DaxibotulinumtoxinA for Blepharospasm

(BLEXI Trial)

Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: University of Pennsylvania
Must be taking: OnabotulinumtoxinA, IncobotulinumtoxinA
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This study aims to provide real-world information about the duration, safety, and overall benefit of DaxibotulinumtoxinA (also called DAXI) treatment for adults living with blepharospasm (BSP), a condition that causes uncontrolled blinking or muscle spasms around the eyes, which can interfere with vision and daily activities. Specifically, it is being done to learn more about how well and how long DAXI works for treating adults with blepharospasm.

This is a single-center, open-label, single-arm study, meaning everyone in the study will receive DAXI, and both participants and researchers will know what treatment is being given. The study will include 20 adult participants.

Participants may receive two to three treatment cycles of DAXI injections over about 12 months. The timing between treatments will depend on how long each injection works for each person. Injections will be given at least every 90 days (3 months) but no later than every 180 days (6 months). Participants and their doctors will decide when another injection is needed based on symptom control using a tool called the Blepharospasm Severity Tracker Form (BSTF). To make the injections more comfortable, participants may use topical lidocaine cream, cooling spray, or another local anesthetic before injection.

DAXI will be prepared by the injecting clinician or trained staff right before use. The medication is made by mixing a measured amount of DAXI powder with a small amount of sterile saline solution (salt water) to reach the correct concentration. The exact injection technique (including the dose, location, and number of injection sites) will be chosen by the injector based on each participant's needs, but treatment will only be given in specific facial muscles (corrugator, procerus, orbicularis oculi, and nasalis). The use of imaging tools such as electromyography (EMG) or ultrasound is optional and typically not required for injections around the eyes.

The starting DAXI dose will be based on each participant's current or previous botulinum toxin treatment:

* If the participant was previously treated with onabotulinumtoxinA (Botox®), the same number of "units" will be used for DAXI (a 1:1 conversion).

* If the participant was previously treated with incobotulinumtoxinA (Xeomin®), the DAXI dose will be adjusted to about two-thirds of the previous incobotulinumtoxinA dose (a 1.5:1 conversion).

If a participant experienced side effects such as droopy eyelids (ptosis), double vision (diplopia), or dry eyes with prior treatments, that information will help guide dosing decisions. For later injection cycles, the injector may adjust the dose or injection pattern based on how well the participant responds. Whenever possible, the same injector will perform all of a participant's treatments to keep results consistent.

Participants will come to the clinic for in-person visits for most study assessments. After each DAXI injection, the peak effect (best response) will be evaluated about one month later, guided by the BSTF. These visits may be done remotely (via phone or video) when appropriate. The same schedule will be followed for future cycles. For the final treatment, this one-month check will occur unless the participant reports that the treatment's full effect happened sooner.

The main goal (primary endpoint) of the study is to measure how long DAXI's effects last, specifically by tracking the median time until the next injection is needed.

Other key goals (secondary endpoints) include:

* How long participants feel the treatment works

* How severe their blepharospasm symptoms are over time

* What side effects or safety concerns occur (adverse events)

Are You a Good Fit for This Trial?

Inclusion Criteria

I am 18 years old or older.
I have blepharospasm that needs regular botulinum toxin injections.
I get botulinum toxin shots for blepharospasm, but they help for less than 12 weeks and my dose can't be increased due to side effects.
See 1 more

Exclusion Criteria

I have a history of serious heart failure or abnormal heart rhythms.
Known hypersensitivity to botulinum toxin or formulation components
Subjects in an investigational drug or device study within the last 30 days prior to Screening
See 14 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Treatment Cycle 1

Participants receive the first cycle of DAXI injections, with follow-up assessments to evaluate peak response and symptom control.

3-6 months
Multiple visits (in-person and remote)

Treatment Cycle 2

Participants receive the second cycle of DAXI injections, with follow-up assessments similar to the first cycle.

3-6 months
Multiple visits (in-person and remote)

Follow-up

Participants are monitored for safety and effectiveness after the final treatment cycle.

3-6 months
Multiple visits (in-person and remote)

What Are the Treatments Tested in This Trial?

Interventions

  • DaxibotulinumtoxinA

How Is the Trial Designed?

1

Treatment groups

Experimental Treatment

Group I: Open label armExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Pennsylvania

Lead Sponsor

Trials
2,118
Recruited
45,270,000+

Revance Therapeutics, Inc.

Industry Sponsor

Trials
32
Recruited
6,600+