Effects of DaxibotulinumtoxinA for Blepharospasm and Hemifacial Spasm

NCT06195241 | Recruiting Phase 4 DMC FDA Drug

• 2 other identifiers: 2023-151512023-IST-DAXI-000186
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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. 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. 2.What percentage of patients achieve a clinical response?

Conditions

Benign Essential Blepharospasm; Hemifacial Spasm

Outcome Measures

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.

  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

Secondary Outcomes (3)

• Waning of Effect

  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

  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

  Up to four weeks post treatment

Study Arms (1)

Benign Essential Blepharospasm or Hemifacial Spasm

EXPERIMENTAL

Patients with either Benign Essential Blepharospasms or Hemifacial Spasms in accordance with Eligibility criteria.

Drug: DaxibotulinumtoxinA

Interventions

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.

Also known as: Daxxify

Benign Essential Blepharospasm or Hemifacial Spasm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Montefiore Medical Center: lead
• Revance Therapeutics, Inc.: collaborator

Study Sites (1)

Montefiore Medical Center

The Bronx, New York, 10466, United States

RECRUITING

Related Publications (9)

• Anwar MS, Zafar H. Efficacy of botulinum toxin in benign essential Blepharospasm: Desirable & undesirable effects. Pak J Med Sci. 2013;29(6):1389-1393. doi:10.12669/pjms.296.3853

  BACKGROUND

• Carruthers JD, Fagien S, Joseph JH, et al. 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;145(1):45-58. doi:10.1097/PRS.0000000000006327

  BACKGROUND

• Dutton JJ, Fowler AM. Botulinum toxin in ophthalmology. Surv Ophthalmol. 2007;52(1):13-31. doi:10.1016/j.survophthal.2006.10.003

  BACKGROUND

• Hellman A, Torres-Russotto D. Botulinum toxin in the management of blepharospasm: current evidence and recent developments. Ther Adv Neurol Disord. 2015;8(2):82-91. doi:10.1177/1756285614557475

  BACKGROUND

• Okumus S, Coskun E, Erbagci I, et al. Botulinum toxin injections for blepharospasm prior to ocular surgeries. Clin Ophthalmol. 2012;6:579-583. doi:10.2147/OPTH.S30277

  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;81(18):2091-2101. doi:10.1007/s40265-021-01631-w

  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;13(12):881. Published 2021 Dec 9. doi:10.3390/toxins13120881

  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;128(6):793-802. doi:10.1007/s00702-021-02343-x

  BACKGROUND

• Bellows S, Jankovic J. Immunogenicity Associated with Botulinum Toxin Treatment. Toxins (Basel). 2019;11(9):491. Published 2019 Aug 26. doi:10.3390/toxins11090491

  BACKGROUND

Related Links

• DailyMed - DAXXIFY- botulinum toxin type A injection, powder, lyophilized, for solution. U.S. National Library of Medicine.

MeSH Terms

Conditions

Benign essential blepharospasm; Hemifacial Spasm

Interventions

Botulinum Toxins, Type A

Condition Hierarchy (Ancestors)

Mouth Diseases; Stomatognathic Diseases; Spasm; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

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

Study Officials

• Anne Barmettler, MD

  Montefiore Medical Center/Albert Einstein College of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Isaac M Weber, BA

CONTACT

347-706-0146; moshe.weber@einsteinmed.edu

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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 Record Dates

• First Submitted: December 22, 2023
• First Posted: January 8, 2024
• Study Start: February 11, 2025
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: April 23, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)