Trial Outcomes & Findings for Propranolol and Botulinum Toxin for Essential Vocal Tremor (NCT NCT02111369)

Results Overview

The QUEST questionnaire is a 30 item self-reported essential tremor-specific quality of life scale that asks participants to rate responses (never/no, rarely, sometimes, frequently, always/yes, or not applicable). Total scores range between 0 to 100 where 0 is the best score and 100 is the worst score. A higher score indicates a lower quality of life.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

18 participants

Primary outcome timeframe

Baseline, 2 weeks, 6 weeks

Results posted on

2016-04-27

Participant Flow

Participants were recruited from the Emory Voice Center from August 2014 to February 2015.

Participants who previously received botulinum toxin injections for essential voice tremor (EVT) underwent a three month washout period prior to participation.

Participant milestones

Participant milestones
Measure	Propranolol/Botulinum After baseline analysis, patient will be given a prescription by the principal investigator for propranolol. This prescription will consist of a starting dose of generic immediate-release at 10 mg three times per day (30 mg each day) with an increase in dose in 5-7 days if there is no effect (60 mg each day) and if the patient had demonstrated no side effects. Dose may be increased to 240 mg each day depending on patient improvement and side effect profile. After second evaluation, patient will receive botulinum toxin injections. The risks and benefits of botulinum toxin therapy will be explained to the patient, and bilateral injections will take place. Propranolol: After a discussion of the risks and benefits of propranolol therapy, the patient will then be given a prescription by the principal investigator for propranolol. This prescription will consist of a starting dose of generic immediate-release at 10 mg three times daily (30 mg each day) with an increase in dose in 5-7 days
Overall Study STARTED	18
Overall Study COMPLETED	15
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Propranolol/Botulinum After baseline analysis, patient will be given a prescription by the principal investigator for propranolol. This prescription will consist of a starting dose of generic immediate-release at 10 mg three times per day (30 mg each day) with an increase in dose in 5-7 days if there is no effect (60 mg each day) and if the patient had demonstrated no side effects. Dose may be increased to 240 mg each day depending on patient improvement and side effect profile. After second evaluation, patient will receive botulinum toxin injections. The risks and benefits of botulinum toxin therapy will be explained to the patient, and bilateral injections will take place. Propranolol: After a discussion of the risks and benefits of propranolol therapy, the patient will then be given a prescription by the principal investigator for propranolol. This prescription will consist of a starting dose of generic immediate-release at 10 mg three times daily (30 mg each day) with an increase in dose in 5-7 days
Overall Study Lost to Follow-up	2
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Propranolol and Botulinum Toxin for Essential Vocal Tremor

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Propranolol/Botulinum n=18 Participants After baseline analysis, participants were given a prescription by the principal investigator for propranolol. This prescription consisted of a starting dose of generic immediate-release at 10 mg three times per day (30 mg each day) with an increase in dose in 5-7 days if there is no effect (60 mg each day) and if the participant demonstrated no side effects. Dose was increased up to 240 mg a day depending on participant improvement and side effect profile. After second evaluation, participants received botulinum toxin injections. The risks and benefits of botulinum toxin therapy were explained to the participant, and bilateral injections took place.
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=93 Participants
Age, Categorical >=65 years	14 Participants n=93 Participants
Sex: Female, Male Female	18 Participants n=93 Participants
Sex: Female, Male Male	0 Participants n=93 Participants
Region of Enrollment United States	18 participants n=93 Participants

PRIMARY outcome

Timeframe: Baseline, 2 weeks, 6 weeks

Population: Participants who completed all three study visits (Baseline Visit 1, Visit 2, and Visit 3).

The QUEST questionnaire is a 30 item self-reported essential tremor-specific quality of life scale that asks participants to rate responses (never/no, rarely, sometimes, frequently, always/yes, or not applicable). Total scores range between 0 to 100 where 0 is the best score and 100 is the worst score. A higher score indicates a lower quality of life.

Outcome measures

Outcome measures
Measure	Propranolol/Botulinum n=15 Participants After baseline analysis, participants were given a prescription by the principal investigator for propranolol. This prescription consisted of a starting dose of generic immediate-release at 10 mg three times per day (30 mg each day) with an increase in dose in 5-7 days if there is no effect (60 mg each day) and if the participant demonstrated no side effects. Dose was increased up to 240 mg a day depending on participant improvement and side effect profile. After second evaluation, participants received botulinum toxin injections. The risks and benefits of botulinum toxin therapy were explained to the participant, and bilateral injections took place. Propranolol: After a discussion of the risks and benefits of propranolol therapy, the patient will then be given a prescription by the principal investigator for propranolol. This prescription will consist of a starting dose of generic immediate-release at 10 mg three times daily (30 mg each day) with an increase in dose in 5-7 days
Change in Quality of Life in Essential Tremor (QUEST) Questionnaire Score Baseline (Visit 1)	39.41 units on a scale Standard Deviation 27.89
Change in Quality of Life in Essential Tremor (QUEST) Questionnaire Score Visit 2	29.50 units on a scale Standard Deviation 23.73
Change in Quality of Life in Essential Tremor (QUEST) Questionnaire Score Visit 3	23.60 units on a scale Standard Deviation 24.49

PRIMARY outcome

Timeframe: Baseline, 2 weeks, 6 weeks

Population: Participants who completed all three study visits (Baseline Visit 1, Visit 2, and Visit 3).

