Essential TremorNCT02711995
Essential Voice Tremor: A Study of Botulinum Toxin Treatment and Injection Augmentation
A study testing RenuGel and Botulinum Toxin in people with Essential Tremor, run by Weill Medical College of Cornell University at 1 site in United States.
Trial summary
Botulinum toxin is the common treatment of choice for patients with Essential Voice Tremor (EVT), but results are not universally beneficial to all patients. Inconsistent results are noted in the literature and are consistent with the PI's clinical experience. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Essential Tremor
- What is being tested?
- RenuGel and Botulinum Toxin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Dec 2015to 29 Sep 2016
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female patients ages 18-100 who have been diagnosed with essential tremor of the voice (EVT) Diagnosis Code 478.75 Laryngeal Spasm.
Who cannot join
- WCMC subjects under age 18
- Pregnant patients
- Patients with laryngeal pathology besides tremor, including but not limited to polyps and other benign lesions, precancerous or cancerous changes, vocal fold paralysis, or other laryngeal disorders of mobility
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Essential Voice Tremor: An Unblinded Crossover Study of Botulinum Toxin Treatment and Injection Augmentation
Botulinum toxin is the common treatment of choice for patients with Essential Voice Tremor (EVT), but results are not universally beneficial to all patients. Inconsistent results are noted in the literature and are consistent with the PI's clinical experience. Injection augmentation, a well-established treatment for glottic insufficiency, which is a prominent factor in the clinical presentation of Essential Voice Tremor (EVT), has not been studied. By treating patients at separate times with botulinum toxin and injection augmentation in an unblinded prospective crossover treatment study, we can assess functional outcomes of these two treatments with the population of patients with Essential Voice Tremor (EVT).
Read the detailed description
The purpose of this study is to compare treatment with botulinum toxin to treatment with injection augmentation in patients diagnosed with essential voice tremor.
Botulinum toxin treatment is the injection of botulinum toxin into the muscles of the vocal cords through the skin of the neck. This is identical to the injections you may have received in the past for your disorder.
Injection augmentation is the injection of a filler material into the vocal cords through the skin of the neck, guided by the view from a flexible laryngoscope inserted through the nostril. The flexible laryngoscopy is identical to the procedure that the doctor has used to examine your vocal cords in the past. It is the routine diagnostic evaluation technique of voice disorders.
Periodic injections with botulinum toxin (Botox) is the current standard of care for patients diagnosed with essential voice tremor, but studies show only about 60% of patients benefit substantially enough to continue treatment. Poor vocal fold closure, a common characteristic of essential voice tremor, is treated in other situations (such as vocal fold paralysis or paresis) with injection augmentation; thus there is reason to think that it may be helpful to patients with essential voice tremor, perhaps even to a greater extent than botulinum toxin.
- Conditions
- Essential Tremor
- Being tested
- RenuGel, Botulinum Toxin
- Keywords
- botulinum toxin, injection augmentation, essential voice tremor
- Medical subject headings
- Essential Tremor
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RenuGelDrug
Also called generic: Carboxymethylcellulose
Injection augmentation is the injection of a filler material (Brand: RenuGel; generic: Carboxymethylcellulose) into the vocal cords through the skin of the neck, guided by the view from a flexible laryngoscope inserted through the nostril. The flexible laryngoscopy is identical to the procedure that the doctor has used to examine your vocal cords in the past. It is the routine diagnostic evaluation technique of voice disorders.
