Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Primary |
Therapeutic Response based on the Jankovic Rating Scale |
Therapeutic response of Daxxify will be measured as the significant difference between the base to peak efficacy scores using the 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 to 4, where 0 indicates no symptoms or frequency and 4 indicates the most severe or frequent symptoms. Scores will be reported as two subscales of severity and frequency of symptoms. 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. |
Prior to treatment at beginning of study |
|
| Primary |
Therapeutic Response based on the Jankovic Rating Scale |
Therapeutic response of Daxxify will be measured as the significant difference between the base to peak efficacy scores using the 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 to 4, where 0 indicates no symptoms or frequency and 4 indicates the most severe or frequent symptoms. Scores will be reported as two subscales of severity and frequency of symptoms. 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. |
3 months post-treatment |
|
| Primary |
Therapeutic Response based on the Jankovic Rating Scale |
Therapeutic response of Daxxify will be measured as the significant difference between the base to peak efficacy scores using the 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 to 4, where 0 indicates no symptoms or frequency and 4 indicates the most severe or frequent symptoms. Scores will be reported as two subscales of severity and frequency of symptoms. 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. |
On a monthly basis after 3 month timepoint until symptom free, up to a maximum of 9 months |
|
| Secondary |
Duration of Treatment Effect |
Mean duration of effect will be measured as the time to loss, in months, of 80% of peak treatment effect from baseline based on Jankovic Rating Scale (JRS) scoring. As noted, 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 to 4, where 0 indicates no symptoms or frequency and 4 indicates the most severe or frequent symptoms. Scores will be reported as two subscales of severity and frequency of symptoms. Basic descriptive statistics will be used to summarize and report the mean duration of time to loss of effect. |
Up to 6 months following treatment |
|
| Secondary |
Subjective Response to Treatment |
Subjective response to treatment will be determined by analyzing significant differences using the Blepharospasm Disability Index (BSDI) or, for hemifacial spasms, using the Facial Clinimetric Evaluation (FaCE) Scale. The BSDI is a patient-rated scale that measures impairment of specific activities of daily living caused by blepharospasms on a 5-point scale ranging from 0 (no impairment) to 4 (activity not possible due to disease). Higher scores are associated with worsening severity of disease. FaCE is also a patient-rated scale used to assess quality of life due to hemifacial spasms. FaCE is comprised of 15 questions assessed on a 5-point scale ranging from 1 to 5 where 1 corresponds to the lowest level of function and 5 to the highest level of function. Scoring range is 0-75 and higher scores are associated with the best possible outcomes. The Wilcoxon signed-ranked test will be used to analyze paired ordinal data. |
Prior to treatment at beginning of study |
|
| Secondary |
Subjective Response to Treatment |
Subjective response to treatment will be determined by analyzing significant differences using the Blepharospasm Disability Index (BSDI) or, for hemifacial spasms, using the Facial Clinimetric Evaluation (FaCE) Scale. The BSDI is a patient-rated scale that measures impairment of specific activities of daily living caused by blepharospasms on a 5-point scale ranging from 0 (no impairment) to 4 (activity not possible due to disease). Higher scores are associated with worsening severity of disease. FaCE is also a patient-rated scale used to assess quality of life due to hemifacial spasms. FaCE is comprised of 15 questions assessed on a 5-point scale ranging from 1 to 5 where 1 corresponds to the lowest level of function and 5 to the highest level of function. Scoring range is 0-75 and higher scores are associated with the best possible outcomes. The Wilcoxon signed-ranked test will be used to analyze paired ordinal data. |
3 months post-treatment |
|
| Secondary |
Subjective Response to Treatment |
Subjective response to treatment will be determined by analyzing significant differences using the Blepharospasm Disability Index (BSDI) or, for hemifacial spasms, using the Facial Clinimetric Evaluation (FaCE) Scale. The BSDI is a patient-rated scale that measures impairment of specific activities of daily living caused by blepharospasms on a 5-point scale ranging from 0 (no impairment) to 4 (activity not possible due to disease). Higher scores are associated with worsening severity of disease. FaCE is also a patient-rated scale used to assess quality of life due to hemifacial spasms. FaCE is comprised of 15 questions assessed on a 5-point scale ranging from 1 to 5 where 1 corresponds to the lowest level of function and 5 to the highest level of function. Scoring range is 0-75 and higher scores are associated with the best possible outcomes. The Wilcoxon signed-ranked test will be used to analyze paired ordinal data. |
On a monthly basis after 3 month timepoint until primary outcome measure is achieved, estimated to be approximately 6 months |
|
| Secondary |
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. |
Up to four week post treatment |
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