Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07489430 | DaxibotulinumtoxinA for Blepharospasm | PHASE2 | NOT YET_RECRUITING | 20 | — | — | Jul 1, 2026 | Dec 31, 2028 | Mar 24, 2026 | 1 | United States |
Time to retreatment (in days) with DAXI between the first and second study injection cycle.
For patients who receive 3 study injection cycles with DAXI over 12 months, a second primary endpoint will be the time to retreatment (in days) between the second-to-last and last injection cycle (i.e., between DAXI cycles 2 and 3).
| Arm | Type | Description |
|---|---|---|
| Open label arm | EXPERIMENTAL | The study cohort will consist of adults with blepharospasm who previously received onabotulinumtoxinA or incobotulinumtoxinA and will transition to DAXI 12 weeks after their last botulinum toxin treatment. Each participant will receive a DAXI dose equivalent to their prior therapy, using a 1:1 conversion for onabotulinumtoxinA or a 1.5:1 conversion for incobotulinumtoxinA, adjusted if prior treatment caused excessive weakness or other side effects. Injections will be limited to muscles directly involved in blepharospasm (the orbicularis oculi, corrugators, procerus, and nasalis) and will use a dilution of 10 units per 0.1 mL. Subjects may use topical anesthetics to reduce injection pain, and efforts will ensure the same injector administers all treatments to maintain consistency. |
| Name | Type | Description |
|---|---|---|
| Botulinum Neurotoxin Type A | DRUG | 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 w...