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Spasmodic Dysphonia (SD) is a neurologic condition causing inappropriate contraction of the laryngeal musculature, leading to abnormal voicing. The three types (adductor, abductor, and mixed) affect varying muscle groups which produce characteristic voice patterns. The vast majority of patients with SD have adductor type, which impacts the lateral cricoarytenoid and thyroarytenoid muscle complex. While many treatment modalities have been investigated, the most effective treatment is botulinum toxin injection to these muscle groups, performed transcervically with or without electromyography (EMG) guidance. Patients undergoing this treatment typically require re-injection every 3 months. Due to its specialized nature, the laryngeal injections are not performed routinely outside of academic medical centers; thus, patients may come from a distance to receive this treatment. Both due to the significant impact on voice quality when the injections wear off and the sometimes challenging access to treatment, a longer-acting agent is desired.
Injectible daxibotuliumtoxinA (DAXI, Revance Therapeutics Inc., Newark, CA) has been shown in large clinical trials to provide safe, effective treatment for glabellar lines and cervical dystonia and may offer a longer-lasting result when compared with onabotulinumtoxinA.
Thus, a study examining the effect of DAXI for patients with adductor spasmodic dysphonia is proposed. This study aims to assess the efficacy of DAXI for transcervical laryngeal injection in patients with adductor spasmodic dysphonia.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Administration of Daxi | Experimental | Injectible daxibotulinumtoxinA (DAXI, Revance Therapeutics Inc., Newark, CA) is an investigational botulinum toxin type A which has been shown in large clinical trials to provide safe, effective treatment for glabellar lines and cervical dystonia and may offer longer-lasting results when compared with onabotulinumtoxinA. Dosage will be same number of units as prior Botox A dose. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| DaxibotulinumtoxinA | Drug | This is a long-acting alternative to traditional Botox A treatment. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Our Primary Outcome is the Difference in VHI-10 Score at 30 Days After Injection With DAXI. | The Voice Handicap Index-10 (VHI-10) is a patient-reported questionnaire (range 0-40) that measures voice-related handicap, with higher scores indicating greater severity. Difference between baseline and 30 days post DAXI injection scores will be determined using a paired t-test with subjects pre-treatment VHI-10 scores as a control. | 30 days |
| Measure | Description | Time Frame |
|---|---|---|
| A Secondary Outcome Will be Comparing the Duration of Effect of DAXI With Patients' Prior Botox Treatment. | Duration of Voice Benefit (DVB) was determined by calculating the difference of time (in days) between injection date and time the patient reported a significant reduction of benefit that would prompt scheduling a repeat treatment. DVB is routinely calculated for all Adductor-type laryngeal dystonia (AdLD) patients during their return visits by asking "how many days ago did you lose injection benefit such that you felt you needed another injection." This time frame was then subtracted from the number of days between injection dates to calculate DVB. For Botox (BtxA), a mean DVB was calculated by averaging the last three DVB values from successful injections to compare to the Daxi DVB. Clinically significant difference in DVB was a priori determined as > 14 days between values to allow for normal inter-injection variability. |
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Inclusion Criteria:
Exclusion Criteria:
age less than 18
exclusively having other neurologic conditions such as:
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| Name | Affiliation | Role |
|---|---|---|
| Clark Rosen, MD | University of California, San Francisco | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF | San Francisco | California | 94118 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28614091 | Background | Carruthers J, Solish N, Humphrey S, Rosen N, Muhn C, Bertucci V, Swift A, Metelitsa A, Rubio RG, Waugh J, Quiring J, Shears G, Carruthers A. Injectable DaxibotulinumtoxinA for the Treatment of Glabellar Lines: A Phase 2, Randomized, Dose-Ranging, Double-Blind, Multicenter Comparison With OnabotulinumtoxinA and Placebo. Dermatol Surg. 2017 Nov;43(11):1321-1331. doi: 10.1097/DSS.0000000000001206. | |
| 30009213 |
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| ID | Title | Description |
|---|---|---|
| FG000 | Administration of Daxi | Injectible daxibotulinumtoxinA (DAXI, Revance Therapeutics Inc., Newark, CA) is an investigational botulinum toxin type A which has been shown in large clinical trials to provide safe, effective treatment for glabellar lines and cervical dystonia and may offer longer-lasting results when compared with onabotulinumtoxinA. Dosage will be same number of units as prior Botox A dose. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Two patients had no change in their voice and no swallowing impact. These patients were thus considered "misses," or injection events that did not reach the intended TA-LCA muscle complexes and thus were not included in the subsequent analysis.
