Essential Voice TremorPhase 1/2NCT01864525
Effects of Octanoic Acid for Treatment of Essential Voice Tremor
A Phase 1/2 study testing Octanoic acid and Inactive capsule in people with Essential Voice Tremor and Vocal Tremor, run by Syracuse University at 1 site in United States.
Trial summary
Essential voice tremor is a neurological condition that produces a regular, shaking quality in the voice. One form of drug treatment that produces some improvement in tremor of the hands is octanoic acid, which is a food additive that is similar to alcohol. Read more about this study
- Who is it for?
- Ages 21 years and older
- Conditions the study is for
- Essential Voice Tremor, Vocal Tremor, Voice Tremor
- What is being tested?
- Octanoic acid and Inactive capsule
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 17 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2013to 31 Mar 2017
- Phase
- Phase 1/2
- 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 21 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants have a diagnosis of essential voice tremor and show signs of tremor during the endoscopy examination (when pictures of the voice box are obtained)during screening appointment
- Participants show measurable voice tremor from recordings of the voice during screening appointment
Who cannot join
- Participants have a diagnosis or show signs of Parkinson's Disease or another non-essential tremor movement disorder
- Participants have a diagnosis or show signs of spasmodic dysphonia (a different neurological voice disorder)
- Participants have a diagnosis of a severe, non-stable medical condition, such as kidney or liver failure, severe heart disease, severe lung disease, severe metabolic disease, uncontrolled hyperthyroidism, or other life-threatening disease such as active cancer
- Participants have a diagnosis of diabetes mellitus
- Participants are unable to suspend/stop a medication that they are currently taking for tremor or voice disorder for 12 weeks to complete this study
- Participants have a dependence on alcohol or allergy to alcohol
Show all 9Show fewer
- Participants are pregnant or lactating
- Participants have an allergy to soy
- Participants have Irritable Bowel Syndrome
Trial locations and contacts
1 site in 1 country.
United States1 site
Syracuse University & Upstate Medical University
Syracuse, New York, 13210
About this study
Essential voice tremor is a neurological condition that produces a regular, shaking quality in the voice. One form of drug treatment that produces some improvement in tremor of the hands is octanoic acid, which is a food additive that is similar to alcohol. Research suggests that octanoic acid may reduce tremor in the hands/arms with few side effects and no intoxication effects. This study will determine whether octanoic acid may be useful for reducing tremor when it affects the voice. Researchers are hypothesizing that octanoic acid will reduce the effects of tremor on the voice.
Read the detailed description
Background:
- Essential tremor of the voice produces regular shaking and hoarseness in the voice, making it difficult speech difficult to understand
- Several previous studies have found that octanoic acid and octanol, which are related to alcohol, can improve tremor in some people without producing many side effects and without producing intoxication
- Researchers are interested in determining whether octanoic acid can improve tremor that affects the voice
Objectives:
- To determine the effects of octanoic voice using voice recordings and listener ratings of voice
- To determine the effects of octanoic acid on level of voice disability experienced by people with essential voice tremor
- Being tested
- Octanoic acid, Inactive capsule
- Keywords
- voice, tremor, essential tremor, essential voice tremor, voice treatment, octanoic acid, octanol, voice disorder, dysphonia
- Medical subject headings
- Tremor, Essential Tremor, Voice Disorders, Dysphonia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Octanoic acidDrug
Also called Caprylic acid
Inactive capsuleDrug
Also called Placebo
| Study group | Role | What they receive |
|---|---|---|
| Inactive capsule | Placebo comparator | Participants will receive a pill/capsule with an inactive ingredient during the placebo arm of this study. Receives: Drug: Inactive capsule |
| Octanoic acid | Experimental | Participants will receive a pill/capsule with octanoic acid (amount determined by the participant's weight) during the experimental arm of this study. Receives: Drug: Octanoic acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Magnitude of Acoustic Amplitude Tremor and Magnitude of Acoustic Frequency Tremor
Voice recordings were used to measure the degree of tremor in the voice. Mean post-test values for each acoustic measure were compared after the octanoic acid and placebo conditions, with and without consideration of baseline values. Mean values represent the average of two testing days. Degree of amplitude tremor shows the extent of amplitude variation as a percent of the mean signal amplitude, with lower numbers indicating less amplitude tremor. Baseline values for magnitude of amplitude tremor across all participants and conditions ranged from 4.06 to 27.09, and post-test values ranged from 1.94 to 26.02. Degree of frequency tremor shows the extent of fundamental frequency variation as a percent of the mean signal frequency, with lower numbers indicating less frequency tremor. Baseline values for magnitude of frequency tremor across all participants and conditions ranged from 1.21 to 15.31, and post-test values ranged from 0.60 to 13.86.
