Adductor Spasmodic Dysphonia, Spasmodic Dysphonia, Voice Disorders
Conditions
Keywords
adductor spasmodic dysphonia
Brief summary
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.
Interventions
This is a long-acting alternative to traditional Botox A treatment.
Sponsors
University of California, San Francisco
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* greater than 18 * diagnosis of adductor spasmodic dysphonia * previous successful treatment with BotoxA * stabilized dose for last 3 treatments
Exclusion criteria
* age less than 18 * exclusively having other neurologic conditions such as: * abductor spasmodic dysphonia * ALS * Multiple sclerosis * Parkinson's disease * Essential tremor
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Our Primary Outcome is the Difference in VHI-10 Score at 30 Days After Injection With DAXI. | 30 days | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| A Secondary Outcome Will be Comparing the Duration of Effect of DAXI With Patients' Prior Botox Treatment. | Up to 1 year | 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. |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORClark Rosen, MD
University of California, San Francisco
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 58.3 Years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 18 Participants |
| Sex: Female, Male Female | 15 Participants |
| Sex: Female, Male Male | 5 Participants |
| VHI-10 | 21.10 units on a scale STANDARD_DEVIATION 6.82 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 22 |
| other Total, other adverse events | 0 / 22 |
| serious Total, serious adverse events | 0 / 22 |
Outcome results
None listed