Botulinum ToxinDrug
Also called Botox
Botulinum toxin treatment is the injection of botulinum toxin into the muscles of the vocal cords through the skin of the neck. This is identical to the injections you may have received in the past for your disorder.
| Study group | Role | What they receive |
|---|---|---|
| RenuGel | Experimental | Injection augmentation is the injection of a filler material (Brand: RenuGel; generic: Carboxymethylcellulose) into the vocal cords through the skin of the neck, guided by the view from a flexible laryngoscope inserted through the nostril. The flexible laryngoscopy is identical to the procedure that the doctor has used to examine your vocal cords in the past. It is the routine diagnostic evaluation technique of voice disorders. Receives: Drug: RenuGel |
| Botulinum toxin | Active comparator | Botulinum toxin treatment is the injection of botulinum toxin into the muscles of the vocal cords through the skin of the neck. This is identical to the injections you may have received in the past for your disorder. Receives: Drug: Botulinum Toxin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Vocal Tremor Scoring System (VTSS)
The Vocal Tremor Scoring System (VTSS) was developed to standardize the evaluation and scaling of vocal tremor. Tremor at a specific site was scored according to severity by the laryngologist. It can be rated as: none (0), mild/intermittent (1), moderate (2), severe (3). Six different regions were evaluated in this study: base of tongue, larynx, palate, pharyngeal walls, supraglottis, and true vocal folds. The scale range for each region was 0-3. The total score was a summation of all six regions, with a scalar range of 0-18.
Measured: Baseline and 30 days after intervention
Secondary outcomes (8)
Acoustic Analysis- Frequencies
For acoustic assessment, subjects produced a sustained /a/ sound at their habitual speaking pitch and loudness and read assessment sentences from the Consensus Audio-Perceptual Evaluation of Voice (CAPE-V)protocol. Tasks were recorded and analyzed using the Analysis of Dysphonia in Speech and Voice (ADSV) and Multi-Dimensional Voice Profile (MDVP) software. A handheld microphone 3 inches from the subjects' mouths was used for all recordings.
The Sustained Vowel and All-Voiced Sentence protocols of the ADSV were used to obtain cepstral peak prominence fundamental frequency (CPP F0), The MDVP was used to obtain amplitude tremor frequency (Fatr), and fundamental frequency tremor frequency (Fftr).
Measured: Baseline and 30 days after intervention
Aerodynamic Data- Airflow
Aerodynamic data were collected using the Phonatory Aerodynamic System (PAS) 6600 (Pentax). Subjects held a facemask coupled to a pneumotachometer with a pressure-sensor tube firmly over the nose and mouth, and rested the pressure-sensor tube in the oral cavity above the tongue. They produced sustained /a/ and "We were away a year ago," from which mean airflow wasanalyzed via the Maximum Sustained Phonation and Running Speech protocols.
Measured: Baseline and 30 days after intervention
Consensus Auditory-Perceptual Evaluation of Voice (CAPE-V)
The Consensus Auditory-Perceptual Evaluation of Voice (CAPE-V) is used to describe the severity of auditory-perceptual attributes of a voice problem. It indicates salient perceptual vocal attributes: (a) Overall Severity; (b) Roughness; (c) Breathiness; (d) Strain; (e) Pitch; and (f) Loudness. The CAPE-V displays each attribute accompanied by a 100- millimeter line forming a visual analog scale (VAS). The clinician indicates the degree of perceived deviance from normal for each parameter on this scale, using a tic mark. For each dimension, scalar extremes are unlabeled.
The scale range is from 0mm to 100mm. Results can indicate distance in mm to describe the degree of deviancy, so the higher the score the more deviancy from the norm there is.
Measured: Baseline and 30 days after intervention
Voice Handicap Index-10 (VHI-10)
The Voice Handicap Index-10 consists of 10 questions (statements about voice), where patients rate their the frequency of their problems as: never (0), almost never (1), sometimes (2), almost always (3), and always (4). The scores from each answer are added, and can range from 0-40. The higher the score, the worse the patient's perception of their voice handicap.
Measured: Baseline and 30 days after intervention
Percent of Normal Function (PNF)
The Percent of Normal Function (PNF) is a scale for patients to rate their recurrent functions in increments of five, from no function (0%) to normal function (100%). The higher the percentage, the more normal the function as experienced by the patient.
