Trial Outcomes & Findings for Effects of Octanoic Acid for Treatment of Essential Voice Tremor (NCT NCT01864525)

Last Updated: 2018-08-20

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

17 participants

Primary outcome timeframe

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

Results posted on

2018-08-20

Participant Flow

Participant milestones

Participant milestones
Measure	Inactive Capsule First Participants who were randomized to receive a capsule with an inactive ingredient (placebo) during the first 3 weeks of the study (placebo arm of crossover design study), and then received octanoic acid in the last 3 weeks of the study. Placebo	Octanoic Acid First Participants who were randomized to receive a capsule with octanoic acid (amount determined by the participant's weight) during the first 3 weeks of the study (experimental arm of crossover design study), and then received the placebo in the last 3 weeks of the study. Octanoic acid
Overall Study STARTED	10	7
Overall Study COMPLETED	9	7
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Inactive Capsule First Participants who were randomized to receive a capsule with an inactive ingredient (placebo) during the first 3 weeks of the study (placebo arm of crossover design study), and then received octanoic acid in the last 3 weeks of the study. Placebo	Octanoic Acid First Participants who were randomized to receive a capsule with octanoic acid (amount determined by the participant's weight) during the first 3 weeks of the study (experimental arm of crossover design study), and then received the placebo in the last 3 weeks of the study. Octanoic acid
Overall Study Adverse Event	1	0

Baseline Characteristics

Effects of Octanoic Acid for Treatment of Essential Voice Tremor

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Inactive Capsule First n=10 Participants Participants who were randomized to receive a capsule with an inactive ingredient (placebo) during the first 3 weeks of the study (placebo arm of crossover design study), and then received octanoic acid in the last 3 weeks of the study. Placebo	Octanoic Acid First n=7 Participants Participants who were randomized to receive a capsule with octanoic acid (amount determined by the participant's weight) during the first 3 weeks of the study (experimental arm of crossover design study), and then received the placebo in the last 3 weeks of the study. Octanoic acid	Total n=17 Participants Total of all reporting groups
Age, Continuous	68.90 years STANDARD_DEVIATION 7.20 • n=5 Participants	70.43 years STANDARD_DEVIATION 11.70 • n=7 Participants	69.53 years STANDARD_DEVIATION 9.01 • n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants	6 Participants n=7 Participants	15 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=5 Participants	7 Participants n=7 Participants	17 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	10 Participants n=5 Participants	7 Participants n=7 Participants	17 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

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

Outcome measures

Outcome measures
Measure	Placebo n=16 Participants Participants who received a capsule with an inactive ingredient (placebo) during either the first or last 3 weeks of the study. Placebo	Octanoic Acid n=16 Participants Participants who received a capsule with octanoic acid (amount determined by the participant's weight) during either the first or last 3 weeks of the study. Octanoic acid
Magnitude of Acoustic Amplitude Tremor and Magnitude of Acoustic Frequency Tremor Baseline Magnitude of Acoustic Amplitude Tremor	12.91 percentage of voice signal with tremor Standard Deviation 6.46	13.49 percentage of voice signal with tremor Standard Deviation 6.48
Magnitude of Acoustic Amplitude Tremor and Magnitude of Acoustic Frequency Tremor Post-test Magnitude of Acoustic Amplitude Tremor	12.43 percentage of voice signal with tremor Standard Deviation 7.37	9.35 percentage of voice signal with tremor Standard Deviation 5.42
Magnitude of Acoustic Amplitude Tremor and Magnitude of Acoustic Frequency Tremor Baseline Magnitude of Acoustic Frequency Tremor	5.57 percentage of voice signal with tremor Standard Deviation 4.29	5.19 percentage of voice signal with tremor Standard Deviation 3.58
Magnitude of Acoustic Amplitude Tremor and Magnitude of Acoustic Frequency Tremor Post-test Magnitude of Acoustic Frequency Tremor	5.35 percentage of voice signal with tremor Standard Deviation 3.76	3.98 percentage of voice signal with tremor Standard Deviation 2.94

SECONDARY outcome

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

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.

Outcome measures

Outcome measures
Measure	Placebo n=16 Participants Participants who received a capsule with an inactive ingredient (placebo) during either the first or last 3 weeks of the study. Placebo	Octanoic Acid n=16 Participants Participants who received a capsule with octanoic acid (amount determined by the participant's weight) during either the first or last 3 weeks of the study. Octanoic acid
Auditory-perceptual Tremor Severity Ratings Sustained vowel, summed binary ratings (0-3)	1.25 units on a scale Standard Deviation 1.13	1.53 units on a scale Standard Deviation 1.13
Auditory-perceptual Tremor Severity Ratings Sentence-level, summed binary ratings (0-3)	1.63 units on a scale Standard Deviation 1.12	1.19 units on a scale Standard Deviation 0.87

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Octanoic Acid

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Placebo n=17 participants at risk Study phase 1 or 2, when participants were randomized to the 3-week intervention phase in which an inactive capsule (placebo or inactive drug) was taken once a day for 3 weeks.	Octanoic Acid n=17 participants at risk Study phase 1 or 2, when participants were randomized to the 3-week intervention phase in which octanoic acid (the active drug) was taken once a day for 3 weeks.
Gastrointestinal disorders Diarrhea	5.9% 1/17 • Number of events 1 • For each participant, adverse event data were collected over approximately an 11 week period. This included data beginning with baseline testing in week 1, Phase 1 (placebo or octanoic acid condition) and continued through the week after post-test data was completed for Phase 2, on the final study visit (final nursing assessment visit). Participants completed symptom questionnaires with an independent nurse at four time-points: baseline before either the placebo or octanoic acid conditions, during the 3 weeks intake for each study phase, and upon completion of the study. Additionally, participants were called daily to inquire about any symptoms/symptom severity for three days after full drug intake was initiated for each study phase (placebo or octanoic acid) and then every 3-5 days thereafter each 3 week dosing period.	0.00% 0/17 • For each participant, adverse event data were collected over approximately an 11 week period. This included data beginning with baseline testing in week 1, Phase 1 (placebo or octanoic acid condition) and continued through the week after post-test data was completed for Phase 2, on the final study visit (final nursing assessment visit). Participants completed symptom questionnaires with an independent nurse at four time-points: baseline before either the placebo or octanoic acid conditions, during the 3 weeks intake for each study phase, and upon completion of the study. Additionally, participants were called daily to inquire about any symptoms/symptom severity for three days after full drug intake was initiated for each study phase (placebo or octanoic acid) and then every 3-5 days thereafter each 3 week dosing period.

Additional Information

Dr. Soren Lowell

Syracuse University

Phone: 315-443-9648

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place