Effects of Octanoic Acid for Treatment of Essential Voice Tremor

NCT01864525 | Completed Phase 1 Results

• 2 other identifiers: 370955-31R03DC012429-01A1
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Essential Voice Tremor; Vocal Tremor; Voice Tremor; Essential Tremor of Voice

Keywords

voice; tremor; essential tremor; essential voice tremor; voice treatment; octanoic acid; octanol; voice disorder; dysphonia

Outcome Measures

Primary Outcomes (1)

• 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 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

  Measured at baseline visits (1 & 2) and after 3 weeks of placebo or octanoic acid on post-test visits (1 & 2).

Study Arms (2)

Inactive capsule

PLACEBO COMPARATOR

Participants will receive a pill/capsule with an inactive ingredient during the placebo arm of this study.

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.

Drug: Octanoic acid

Interventions

Octanoic acid DRUG

Also known as: Caprylic acid

Octanoic acid

Inactive capsule DRUG

Also known as: Placebo

Inactive capsule

Eligibility Criteria

• Age: 21 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• Participants are pregnant or lactating
• Participants have an allergy to soy
• Participants have Irritable Bowel Syndrome

Sponsors & Collaborators

• Syracuse University: lead
• National Institute on Deafness and Other Communication Disorders (NIDCD): collaborator

Study Sites (1)

Syracuse University & Upstate Medical University

Syracuse, New York, 13210, United States

Location

Related Publications (5)

• 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.

  PMID: 23408867 RESULT

• Nahab 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.

  PMID: 21594724 RESULT

• Nahab 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.

  PMID: 22454323 RESULT

• Shill 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.

  PMID: 15210907 RESULT

• Bushara 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.

  PMID: 14718713 RESULT

MeSH Terms

Conditions

Tremor; Essential Tremor; Voice Disorders; Dysphonia

Interventions

octanoic acid

Condition Hierarchy (Ancestors)

Dyskinesias; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Movement Disorders; Central Nervous System Diseases; Laryngeal Diseases; Respiratory Tract Diseases; Otorhinolaryngologic Diseases

Results Point of Contact

• Title: Dr. Soren Lowell
• Organization: Syracuse University

slowell@syr.edu

315-443-9648

Study Officials

• Soren Y Lowell, PhD

  Syracuse University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor, Communication Sciences & Disorders

Study Record Dates

• First Submitted: May 21, 2013
• First Posted: May 29, 2013
• Study Start: July 1, 2013
• Primary Completion: December 22, 2016
• Study Completion: March 31, 2017
• Last Updated: August 20, 2018
• Results First Posted: August 20, 2018

Record last verified: 2018-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)