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: 1. 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? 2. 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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
40
Patients with Benign Essential Blepharospasm (BEB) or Hemifacial spasms (HFS) will be treated with DaxibotulinumtoxinA-Lanm (Daxxify) using a 2:1 conversion rate of Daxxify to Botox.
Montefiore Medical Center
The Bronx, New York, United States
RECRUITINGTherapeutic Response based on the Jankovic Rating Scale
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.
Time frame: Prior to treatment at beginning of study
Therapeutic Response based on the Jankovic Rating Scale
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.
Time frame: 1 month post-treatment
Therapeutic Response based on the Jankovic Rating Scale
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.
Time frame: 3 months post-treatment
Therapeutic Response based on the Jankovic Rating Scale
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.
Time frame: On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months
Waning of Effect
Waning of effect will be assessed by quantifying the patient-reported time, in months, to noticing a loss of peak treatment effect. The number of months until noticing a loss of peak treatment effect will be summarized and reported using basic descriptive statistics.
Time frame: Prior to treatment at beginning of study
Waning of Effect
Waning of effect will be assessed by quantifying the patient-reported time, in months, to noticing a loss of peak treatment effect. The number of months until noticing a loss of peak treatment effect will be summarized and reported using basic descriptive statistics.
Time frame: 1 month post-treatment
Waning of Effect
Waning of effect will be assessed by quantifying the patient-reported time, in months, to noticing a loss of peak treatment effect. The number of months until noticing a loss of peak treatment effect will be summarized and reported using basic descriptive statistics.
Time frame: 3 months post-treatment
Waning of Effect
Waning of effect will be assessed by quantifying the patient-reported time, in months, to noticing a loss of peak treatment effect. The number of months until noticing a loss of peak treatment effect will be summarized and reported using basic descriptive statistics.
Time frame: On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months
Loss of Efficacy
Loss of efficacy will be assessed by quantifying the patient-reported time, in months, to noticing a complete loss of efficacy. The number of months to complete loss of efficacy will be summarized and reported using basic descriptive statistics.
Time frame: Prior to treatment at beginning of study
Loss of Efficacy
Loss of efficacy will be assessed by quantifying the patient-reported time, in months, to noticing a complete loss of efficacy. The number of months to complete loss of efficacy will be summarized and reported using basic descriptive statistics.
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Time frame: 1 month post-treatment
Loss of Efficacy
Loss of efficacy will be assessed by quantifying the patient-reported time, in months, to noticing a complete loss of efficacy. The number of months to complete loss of efficacy will be summarized and reported using basic descriptive statistics.
Time frame: 3 months post-treatment
Loss of Efficacy
Loss of efficacy will be assessed by quantifying the patient-reported time, in months, to noticing a complete loss of efficacy. The number of months to complete loss of efficacy will be summarized and reported using basic descriptive statistics.
Time frame: On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months
Incidence Rate of Treatment Failure
Participant incidence rate of treatment failure will be measured as a lack of response or primary non-response (defined as a \< 25% response from the first injection and subsequent injections of increasing dosages) to medication up to four weeks later for either benign essential blepharospasm or hemifacial spasm. Incidence rate of treatment failure will be defined as the percentage of participants who did not respond to treatment and will be summarized using basic descriptive statistics.
Time frame: Up to four week post treatment