Measured: Baseline and 30 days after intervention
Aerodynamic Data- Peak Air Pressure
Aerodynamic data were collected using the Phonatory Aerodynamic System (PAS) 6600 (Pentax). Subjects held a facemask coupled to a pneumotachometer with a pressure-sensor tube firmly over the nose and mouth, and rested the pressure-sensor tube in the oral cavity above the tongue. A string of five consonant-vowel syllables (/pa/) at a comfortable pitch and loudness were analyzed through the Voicing Efficiency protocol to determine mean peak air pressure.
Measured: Baseline and 30 days after intervention
Aerodynamic Data- Loudness
Aerodynamic data were collected using the Phonatory Aerodynamic System (PAS) 6600 (Pentax). Subjects held a facemask coupled to a pneumotachometer with a pressure-sensor tube firmly over the nose and mouth, and rested the pressure-sensor tube in the oral cavity above the tongue. They produced sustained /a/ and "We were away a year ago," from which loudness was analyzed via the Maximum Sustained Phonation and Running Speech protocols.
Measured: Baseline and 30 days after intervention
Aerodynamic Data- Maximum Phonation Time
Aerodynamic data were collected using the Phonatory Aerodynamic System (PAS) 6600 (Pentax). Subjects held a facemask coupled to a pneumotachometer with a pressure-sensor tube firmly over the nose and mouth, and rested the pressure-sensor tube in the oral cavity above the tongue. They produced sustained /a/ from which Maximum Sustained Phonation time was recorded.
Measured: Baseline and 30 days after intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2016 (estimated)
- Study start15 Dec 2015
- Primary completion29 Sep 2016
- Study completion29 Sep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Essential TremorStudies of the same condition, open and under way.
- More trials run by Weill Medical College of Cornell UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Weill Medical College of Cornell University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lucian Sulica, MD, Principal investigator, Weill Medical College of Cornell University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02711995
- Sponsor's protocol ID
- 1505016165
- Status last verified
- Jan 2018
- First submitted
- 24 Feb 2016
- Last updated
- 5 Feb 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Essential Voice Tremor: An Unblinded Crossover Study of Botulinum Toxin Treatment and Injection Augmentation
- ClinicalTrials.gov ID
- NCT02711995
- Acronym / sponsor ID
- 1505016165
- Conditions
- Essential Tremor
- Keywords
- botulinum toxin, injection augmentation, essential voice tremor
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Study start
- 15 Dec 2015
- Primary completion
- 29 Sep 2016 (actual)
- Study completion
- 29 Sep 2016 (actual)
- First posted
- 17 Mar 2016 (estimated)
- Results
- Posted 10 Jan 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Botulinum toxin is the common treatment of choice for patients with Essential Voice Tremor (EVT), but results are not universally beneficial to all patients. Inconsistent results are noted in the literature and are consistent with the PI's clinical experience. Injection augmentation, a well-established treatment for glottic insufficiency, which is a prominent factor in the clinical presentation of Essential Voice Tremor (EVT), has not been studied. By treating patients at separate times with botulinum toxin and injection augmentation in an unblinded prospective crossover treatment study, we can assess functional outcomes of these two treatments with the population of patients with Essential Voice Tremor (EVT).
Detailed description
The purpose of this study is to compare treatment with botulinum toxin to treatment with injection augmentation in patients diagnosed with essential voice tremor.
Botulinum toxin treatment is the injection of botulinum toxin into the muscles of the vocal cords through the skin of the neck. This is identical to the injections you may have received in the past for your disorder.
Injection augmentation is the injection of a filler material into the vocal cords through the skin of the neck, guided by the view from a flexible laryngoscope inserted through the nostril. The flexible laryngoscopy is identical to the procedure that the doctor has used to examine your vocal cords in the past. It is the routine diagnostic evaluation technique of voice disorders.