Measured: Measured at baseline visits (1 & 2) and after 3 weeks of placebo or octanoic acid on post-test visits (1 & 2)
Secondary outcomes (1)
Auditory-perceptual Tremor Severity Ratings
Three experienced listeners independently rated each participant's voice from paired sample recordings comparing the baseline to post-test samples in randomized order for each condition. Sustained vowel and sentence-level recordings were rated, with decoded samples later analyzed for 1=better for post-test compared to baseline, 0= no difference between post-test and baseline. Maximum score for each participant was 3 (post-test was better for each of three raters). The range of possible scores was the sum of each of three raters' scores (0 to 3), with 0 indicating no difference between baseline and post-test voice tremor severity rating, and 3 indicating better voice (less tremor severity) at post-testing compared to pre-testing. Mean post-test values for task were compared for the octanoic acid and placebo conditions, and all raters were blind to which sample was a baseline versus a post-test recording, and which samples were associated with the [placebo or octanoic acid conditions.
Measured: Measured at baseline visits (1 & 2) and after 3 weeks of placebo or octanoic acid on post-test visits (1 & 2).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 May 2013 (estimated)
- Study startJul 2013
- Primary completion22 Dec 2016
- Study completion31 Mar 2017
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.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Essential Voice TremorStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Syracuse University
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute on Deafness and Other Communication Disorders (NIDCD)
- Responsible party
- Principal investigator, Soren Lowell, Syracuse University
- Investigators
- Soren Y Lowell, PhD, Principal investigator, Syracuse University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01864525
- Sponsor's protocol ID
- 370955-3
- Other IDs
- 1R03DC012429-01A1
- Status last verified
- Jul 2018
- First submitted
- 21 May 2013
- Last updated
- 20 Aug 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Haubenberger D, McCrossin G, Lungu C, Considine E, Toro C, Nahab FB, Auh S, Buchwald P, Grimes GJ, Starling J, Potti G, Scheider L, Kalowitz D, Bowen D, Carnie A, Hallett M. Octanoic acid in alcohol-responsive essential tremor: a randomized controlled study. Neurology. 2013 Mar 5;80(10):933-40. doi: 10.1212/WNL.0b013e3182840c4f. Epub 2013 Feb 13.
PubMed 23408867Nahab FB, Wittevrongel L, Ippolito D, Toro C, Grimes GJ, Starling J, Potti G, Haubenberger D, Bowen D, Buchwald P, Dong C, Kalowitz D, Hallett M. An open-label, single-dose, crossover study of the pharmacokinetics and metabolism of two oral formulations of 1-octanol in patients with essential tremor. Neurotherapeutics. 2011 Oct;8(4):753-62. doi: 10.1007/s13311-011-0045-1.
PubMed 21594724Nahab FB, Handforth A, Brown T, Shin C, Quesada A, Dong C, Haubenberger D, Hallett M. Octanoic acid suppresses harmaline-induced tremor in mouse model of essential tremor. Neurotherapeutics. 2012 Jul;9(3):635-8. doi: 10.1007/s13311-012-0121-1.
PubMed 22454323Shill HA, Bushara KO, Mari Z, Reich M, Hallett M. Open-label dose-escalation study of oral 1-octanol in patients with essential tremor. Neurology. 2004 Jun 22;62(12):2320-2. doi: 10.1212/wnl.62.12.2320.
PubMed 15210907Bushara KO, Goldstein SR, Grimes GJ Jr, Burstein AH, Hallett M. Pilot trial of 1-octanol in essential tremor. Neurology. 2004 Jan 13;62(1):122-4. doi: 10.1212/01.wnl.0000101722.95137.19.
PubMed 14718713
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Octanoic Acid for Treatment of Essential Voice Tremor
- ClinicalTrials.gov ID
- NCT01864525
- Acronym / sponsor ID
- 370955-3
- Keywords
- voice, tremor, essential tremor, essential voice tremor, voice treatment, octanoic acid, octanol, voice disorder
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Syracuse UniversityUniversity, hospital or other
- Collaborators
- National Institute on Deafness and Other Communication Disorders (NIDCD)
- Study start
- Jul 2013
- Primary completion
- 22 Dec 2016 (actual)
- Study completion
- 31 Mar 2017 (actual)
- First posted
- 29 May 2013 (estimated)
- Results
- Posted 20 Aug 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Essential voice tremor is a neurological condition that produces a regular, shaking quality in the voice. One form of drug treatment that produces some improvement in tremor of the hands is octanoic acid, which is a food additive that is similar to alcohol. Research suggests that octanoic acid may reduce tremor in the hands/arms with few side effects and no intoxication effects. This study will determine whether octanoic acid may be useful for reducing tremor when it affects the voice. Researchers are hypothesizing that octanoic acid will reduce the effects of tremor on the voice.
