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NOT YET RECRUITING

NCT07489430

PHASE2

DaxibotulinumtoxinA for Blepharospasm

Sponsor: University of Pennsylvania

View on ClinicalTrials.gov

Summary

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)

Official title: BLEXI: A Study of BLEpharospasm Management With daXIbotulinumtoxinA

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

Start Date

2026-07-01

Completion Date

2028-12-31

Last Updated

2026-03-24

Healthy Volunteers

Conditions

Blepharospasm of Both Eyelids Blepharospasm of Left Eyelid Blepharospasm of Right Eyelid Blepharospasm, Benign Essential Blepharospasm

Interventions

DRUG

Botulinum Neurotoxin Type A

DaxibotulinumtoxinA (DAXI) for injection is a sterile, lyophilized powder containing 100 units of active daxibotulinumtoxinA per vial, along with inactive ingredients including RTP004, trehalose dihydrate, L-histidine, L-histidine hydrochloride, and polysorbate 20. It is reconstituted with Bacteriostatic Sodium Chloride Injection 0.9% (Pfizer) and stored at 2-8°C. DAXI will be prepared by trained injectors or staff, with all preparations documented. Experienced movement disorder neurologists will administer injections, primarily from UPenn's Parkinson's Disease and Movement Disorders Center. Dosing will follow prior treatment patterns using a 1:1 conversion from onabotulinumtoxinA or 1.5:1 from incobotulinumtoxinA, adjusted for prior adverse effects. Injection sites will be selected based on clinical presentation, with optional EMG or imaging guidance.

Inclusion Criteria: * Adults (18 years of age, or older) * Clinical diagnosis of Blepharospasm that is disruptive enough to warrant ongoing clinical botulinum toxin injections and scoring ≥2 on the BSRS during screening. Blepharospasm may be focal (i.e. Benign essential blepharospasm), associated with non-drug-induced secondary cause (e.g., typical or atypical parkinsonism) or as part of a diagnosis of segmental or generalized dystonia involving the upper cranial region, if blepharospasm is the only indication requiring botulinum toxin injections. * Currently receiving onabotulinumtoxinA or incobotulinumtoxinA for the treatment of blepharospasm with: 1) documented duration of benefit \<12 weeks from most recent injection; and 2) no further dose escalation possible or advised due to side effect risk (e.g., ptosis, diplopia, etc.), as determined by treating neurologist; and 3) ability to provide informed consent and comply with study procedures, or subject has a reliable caregiver/proxy. * Written informed consent including authorization to release health information. Exclusion Criteria: * Tardive or medication-induced blepharospasm, as well as psychogenic/functional blepharospasm. * Active ocular pathology interfering with evaluation or injection safety. * Neuromuscular junction or motor neuron disorders (e.g., myasthenia gravis, Lambert-Eaton Myasthenic Syndrome, Amyotrophic Lateral Sclerosis, etc.). * Known hypersensitivity to botulinum toxin or formulation components. * Pregnant or breastfeeding women (including those who are found to be pregnant after a positive pregnancy test at Screening), women who are hoping to get pregnant over the upcoming 15 months, or women of childbearing potential who are unwilling to use contraception while enrolled in the BLEXI study. * Any signal suggestive of active suicidality, as per C-SSRS at screening. * Subjects who have undergone Deep Brain Stimulation (DBS) surgery for the management of blepharospasm and are not willing to keep the DBS settings unchanged for the duration of the trial. * History of primary or secondary non-response to BoNT-A injections, particularly those known to have neutralizing antibodies to BoNT-A. * Subjects on oral medications for dystonia (e.g., anticholinergics, muscle relaxants, benzodiazepines, dopamine depleter) who have not been stable on their regimen for at least 4 weeks prior to Screening, or who are not willing to keep them stable for the duration of the trial. * Use of aminoglycoside antibiotics, polymyxins, lincosamides (e.g., clindamycin), or other agents that might interfere with neuromuscular transmission (e.g., curare-like drugs, quinidine, magnesium sulfate, anticholinesterases, succinylcholine chloride) within 14 days prior to Screening. * History of severe (stage 3) chronic obstructive pulmonary disease, or unstable pulmonary disease within 30 days prior to Screening. * History of chronic or recurrent hypokalemia. * History of congestive heart failure (New York Heart Association Class III or IV), Torsade de Pointe (TdP), and/or Long QT Syndrome. * Subjects in an investigational drug or device study within the last 30 days prior to Screening. * Active skin infections at the injection sites which would put the subject at increased risk of morbidity with BoNT injections * History of blood coagulation disorder that makes facial injection an absolute contraindication * Any acute illnesses or medical conditions including cognitive impairment (e.g., dementia), significant psychiatric illnesses (e.g., major depressive or bipolar disorder) or symptoms (e.g., suicidal ideation, psychosis) that are not stable, and in the Investigator's opinion, could put the subject at increased risk of morbidity, confound the study results, or interfere significantly with the subject's participation in the study.

Locations (1)

Parkinson Disease and Movement Disorders Center at the University of Pennsylvania

Philadelphia, Pennsylvania, United States