Periodic injections with botulinum toxin (Botox) is the current standard of care for patients diagnosed with essential voice tremor, but studies show only about 60% of patients benefit substantially enough to continue treatment. Poor vocal fold closure, a common characteristic of essential voice tremor, is treated in other situations (such as vocal fold paralysis or paresis) with injection augmentation; thus there is reason to think that it may be helpful to patients with essential voice tremor, perhaps even to a greater extent than botulinum toxin.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| RenuGel | Experimental | Drug: RenuGelInjection augmentation is the injection of a filler material (Brand: RenuGel; generic: Carboxymethylcellulose) into the vocal cords through the skin of the neck, guided by the view from a flexible laryngoscope inserted through the nostril. The flexible laryngoscopy is identical to the procedure that the doctor has used to examine your vocal cords in the past. It is the routine diagnostic evaluation technique of voice disorders. |
| Botulinum toxin | Active comparator | Drug: Botulinum ToxinBotulinum toxin treatment is the injection of botulinum toxin into the muscles of the vocal cords through the skin of the neck. This is identical to the injections you may have received in the past for your disorder. |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Male and female patients ages 18-100 who have been diagnosed with essential tremor of the voice (EVT) Diagnosis Code 478.75 Laryngeal Spasm.
Key exclusion criteria (3 listed)
- WCMC subjects under age 18
- Pregnant patients
- Patients with laryngeal pathology besides tremor, including but not limited to polyps and other benign lesions, precancerous or cancerous changes, vocal fold paralysis, or other laryngeal disorders of mobility
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RenuGelAlso: generic: CarboxymethylcelluloseInjection augmentation is the injection of a filler material (Brand: RenuGel; generic: Carboxymethylcellulose) into the vocal cords through the skin of the neck, guided by the view from a flexible laryngoscope inserted through the nostril. The flexible laryngoscopy is identical to the procedure that the doctor has used to examine your vocal cords in the past. It is the routine diagnostic evaluation technique of voice disorders. | Drug | RenuGel |
| Botulinum ToxinAlso: BotoxBotulinum toxin treatment is the injection of botulinum toxin into the muscles of the vocal cords through the skin of the neck. This is identical to the injections you may have received in the past for your disorder. | Drug | Botulinum toxin |
Primary outcome
Vocal Tremor Scoring System (VTSS)
Time frame: Baseline and 30 days after intervention
The Vocal Tremor Scoring System (VTSS) was developed to standardize the evaluation and scaling of vocal tremor. Tremor at a specific site was scored according to severity by the laryngologist. It can be rated as: none (0), mild/intermittent (1), moderate (2), severe (3). Six different regions were evaluated in this study: base of tongue, larynx, palate, pharyngeal walls, supraglottis, and true vocal folds. The scale range for each region was 0-3. The total score was a summation of all six regions, with a scalar range of 0-18.
8 secondary outcomes
Acoustic Analysis- Frequencies
Time frame: Baseline and 30 days after intervention
Aerodynamic Data- Airflow
Time frame: Baseline and 30 days after intervention
Consensus Auditory-Perceptual Evaluation of Voice (CAPE-V)
Time frame: Baseline and 30 days after intervention
Voice Handicap Index-10 (VHI-10)
Time frame: Baseline and 30 days after intervention
Percent of Normal Function (PNF)
Time frame: Baseline and 30 days after intervention
Aerodynamic Data- Peak Air Pressure
Time frame: Baseline and 30 days after intervention
Aerodynamic Data- Loudness
Time frame: Baseline and 30 days after intervention
Aerodynamic Data- Maximum Phonation Time
Time frame: Baseline and 30 days after intervention
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lucian Sulica, MD principal investigator
Weill Medical College of Cornell University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing RenuGel and Botulinum Toxin in people with Essential Tremor, run by Weill Medical College of Cornell University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Feb 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Jan 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Vocal Tremor Scoring System (VTSS) (time frame: Baseline and 30 days after intervention). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2018.