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Propranolol and Botulinum Toxin for Essential Vocal Tremor

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ClinicalTrials.gov Identifier: NCT02111369
Recruitment Status : Completed
First Posted : April 11, 2014
Results First Posted : April 27, 2016
Last Update Posted : April 27, 2016
Sponsor:
Information provided by (Responsible Party):
Michael M Johns MD, Emory University

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Essential Vocal Tremor
Essential Voice Tremor
Essential Tremor
Voice Tremor
Vocal Tremor
Interventions Drug: Propranolol
Procedure: Botulinum Toxin
Enrollment 18
Recruitment Details Participants were recruited from the Emory Voice Center from August 2014 to February 2015.
Pre-assignment Details Participants who previously received botulinum toxin injections for essential voice tremor (EVT) underwent a three month washout period prior to participation.
Arm/Group Title Propranolol/Botulinum
Hide Arm/Group Description

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

Period Title: Overall Study
Started 18
Completed 15
Not Completed 3
Reason Not Completed
Lost to Follow-up             2
Withdrawal by Subject             1
Arm/Group Title Propranolol/Botulinum
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 18
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
<=18 years
0
   0.0%
Between 18 and 65 years
4
  22.2%
>=65 years
14
  77.8%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants
Female
18
 100.0%
Male
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 18 participants
18
1.Primary Outcome
Title Change in Quality of Life in Essential Tremor (QUEST) Questionnaire Score
Hide Description 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.
Time Frame Baseline, 2 weeks, 6 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who completed all three study visits (Baseline Visit 1, Visit 2, and Visit 3).
Arm/Group Title Propranolol/Botulinum
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline (Visit 1) 39.41  (27.89)
Visit 2 29.50  (23.73)
Visit 3 23.60  (24.49)
2.Primary Outcome
Title Change in Voice-Related Quality Of Life (VRQOL) Questionnaire Score
Hide Description 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.
Time Frame Baseline, 2 weeks, 6 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who completed all three study visits (Baseline Visit 1, Visit 2, and Visit 3).
Arm/Group Title Propranolol/Botulinum
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline (Visit 1) 42.22  (23.64)
Visit 2 51.53  (26.21)
Visit 3 64.16  (22.23)
3.Primary Outcome
Title Change in Consensus Auditory-Perceptual Evaluation of Voice (CAPE-V) Score
Hide Description 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.
Time Frame Baseline, 2 weeks, 6 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who completed all three study visits (Baseline Visit 1, Visit 2, and Visit 3).
Arm/Group Title Propranolol/Botulinum
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline (Visit 1) 43.61  (23.26)
Visit 2 45.89  (24.57)
Visit 3 35.67  (22.14)
4.Primary Outcome
Title Change in Global Voice Rating
Hide Description

Patient-Reported Measure

• 0-7 ranking

Time Frame Baseline, 2 weeks, 6 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Data were not collected as investigators found the Voice Related Quality of Life (VRQOL) scale to be a better measure.
Arm/Group Title Propranolol/Botulinum
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
5.Primary Outcome
Title Change in Acoustic Spectrograms
Hide Description

Objective Voice Assessment

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

Time Frame baseline, 2 weeks, 6 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Data were not collected as investigators found the Voice Related Quality of Life (VRQOL) scale to be a better measure.
Arm/Group Title Propranolol/Botulinum
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Propranolol/Botulinum
Hide Arm/Group Description 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.
All-Cause Mortality
Propranolol/Botulinum
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Propranolol/Botulinum
Affected / at Risk (%)
Total   0/18 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Propranolol/Botulinum
Affected / at Risk (%)
Total   0/18 (0.00%) 
The study lacks a placebo or control group, with participants representing their own internal controls. Additionally, inter-rater reliability among listeners for the CAPE-V outcome measure analysis was low.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Michael Johns
Organization: Emory University
Phone: 323-865-9839
Layout table for additonal information
Responsible Party: Michael M Johns MD, Emory University
ClinicalTrials.gov Identifier: NCT02111369     History of Changes
Other Study ID Numbers: IRB00072929
First Submitted: April 8, 2014
First Posted: April 11, 2014
Results First Submitted: February 24, 2016
Results First Posted: April 27, 2016
Last Update Posted: April 27, 2016