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| ID | Title | Description |
|---|---|---|
| BG000 | Administration of Daxi | Injectible daxibotulinumtoxinA (DAXI, Revance Therapeutics Inc., Newark, CA) is an investigational botulinum toxin type A which has been shown in large clinical trials to provide safe, effective treatment for glabellar lines and cervical dystonia and may offer longer-lasting results when compared with onabotulinumtoxinA. Dosage will be same number of units as prior Botox A dose. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Our Primary Outcome is the Difference in VHI-10 Score at 30 Days After Injection With DAXI. | The Voice Handicap Index-10 (VHI-10) is a patient-reported questionnaire (range 0-40) that measures voice-related handicap, with higher scores indicating greater severity. Difference between baseline and 30 days post DAXI injection scores will be determined using a paired t-test with subjects pre-treatment VHI-10 scores as a control. | Two patients had no change in their voice and no swallowing impact. These patients were thus considered "misses," or injection events that did not reach the intended TA-LCA muscle complexes and thus were not included in the subsequent analysis. | Posted | Mean | Standard Deviation | units on a scale | 30 days |
|
from enrollment until end of follow-up, up to 6 months
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Administration of Daxi | Injectible daxibotulinumtoxinA (DAXI, Revance Therapeutics Inc., Newark, CA) is an investigational botulinum toxin type A which has been shown in large clinical trials to provide safe, effective treatment for glabellar lines and cervical dystonia and may offer longer-lasting results when compared with onabotulinumtoxinA. Dosage will be same number of units as prior Botox A dose. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clark Rosen | University of California, San Francisco | (415)-885-7700 | clark.rosen@ucsf.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Nov 23, 2022 | Mar 4, 2026 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D014832 | Voice Disorders |
| D055154 | Dysphonia |
| ID | Term |
|---|---|
| D007818 | Laryngeal Diseases |
| D012140 | Respiratory Tract Diseases |
| D010038 | Otorhinolaryngologic Diseases |
| D009461 | Neurologic Manifestations |
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| ID | Term |
|---|---|
| D019274 | Botulinum Toxins, Type A |
| ID | Term |
|---|---|
| D001905 | Botulinum Toxins |
| D008666 | Metalloendopeptidases |
| D010450 | Endopeptidases |
| D010447 | Peptide Hydrolases |
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| Up to 1 year |
| Background |
| Jankovic J, Truong D, Patel AT, Brashear A, Evatt M, Rubio RG, Oh CK, Snyder D, Shears G, Comella C. Injectable DaxibotulinumtoxinA in Cervical Dystonia: A Phase 2 Dose-Escalation Multicenter Study. Mov Disord Clin Pract. 2018 Apr 26;5(3):273-282. doi: 10.1002/mdc3.12613. eCollection 2018 May-Jun. |
| 31609882 | Background | Carruthers JD, Fagien S, Joseph JH, Humphrey SD, Biesman BS, Gallagher CJ, Liu Y, Rubio RG; SAKURA 1 and SAKURA 2 Investigator Group; SAKURA 1 and SAKURA 2 Investigator Group includes the following. DaxibotulinumtoxinA for Injection for the Treatment of Glabellar Lines: Results from Each of Two Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 3 Studies (SAKURA 1 and SAKURA 2). Plast Reconstr Surg. 2020 Jan;145(1):45-58. doi: 10.1097/PRS.0000000000006327. |
| 21658400 | Background | Stone HF, Zhu Z, Thach TQ, Ruegg CL. Characterization of diffusion and duration of action of a new botulinum toxin type A formulation. Toxicon. 2011 Aug;58(2):159-67. doi: 10.1016/j.toxicon.2011.05.012. Epub 2011 May 31. |
| 29655932 | Background | Rumbach A, Aiken P, Novakovic D. RETRACTED: Outcome Measurement in the Treatment of Spasmodic Dysphonia: A Systematic Review of the Literature. J Voice. 2019 Sep;33(5):810.e13-810.e39. doi: 10.1016/j.jvoice.2018.03.011. Epub 2018 Apr 11. |
|
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants | No |
|
| VHI-10 | The Voice Handicap Index-10 (VHI-10) is a patient-reported questionnaire (range 0-40) that measures voice-related handicap, with higher scores indicating greater severity. | Mean | Standard Deviation | units on a scale |
|
|
|
|
| Secondary | A Secondary Outcome Will be Comparing the Duration of Effect of DAXI With Patients' Prior Botox Treatment. | Duration of Voice Benefit (DVB) was determined by calculating the difference of time (in days) between injection date and time the patient reported a significant reduction of benefit that would prompt scheduling a repeat treatment. DVB is routinely calculated for all Adductor-type laryngeal dystonia (AdLD) patients during their return visits by asking "how many days ago did you lose injection benefit such that you felt you needed another injection." This time frame was then subtracted from the number of days between injection dates to calculate DVB. For Botox (BtxA), a mean DVB was calculated by averaging the last three DVB values from successful injections to compare to the Daxi DVB. Clinically significant difference in DVB was a priori determined as > 14 days between values to allow for normal inter-injection variability. | Two patients had no change in their voice and no swallowing impact. These patients were thus considered "misses," or injection events that did not reach the intended TA-LCA muscle complexes and thus were not included in the subsequent analysis. The overall cohort was split into three subgroups based on their Duration of Voice Benefit (DVB) with DAXI and Botox (BtxA) for statistical analysis: Group A (DVB DAXI>BtxA), Group B (DVB DAXI=BtxA), and Group C (DVB DAXI\ | Posted | Mean | Full Range | days | Up to 1 year |
|
|
|
|
| 0 |
| 22 |
| 0 |
| 22 |
| 0 |
| 22 |
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| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D006867 |
| Hydrolases |
| D004798 | Enzymes |
| D045762 | Enzymes and Coenzymes |
| D045726 | Metalloproteases |
| D001426 | Bacterial Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D001427 | Bacterial Toxins |
| D014118 | Toxins, Biological |
| D001685 | Biological Factors |
|
| Group B (DVB DAXI=BtxA) Mean DVB for Daxi |
|
|
| Group B (DVB DAXI=BtxA) Mean DVB for BtxA |
|
|
| Group C (DVB DAXI<BtxA) Mean DVB for DAXI |
|
|
| Group C (DVB DAXI<BtxA) Mean DVB for BtxA |
|
|
| 0.04 |
| Superiority |
| For Group C (DVB for Daxi\ | t-test, 2 sided | 0.001 | Superiority |