The VRQOL is a ten question self-reported measure that asks patients to rate responses from 1-5 (1=none, not a problem, 2=a small amount, 3=a moderate (medium) amount, 4=a lot, 5=problem is as "bad as it can be"). Total scores range from 0 to 100. A higher score indicates more problems interfering with day to day activities.

Outcome measures

Outcome measures
Measure	Propranolol/Botulinum n=15 Participants After baseline analysis, participants were given a prescription by the principal investigator for propranolol. This prescription consisted of a starting dose of generic immediate-release at 10 mg three times per day (30 mg each day) with an increase in dose in 5-7 days if there is no effect (60 mg each day) and if the participant demonstrated no side effects. Dose was increased up to 240 mg a day depending on participant improvement and side effect profile. After second evaluation, participants received botulinum toxin injections. The risks and benefits of botulinum toxin therapy were explained to the participant, and bilateral injections took place. Propranolol: After a discussion of the risks and benefits of propranolol therapy, the patient will then be given a prescription by the principal investigator for propranolol. This prescription will consist of a starting dose of generic immediate-release at 10 mg three times daily (30 mg each day) with an increase in dose in 5-7 days
Change in Voice-Related Quality Of Life (VRQOL) Questionnaire Score Baseline (Visit 1)	42.22 units on a scale Standard Deviation 23.64
Change in Voice-Related Quality Of Life (VRQOL) Questionnaire Score Visit 2	51.53 units on a scale Standard Deviation 26.21
Change in Voice-Related Quality Of Life (VRQOL) Questionnaire Score Visit 3	64.16 units on a scale Standard Deviation 22.23

PRIMARY outcome

Timeframe: Baseline, 2 weeks, 6 weeks

Population: Participants who completed all three study visits (Baseline Visit 1, Visit 2, and Visit 3).

The CAPE-V is a clinically validated perceptual voice assessment tool that is used to describe the severity of auditory-perceptual attributes of a voice problem, in a way that can be communicated among clinicians. Participant's speech is recorded and evaluated by trained "listeners". Listeners indicate overall tremor severity by making a tick mark on a 1 to 100 mm visual analog scale. Total scores range from 0 to 100 where 0 is the best score and 100 is the worst score.

Outcome measures

Outcome measures
Measure	Propranolol/Botulinum n=15 Participants After baseline analysis, participants were given a prescription by the principal investigator for propranolol. This prescription consisted of a starting dose of generic immediate-release at 10 mg three times per day (30 mg each day) with an increase in dose in 5-7 days if there is no effect (60 mg each day) and if the participant demonstrated no side effects. Dose was increased up to 240 mg a day depending on participant improvement and side effect profile. After second evaluation, participants received botulinum toxin injections. The risks and benefits of botulinum toxin therapy were explained to the participant, and bilateral injections took place. Propranolol: After a discussion of the risks and benefits of propranolol therapy, the patient will then be given a prescription by the principal investigator for propranolol. This prescription will consist of a starting dose of generic immediate-release at 10 mg three times daily (30 mg each day) with an increase in dose in 5-7 days
Change in Consensus Auditory-Perceptual Evaluation of Voice (CAPE-V) Score Baseline (Visit 1)	43.61 units on a scale Standard Deviation 23.26
Change in Consensus Auditory-Perceptual Evaluation of Voice (CAPE-V) Score Visit 2	45.89 units on a scale Standard Deviation 24.57
Change in Consensus Auditory-Perceptual Evaluation of Voice (CAPE-V) Score Visit 3	35.67 units on a scale Standard Deviation 22.14

PRIMARY outcome

Timeframe: Baseline, 2 weeks, 6 weeks

Population: Data were not collected as investigators found the Voice Related Quality of Life (VRQOL) scale to be a better measure.

Patient-Reported Measure • 0-7 ranking

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: baseline, 2 weeks, 6 weeks

Population: Data were not collected as investigators found the Voice Related Quality of Life (VRQOL) scale to be a better measure.

Objective Voice Assessment • Using the Computerized Speech Laboratory speech and voice analysis system (KayPENTAX, Montvale, NJ)

Outcome measures

Outcome data not reported

Adverse Events

Propranolol/Botulinum

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Michael Johns

Emory University

Phone: 323-865-9839

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place