A Phase 2 interventional study of Botulinum Neurotoxin Type A in Blepharospasm of Both Eyelids, Blepharospasm of Left Eyelid and Blepharospasm of Right Eyelid, sponsored by University of Pennsylvania. Recruiting at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-29.
Sponsored by University of Pennsylvania · Phase 2, Interventional, and Treatment
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 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:
This study is an open-label, single-center, single-arm, longitudinal clinical trial designed to evaluate the use of daxibotulinumtoxinA (DAXI) for injection in twenty adult participants with blepharospasm (BSP). Each subject will be eligible to receive between two and three treatment cycles with DAXI over a twelve-month period. The timing of reinjection will depend on each participant's duration of benefit, as determined using the Blepharospasm Severity Tracker Form (BSTF). Treatments must be spaced at least ninety days apart but not more than one hundred eighty days. Participants will decide when to receive another injection and may use topical lidocaine cream, cooling spray, or other anesthetic methods to reduce pain during the procedure.
The study will enroll adults of any race or gender, aged eighteen or older, who are in generally good health and have a clinical diagnosis of blepharospasm. Eligible participants may have either isolated blepharospasm, which could be focal or part of segmental or generalized dystonia, or blepharospasm as a complication of a parkinsonian condition. To qualify, participants must have a baseline blepharospasm severity rating of at least two points on the Blepharospasm Severity Rating Scale (BSRS) and must already be receiving either onabotulinumtoxinA or incobotulinumtoxinA with less than twelve weeks of benefit from that treatment.
For each injection session, the study drug will be prepared by the injector or a qualified staff member by diluting one milliliter of Bacteriostatic Sodium Chloride Injection (0.9%) with one hundred units of DAXI to create a final concentration of ten units per 0.1 milliliter for intramuscular injection. The specific dose, muscles injected, and number of injection sites will be determined by the injector. Only four muscles may be treated: the corrugators, procerus, orbicularis oculi, and nasalis. Use of electromyography or ultrasonography for guidance is optional but generally unnecessary for facial injections.
The initial DAXI dose will be based on the participant's previous injection cycle. Those currently treated with onabotulinumtoxinA will convert on a one-to-one unit basis, while those treated with incobotulinumtoxinA will use a 1.5:1 conversion ratio, meaning 1.5 units of incobotulinumtoxinA is equivalent to one unit of DAXI. If the previous dose resulted in excessive weakness or related side effects such as eyelid drooping, double vision, or dry eye, dosing will be adjusted accordingly. Subsequent DAXI dosing may be further modified at the injector's discretion, and efforts will be made for each subject to be treated by the same injector throughout the study.
Following each treatment, efficacy will be evaluated at the time of peak response, which typically occurs about one month after injection. The first DAXI administration occurs around day ninety-five (visit three), and this timing determines when follow-up evaluations, usually by remote visit, are performed. Future injection cycles will follow similar timing, although exact intervals will vary depending on individual responses. The overall study period for each subject spans roughly one year, ending with either an end-of-study or early-termination visit between days 365 and 460.
The primary measure of efficacy is the median time to reinjection, serving as an indicator of the duration of DAXI's therapeutic effect. Secondary outcomes include the participant's perceived duration of benefit, changes in blepharospasm severity, quality-of-life improvements, and overall treatment satisfaction. Specific evaluation tools include the Blepharospasm Severity Tracking Form (BSTF), Blepharospasm Severity Rating Scale (BSRS), Craniocervical Dystonia Questionnaire (CDQ-24), Clinical Global Impression of Change (CGIC), Patient Global Impression of Change (PGIC), and a Treatment Satisfaction Questionnaire.
Safety monitoring is a critical component of the trial. Assessments will include pregnancy testing for women of childbearing potential, physical and neurological examinations, the Columbia-Suicide Severity Rating Scale (C-SSRS), vital signs, and inspection of injection sites. Investigators will also record concomitant medications and monitor all adverse events, including any deemed to be of special interest.
42 studies on the registry are indexed under Blepharospasm; 1 is open to participants now.
This study's planned enrollment of 20 is below the median of 40 across 28 interventional studies indexed under Blepharospasm.
Browse Blepharospasm studies →University of Pennsylvania is the lead sponsor of 1,668 studies on the registry; 235 are open to participants now.
