Effects of DaxibotulinumtoxinA for Blepharospasm and Hemifacial Spasm
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age18+
SponsorMontefiore Medical Center
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Patients are required to have a minimum score of 4 out of 8 on the modified Jankovic Rating Scale for Severity/Frequency.
Disqualifiers
Patients will be excluded if age < 18, are pregnant, are non-willing, or have contra-indications to botulinum toxin.
Trial design
Design model
Single group
Treatments tested in this trial
DaxibotulinumtoxinA
DrugPatients 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 units. BEB dosing: participants will receive Daxxify at 10 sites using a 2:1 conversion ratio of Daxxify to Botox units. The location of these injections are in the superficial dermis in the sites typical for BEB. HFS dosing: participants will then receive Daxxify at 8 sites using a 2:1 conversion ratio of Daxxify to Botox units. The location of these injections are in the superficial dermis in the sites typical for HFS. Patients will be followed monthly to measure duration and efficacy of the Daxxify. Patients will receive their single dose of Daxxify at the same time point in which they would have normally received their next Botox treatment, without a washout period.
Treatment groups
40 Participants
are divided into 1 treatment groupGroup A: Benign Essential Blepharospasm or Hemifacial SpasmExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Therapeutic Response based on the modified 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. The modified JRS will be modified slightly to include hemifacial spasm as opposed to just including blepharospasm. 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, 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 monthsSecondary outcomes
1
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 (i.e., start of waning). 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, 3 months post-treatment, and on a monthly basis after 3-month timepoint until symptoms return to baseline, up to a maximum of 9 months2
Loss of Efficacy
Loss of efficacy will be assessed by quantifying the patient-reported time, in months, to noticing a complete loss of efficacy (return to baseline symptom status). The number of months to complete loss of efficacy will be summarized and reported using basic descriptive statistics.
Time frame
1 month post-treatment, 3 months post-treatment, and on a monthly basis after 3-month timepoint until symptoms return to baseline, up to a maximum of 9 months3
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 weeks post treatmentSponsors and contacts
Click on the lead sponsor to view all of their trials.
Montefiore Medical Center
Lead sponsor
Revance Therapeutics, Inc.
Collaborator
Source ClinicalTrials.gov