Brief Summary
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.
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.
Brief Title
Effects of DaxibotulinumtoxinA for Blepharospasm and Hemifacial Spasm
Detailed Description
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.
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.
Categories
Central Contacts
Central Contact Role
Contact
Central Contact Phone
718-920-2020
Central Contact Email
rickyparamo@gmail.com
Completion Date
Completion Date Type
Estimated
Conditions
Benign Essential Blepharospasm
Hemifacial Spasm
Eligibility Criteria
Inclusion Criteria:
* 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.
Exclusion Criteria:
* Patients will be excluded if age \< 18, are pregnant, non-willing, or with contra-indications to botulinum toxin.
* 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.
Exclusion Criteria:
* Patients will be excluded if age \< 18, are pregnant, non-willing, or with contra-indications to botulinum toxin.
Inclusion Criteria
Inclusion Criteria:
* 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.
* 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.
Gender
All
Gender Based
false
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Minimum Age
18 Years
NCT Id
NCT06195241
Org Class
Other
Org Full Name
Montefiore Medical Center
Org Study Id
2023-15151
Overall Status
Recruiting
Phases
Phase 4
Primary Completion Date
Primary Completion Date Type
Estimated
Official Title
The Safety and Efficacy of DaxibotulinumtoxinA-Lanm for Benign Essential Blepharospasm and Hemifacial Spasm
Primary Outcomes
Outcome Description
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.
Outcome Measure
Therapeutic Response based on the Jankovic Rating Scale
Outcome Time Frame
Prior to treatment at beginning of study
Outcome Description
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.
Outcome Measure
Therapeutic Response based on the Jankovic Rating Scale
Outcome Time Frame
1 month post-treatment
Outcome Description
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.
Outcome Measure
Therapeutic Response based on the Jankovic Rating Scale
Outcome Time Frame
3 months post-treatment
Outcome Description
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.
Outcome Measure
Therapeutic Response based on the Jankovic Rating Scale
Outcome Time Frame
On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months
Secondary Ids
Secondary Id
2023-IST-DAXI-000186
Secondary Outcomes
Outcome Description
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.
Outcome Time Frame
Prior to treatment at beginning of study
Outcome Measure
Waning of Effect
Outcome Description
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.
Outcome Time Frame
1 month post-treatment
Outcome Measure
Waning of Effect
Outcome Description
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.
Outcome Time Frame
3 months post-treatment
Outcome Measure
Waning of Effect
Outcome Description
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.
Outcome Time Frame
On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months
Outcome Measure
Waning of Effect
Outcome Description
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.
Outcome Time Frame
Prior to treatment at beginning of study
Outcome Measure
Loss of Efficacy
Outcome Description
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.
Outcome Time Frame
1 month post-treatment
Outcome Measure
Loss of Efficacy
Outcome Description
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.
Outcome Time Frame
3 months post-treatment
Outcome Measure
Loss of Efficacy
Outcome Description
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.
Outcome Time Frame
On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months
Outcome Measure
Loss of Efficacy
Outcome Description
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.
Outcome Time Frame
Up to four week post treatment
Outcome Measure
Incidence Rate of Treatment Failure
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
18
Investigators
Investigator Type
Principal Investigator
Investigator Name
Anne Barmettler
Investigator Email
abarmett@montefiore.org
Investigator Department
Ophthalmology & Visual Sciences
Investigator Sponsor Organization
External
Study Department
Ophthalmology and Visual Sciences
Study Division
Not Applicable
Categories Mesh Debug
Digestive System --- MOUTH DISEASES
Digestive System --- STOMATOGNATHIC DISEASES
Orthopedics, Muscle & Bone --- NEUROMUSCULAR MANIFESTATIONS
Brain, Spinal Cord & Nervous System --- NEUROLOGIC MANIFESTATIONS
Headaches & Migraine --- NEUROLOGIC MANIFESTATIONS
Substance Use and Addiction --- NEUROLOGIC MANIFESTATIONS
Brain, Spinal Cord & Nervous System --- NERVOUS SYSTEM DISEASES
Brain, Spine & Nerve Cancers --- NERVOUS SYSTEM DISEASES
MeSH Terms
BENIGN ESSENTIAL BLEPHAROSPASM
HEMIFACIAL SPASM
MOUTH DISEASES
STOMATOGNATHIC DISEASES
SPASM
NEUROMUSCULAR MANIFESTATIONS
NEUROLOGIC MANIFESTATIONS
NERVOUS SYSTEM DISEASES
SIGNS AND SYMPTOMS
PATHOLOGICAL CONDITIONS, SIGNS AND SYMPTOMS
BOTULINUM TOXINS, TYPE A
BOTULINUM TOXINS
METALLOENDOPEPTIDASES
ENDOPEPTIDASES
PEPTIDE HYDROLASES
HYDROLASES
ENZYMES
ENZYMES AND COENZYMES
METALLOPROTEASES
BACTERIAL PROTEINS
PROTEINS
AMINO ACIDS, PEPTIDES, AND PROTEINS
BACTERIAL TOXINS
TOXINS, BIOLOGICAL
BIOLOGICAL FACTORS