Of its 158 completed or terminated interventional studies of FDA-regulated products, 109 (69%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Chronic obstructive pulmonary disease: long-term lung disease that makes it hard to breathe out, including emphysema and chronic bronchitis.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
COPD
Chronic obstructive pulmonary disease: long-term lung disease that makes it hard to breathe out, including emphysema and chronic bronchitis.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
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.
Also known as: daxibotulinumtoxinA, DAXI
First time to retreatment
Time to retreatment (in days) with DAXI between the first and second study injection cycle.
Time frame: Outcome will be assessed during Visit 5 (2nd study injection visit) and reported at study conclusion. Visit 5 will take place for each individual subject between days 180 and 280.
Final time to retreatment
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).
Time frame: Outcome will be assessed during Visit 7 (3rd study injection visit) and reported at study conclusion. Visit 7 will take place for each individual study subject between days 270 and 280.
Difference in time to return of symptoms after first DAXI cycle
Change in time (in days) to return of baseline blepharospasm symptoms, as documented in the Blepharospasm Severity Tracker Form between data relating to the last clinical injection cycle prior to screening and the first DAXI study injection cycle.
Time frame: Outcome will be assessed during Visit 3 (1st study injection visit) and reported at study conclusion. Visit 3 will take place for each individual study subject between days 90 and 100.
Difference in time to return of symptoms after final DAXI cycle
Change in time (in days) to return of baseline BSP symptoms, as documented in the Blepharospasm Severity Tracker Form between data relating to the last clinical injection cycle prior to screening and the last DAXI study injection cycle.
Time frame: Outcome will be assessed during Visit 5 (days 180-280) for subjects only receiving 2 injections during study participation, and during Visit 7 (days 270-280) for subjects receiving 3. Outcomes will be reported at study conclusion.
Change in severity after first DAXI injection cycle
Change in the Blepharospasm Severity Rating Scale (BSRS) total score between Visit 2 (peak efficacy assessment of last clinical injection) and Visit 4 (peak efficacy assessment of first DAXI injection). The BSRS is a validated scale with scores ranging from 0 to 18, with higher numbers reflecting worsening blepharospasm severity.
Time frame: Outcome will be assessed during Visit 4 (1st study injection peak effect assessment) and reported at study conclusion. Visit 4 will take place for each individual study subject between days 114 and 156.
Change in severity after final DAXI injection cycle
Change in Blepharospasm Severity Rating Scale (BSRS) total score between Visit 2 (the peak efficacy assessment of the last clinical injection) and the peak efficacy assessment following the subject's final DAXI study injection (which may occur during either the second or third injection cycle, depending on how long the individual subject chooses to wait before reinjection with DAXI). The BSRS is a validated scale ranging from 0 to 18, with higher scores indicating greater blepharospasm severity.
Time frame: BSRS will be assessed during Visit 6 (days 200-340) for subjects only receiving 2 injections during study participation, and during Visit 8 (days 294-336) for subjects receiving 3. Outcomes will be reported at study conclusion.
First change in the Patient Global Impression of Change (PGIC)
Change in Patient Global Impression of Change (PGIC) score between V2 (peak efficacy assessment of last clinical injection) and V4 (peak efficacy assessment of first study DAXI injection). The PGIC is a patient-reported outcome that acts as a 7-point Likert scale, with values ranging from -3 (very much worse) to +3 (very much better).
Time frame: Outcome will be assessed during Visit 4 (peak efficacy assessment of first study DAXI injection) taking place on days 114-156, and reported at study conclusion.
Final change in the Patient Global Impression of Change (PGIC)
Change in Patient Global Impression of Change (PGIC) score between V2 (peak efficacy assessment of last clinical injection) and during the peak efficacy assessment of the study subject's final DAXI study injection (which may occur during either the second or third injection cycle, depending on how long the individual subject chooses to wait before reinjection with DAXI). The PGIC is a patient-reported outcome that acts as a 7-point Likert scale, with values ranging from -3 (very much worse) to +3 (very much better).
Time frame: PGIC will be assessed during Visit 6 (days 200-340) for subjects only receiving 2 injections during study participation, and during Visit 8 (days 294-336) for subjects receiving 3. Outcomes will be reported at study conclusion.
First change in the Clinical Global Impression of Change (CGIC)
Change in Clinical Global Impression of Change (CGIC) score between V2 (peak efficacy assessment of last clinical injection) and V4 (peak efficacy assessment of first DAXI injection). The CGIC is a validated clinical outcome measure that acts as a 7-point Likert scale, with values ranging from -3 (very much worse) to +3 (very much better).
