Effects of DaxibotulinumtoxinA for Blepharospasm and Hemifacial Spasm

Официальное название

The Safety and Efficacy of DaxibotulinumtoxinA-Lanm for Benign Essential Blepharospasm and Hemifacial Spasm

Обзор

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.

Подробное описание

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.

Вмешательства

• Препарат DaxibotulinumtoxinA
  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

Первичные конечные точки

• Therapeutic Response based on the modified Jankovic Rating Scale [Срок оценки: 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]

Вторичные конечные точки (3)

• Waning of Effect [Срок оценки: 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]
• Loss of Efficacy [Срок оценки: 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 [Срок оценки: Up to four weeks post treatment]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• Patients will be excluded if age < 18, are pregnant, are non-willing, or have contra-indications to botulinum toxin.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 1 центр

• Montefiore Medical Center — The Bronx

Публикации

• 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. PMID 24550960
• 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). Plas PMID 31609882
• 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. PMID 17212988
• 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. PMID 25922620
• 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. PMID 22654489
• 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. PMID 34787840
• 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. PMID 34941718
• 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. PMID 33963897

Идентификаторы

NCT: NCT06195241 · 2023-15151 · 2023-IST-DAXI-000186

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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