Detailed description
Background:
- Essential tremor of the voice produces regular shaking and hoarseness in the voice, making it difficult speech difficult to understand
- Several previous studies have found that octanoic acid and octanol, which are related to alcohol, can improve tremor in some people without producing many side effects and without producing intoxication
- Researchers are interested in determining whether octanoic acid can improve tremor that affects the voice
Objectives:
- To determine the effects of octanoic voice using voice recordings and listener ratings of voice
- To determine the effects of octanoic acid on level of voice disability experienced by people with essential voice tremor
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Inactive capsule | Placebo comparator | Drug: Inactive capsuleParticipants will receive a pill/capsule with an inactive ingredient during the placebo arm of this study. |
| Octanoic acid | Experimental | Drug: Octanoic acidParticipants will receive a pill/capsule with octanoic acid (amount determined by the participant's weight) during the experimental arm of this study. |
Who may qualify
- Ages
- 21 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Participants have a diagnosis of essential voice tremor and show signs of tremor during the endoscopy examination (when pictures of the voice box are obtained)during screening appointment
- Participants show measurable voice tremor from recordings of the voice during screening appointment
Key exclusion criteria (9 listed)
- Participants have a diagnosis or show signs of Parkinson's Disease or another non-essential tremor movement disorder
- Participants have a diagnosis or show signs of spasmodic dysphonia (a different neurological voice disorder)
- Participants have a diagnosis of a severe, non-stable medical condition, such as kidney or liver failure, severe heart disease, severe lung disease, severe metabolic disease, uncontrolled hyperthyroidism, or other life-threatening disease such as active cancer
- Participants have a diagnosis of diabetes mellitus
- Participants are unable to suspend/stop a medication that they are currently taking for tremor or voice disorder for 12 weeks to complete this study
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 |
|---|---|---|
| Octanoic acidAlso: Caprylic acid | Drug | Octanoic acid |
| Inactive capsuleAlso: Placebo | Drug | Inactive capsule |
Primary outcome
Magnitude of Acoustic Amplitude Tremor and Magnitude of Acoustic Frequency Tremor
Time frame: Measured at baseline visits (1 & 2) and after 3 weeks of placebo or octanoic acid on post-test visits (1 & 2)
Voice recordings were used to measure the degree of tremor in the voice. Mean post-test values for each acoustic measure were compared after the octanoic acid and placebo conditions, with and without consideration of baseline values. Mean values represent the average of two testing days. Degree of amplitude tremor shows the extent of amplitude variation as a percent of the mean signal amplitude, with lower numbers indicating less amplitude tremor. Baseline values for magnitude of amplitude tremor across all participants and conditions ranged from 4.06 to 27.09, and post-test values ranged from 1.94 to 26.02. Degree of frequency tremor shows the extent of fundamental frequency variation as a percent of the mean signal frequency, with lower numbers indicating less frequency tremor. Baseline values for magnitude of frequency tremor across all participants and conditions ranged from 1.21 to 15.31, and post-test values ranged from 0.60 to 13.86.
1 secondary outcome
Auditory-perceptual Tremor Severity Ratings
Time frame: Measured at baseline visits (1 & 2) and after 3 weeks of placebo or octanoic acid on post-test visits (1 & 2).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 23408867Haubenberger D, McCrossin G, Lungu C, Considine E, Toro C, Nahab FB, Auh S, Buchwald P, Grimes GJ, Starling J, Potti G, Scheider L, Kalowitz D, Bowen D, Carnie A, Hallett M. Octanoic acid in alcohol-responsive essential tremor: a randomized controlled study. Neurology. 2013 Mar 5;80(10):933-40. doi: 10.1212/WNL.0b013e3182840c4f. Epub 2013 Feb 13.
- Results publication: PubMed 21594724Nahab FB, Wittevrongel L, Ippolito D, Toro C, Grimes GJ, Starling J, Potti G, Haubenberger D, Bowen D, Buchwald P, Dong C, Kalowitz D, Hallett M. An open-label, single-dose, crossover study of the pharmacokinetics and metabolism of two oral formulations of 1-octanol in patients with essential tremor. Neurotherapeutics. 2011 Oct;8(4):753-62. doi: 10.1007/s13311-011-0045-1.
- Results publication: PubMed 22454323Nahab FB, Handforth A, Brown T, Shin C, Quesada A, Dong C, Haubenberger D, Hallett M. Octanoic acid suppresses harmaline-induced tremor in mouse model of essential tremor. Neurotherapeutics. 2012 Jul;9(3):635-8. doi: 10.1007/s13311-012-0121-1.
- Results publication: PubMed 15210907Shill HA, Bushara KO, Mari Z, Reich M, Hallett M. Open-label dose-escalation study of oral 1-octanol in patients with essential tremor. Neurology. 2004 Jun 22;62(12):2320-2. doi: 10.1212/wnl.62.12.2320.
- Results publication: PubMed 14718713Bushara KO, Goldstein SR, Grimes GJ Jr, Burstein AH, Hallett M. Pilot trial of 1-octanol in essential tremor. Neurology. 2004 Jan 13;62(1):122-4. doi: 10.1212/01.wnl.0000101722.95137.19.
- 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.
Soren Y Lowell, PhD principal investigator
Syracuse University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Octanoic acid and Inactive capsule in people with Essential Voice Tremor and Vocal Tremor, run by Syracuse 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 2 inclusion and 9 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 20 Aug 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Aug 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?
Magnitude of Acoustic Amplitude Tremor and Magnitude of Acoustic Frequency Tremor (time frame: Measured at baseline visits (1 & 2) and after 3 weeks of placebo or octanoic acid on post-test visits (1 & 2)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01864525, which also lists 5 linked publications.
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 20 Aug 2018.