subscale is based on a 5-point scale ranging from 0-4, where 0 indicates no symptoms or frequency and 4 indicates the most severe or frequent symptoms. Overall scores will be reported as the sum of the two subscales of severity and frequency of symptoms and will range from 0-8. Basic descriptive statistics will be used to summarize and report therapeutic response. The Wilcoxon signed-ranked test will be used to analyze paired ordinal data gathered from the JRS scores.
次要结局
- Waning of Effect(On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months)
- Loss of Efficacy(On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months)
- Incidence Rate of Treatment Failure(Up to four week post treatment)
- Waning of Effect(Prior to treatment at beginning of study, 1 month post-treatment, 3 months post-treatment, and on a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months)
- Loss of Efficacy(Prior to treatment at beginning of study, 1 month post-treatment, 3 months post-treatment, and on a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months)