Time frame: Outcome will be assessed during Visit 4 (peak efficacy assessment of first study DAXI injection) taking place on days 114-156, and reported at study conclusion.
Final change in the Clinical Global Impression of Change (CGIC)
Change in Clinical Global Impression of Change (CGIC) score between V2 (peak efficacy assessment of last clinical injection) and during the peak efficacy assessment of the study subject's final DAXI study injection (which may occur during either the second or third injection cycle, depending on how long the individual subject chooses to wait before reinjection with DAXI). The CGIC is a validated clinical outcome measure that acts as a 7-point Likert scale, with values ranging from -3 (very much worse) to +3 (very much better).
Time frame: CGIC will be assessed during Visit 6 (days 200-340) for subjects only receiving 2 injections during study participation, and during Visit 8 (days 294-336) for subjects receiving 3. Outcomes will be reported at study conclusion.
First change in satisfaction
Change in Treatment Satisfaction Questionnaire score between V3 (day of first DAXI study injection) and V5 (day of second DAXI study injection). The Treatment Satisfaction Questionnaire is a patient-reported outcome that acts as a 7-point Likert scale, with values ranging from -3 (very dissatisfied) to +3 (very satisfied).
Time frame: Outcome will be assessed during Visit 5 (days 180-280). Outcomes will be reported at study conclusion.
Final change in satisfaction
Change in Treatment Satisfaction Questionnaire (TSQ) score between V3 (day of first DAXI study injection) and EOS/ET (last day of study). The Treatment Satisfaction Questionnaire is a patient-reported outcome that acts as a 7-point Likert scale, with values ranging from -3 (very dissatisfied) to +3 (very satisfied).
Time frame: Outcome will be assessed during Visit 7 (days 270-280) and Visit 9 (days 365-460). Outcomes will be reported at study conclusion.
Incidence of Treatment-Emergent Adverse Events (suicidality, toxin-related side effects and injection site reactions)
Safety, which includes screening for suicidality (C-SSRS) and adverse events (AEs), particularly adverse events of special interest: diplopia (or other visual disturbances), dry eyes, ptosis, injection site reactions and toxin effect spread to the lower face.
Time frame: Outcomes will be assessed during Visit 1 (day 0), Visit 2 (day 30 +/-5), Visit 3 (day 95 +/-5), Visit 4 (day 135 +/- 21), Visit 5 (day 180-280), Visit 6 (day 200-340), Visit 7 (day 275 +/-5), Visit 8 (day 315 +/- 21), Visit 9 (day 365-460).
First change in craniocervical dystonia questionnaire
Change in Craniocervical dystonia questionnaire (CDQ-24) total score between V2 (peak efficacy assessment of last clinical injection) and V4 (peak efficacy assessment of first DAXI injection). The CDQ-24 is a validated scale that tracks patient-reported outcomes, with scores ranging from 0 to 96. Higher scores indicate worsening dystonia symptoms.
Time frame: Outcome will be assessed during Visit 4 (1st study injection peak effect assessment) and reported at study conclusion. Visit 4 will take place for each individual study subject between days 114 and 156.
Final change in craniocervical questionnaire
Change in Craniocervical dystonia questionnaire (CDQ-24) total score between V2 (peak efficacy assessment of last clinical injection) and the peak efficacy assessment following the subject's final DAXI study injection (which may occur during either the second or third injection cycle, depending on how long the individual subject chooses to wait before reinjection with DAXI). The CDQ-24 is a validated scale that tracks patient-reported outcomes, with scores ranging from 0 to 96. Higher scores indicate worsening dystonia symptoms.
Time frame: Outcome will be assessed during Visit 6 (days 200-340) for subjects only receiving 2 injections during study participation, and during Visit 8 (days 294-336) for subjects receiving 3. Outcomes will be reported at study conclusion.
Average total DAXI doses at first and final study cycle
Average total dose in units (with standard deviation) of daxibotulinumtoxinA for each of the different facial muscles injected during the first and last study injection cycle.
Time frame: Outcome will be assessed during Visit 5 (days 180-280) for subjects only receiving 2 injections during study participation, and during Visit 7 (days 270-280) for subjects receiving 3. Outcomes will be reported at study conclusion.
Plan to share: No — This is an investigator-initiated trial (IIT), and I will be the sponsor and sole investigator responsible for and privy to all trial data.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
No contact was published for this record. The registry link below has the sponsor’s details.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Botulinum toxin type A