Effects of DaxibotulinumtoxinA for Blepharospasm and Hemifacial Spasm

Phase 4

Recruiting

• Conditions: Benign Essential Blepharospasm; Hemifacial Spasm
• Interventions: Drug: DaxibotulinumtoxinA

• Registration Number: NCT06195241
• Lead Sponsor: Montefiore Medical Center

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.

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.

Study Timeline

• Study First Submitted: 2023-12-22
• Study First Submitted QC: 2023-12-22
• Study First Posted: 2024-01-08
• Study Start: 2025-02-11
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-07
• Last Update Posted: 2025-04-09
• Primary Completion: 2027-03
• Study Completion: 2027-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Benign Essential Blepharospasm or Hemifacial Spasm	DaxibotulinumtoxinA	Patients with either Benign Essential Blepharospasms or Hemifacial Spasms

Primary Outcome Measures

Name	Time	Method
Therapeutic Response based on the Jankovic Rating Scale	On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months	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.

Secondary Outcome Measures

Name	Time	Method
Waning of Effect	On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months	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.
Loss of Efficacy	On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months	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.
Incidence Rate of Treatment Failure	Up to four week post treatment	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.

Trial Locations

Locations (1)

Montefiore Medical Center; 🇺🇸 Bronx, New York, United States