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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-IST-DAXI-000186 | Other Identifier | Revance Therapeutics |
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| Name | Class |
|---|---|
| Revance Therapeutics, Inc. | INDUSTRY |
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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:
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Benign Essential Blepharospasm or Hemifacial Spasm | Experimental | Patients with either Benign Essential Blepharospasms or Hemifacial Spasms in accordance with Eligibility criteria. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| DaxibotulinumtoxinA | Drug | 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 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Isaac M Weber, BA | Contact | 347-706-0146 | moshe.weber@einsteinmed.edu |
| Name | Affiliation | Role |
|---|---|---|
| Anne Barmettler, MD | Montefiore Medical Center/Albert Einstein College of Medicine | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Montefiore Medical Center | Recruiting | The Bronx | New York | 10466 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 24550960 | Background | Anwar MS, Zafar H. Efficacy of botulinum toxin in benign essential Blepharospasm: Desirable & undesirable effects. Pak J Med Sci. 2013 Nov;29(6):1389-93. doi: 10.12669/pjms.296.3853. | |
| 31609882 | Background | Carruthers JD, Fagien S, Joseph JH, Humphrey SD, Biesman BS, Gallagher CJ, Liu Y, Rubio RG; SAKURA 1 and SAKURA 2 Investigator Group; SAKURA 1 and SAKURA 2 Investigator Group includes the following. DaxibotulinumtoxinA for Injection for the Treatment of Glabellar Lines: Results from Each of Two Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 3 Studies (SAKURA 1 and SAKURA 2). Plast Reconstr Surg. 2020 Jan;145(1):45-58. doi: 10.1097/PRS.0000000000006327. |
| Label | URL |
|---|---|
| DailyMed - DAXXIFY- botulinum toxin type A injection, powder, lyophilized, for solution. U.S. National Library of Medicine. | View source |
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| ID | Term |
|---|---|
| C535428 | Benign essential blepharospasm |
| D019569 | Hemifacial Spasm |
| ID | Term |
|---|---|
| D009059 | Mouth Diseases |
| D009057 | Stomatognathic Diseases |
| D013035 | Spasm |
| D020879 | Neuromuscular Manifestations |
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| ID | Term |
|---|---|
| D019274 | Botulinum Toxins, Type A |
| ID | Term |
|---|---|
| D001905 | Botulinum Toxins |
| D008666 | Metalloendopeptidases |
| D010450 | Endopeptidases |
| D010447 | Peptide Hydrolases |
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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.
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| 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. | 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 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. | Up to four weeks post treatment |
| 17212988 | Background | Dutton JJ, Fowler AM. Botulinum toxin in ophthalmology. Surv Ophthalmol. 2007 Jan-Feb;52(1):13-31. doi: 10.1016/j.survophthal.2006.10.003. |
| 25922620 | Background | Hellman A, Torres-Russotto D. Botulinum toxin in the management of blepharospasm: current evidence and recent developments. Ther Adv Neurol Disord. 2015 Mar;8(2):82-91. doi: 10.1177/1756285614557475. |
| 22654489 | Background | Okumus S, Coskun E, Erbagci I, Tatar MG, Comez A, Kaydu E, Yayuspayi R, Gurler B. Botulinum toxin injections for blepharospasm prior to ocular surgeries. Clin Ophthalmol. 2012;6:579-83. doi: 10.2147/OPTH.S30277. Epub 2012 May 3. |
| 34787840 | Background | Solish N, Carruthers J, Kaufman J, Rubio RG, Gross TM, Gallagher CJ. Overview of DaxibotulinumtoxinA for Injection: A Novel Formulation of Botulinum Toxin Type A. Drugs. 2021 Dec;81(18):2091-2101. doi: 10.1007/s40265-021-01631-w. Epub 2021 Nov 17. |
| 34941718 | Background | Tambasco N, Filidei M, Nigro P, Parnetti L, Simoni S. Botulinum Toxin for the Treatment of Hemifacial Spasm: An Update on Clinical Studies. Toxins (Basel). 2021 Dec 9;13(12):881. doi: 10.3390/toxins13120881. |
| 33963897 | Background | Wabbels B, Yaqubi A. Validation of a new hemifacial spasm grading questionnaire (HFS score) assessing clinical and quality of life parameters. J Neural Transm (Vienna). 2021 Jun;128(6):793-802. doi: 10.1007/s00702-021-02343-x. Epub 2021 May 8. |
| 31454941 | Background | Bellows S, Jankovic J. Immunogenicity Associated with Botulinum Toxin Treatment. Toxins (Basel). 2019 Aug 26;11(9):491. doi: 10.3390/toxins11090491. |
| D009461 |
| Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D006867 |
| Hydrolases |
| D004798 | Enzymes |
| D045762 | Enzymes and Coenzymes |
| D045726 | Metalloproteases |
| D001426 | Bacterial Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D001427 | Bacterial Toxins |
| D014118 | Toxins, Biological |
| D001685 | Biological Factors |