Clinical trial NCT06195241: Effects of DaxibotulinumtoxinA for Blepharospasm and Hemifacial Spasm
Official title: The Safety and Efficacy of DaxibotulinumtoxinA-Lanm for Benign Essential Blepharospasm and Hemifacial Spasm
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Benign Essential Blepharospasm
Hemifacial Spasm
Lead sponsor: Montefiore Medical Center
Sponsor class: OTHER
Collaborators: name: Revance Therapeutics, Inc.
First posted: 2024-01-08
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: This will be a single-arm, crossover study, in which each patient is his/her own historical control using a 2:1 conversion ratio of Daxxify to Botox units.
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Therapeutic Response based on the Jankovic Rating Scale
timeFrame: Prior to treatment at beginning of study
measure: Therapeutic Response based on the Jankovic Rating Scale
timeFrame: 1 month post-treatment
measure: Therapeutic Response based on the Jankovic Rating Scale
timeFrame: 3 months post-treatment
measure: Therapeutic Response based on the Jankovic Rating Scale
timeFrame: On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months
Secondary outcomes:
measure: Waning of Effect
timeFrame: Prior to treatment at beginning of study
measure: Waning of Effect
timeFrame: 1 month post-treatment
measure: Waning of Effect
timeFrame: 3 months post-treatment
measure: Waning of Effect
timeFrame: On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months
measure: Loss of Efficacy
timeFrame: Prior to treatment at beginning of study
measure: Loss of Efficacy
timeFrame: 1 month post-treatment
measure: Loss of Efficacy
timeFrame: 3 months post-treatment
measure: Loss of Efficacy
timeFrame: On a monthly basis after 3-month timepoint until symptom free, up to a maximum of 9 months
measure: Incidence Rate of Treatment Failure
timeFrame: Up to four week post treatment
Other outcomes:
Locations:
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
Study officials:
affiliation: Montefiore Medical Center/Albert Einstein College of Medicine
name: Anne Barmettler, MD
role: PRINCIPAL_INVESTIGATOR
References: