Trial Outcomes & Findings for Voice Tremor in Spasmodic Dysphonia: Central Mechanisms and Treatment Response (NCT NCT01961297)
NCT ID: NCT01961297
Last Updated: 2017-11-22
Results Overview
Number of participants who had reported positive effects of at least one alcohol drink on their voice symptoms
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
53 participants
Primary outcome timeframe
Day 1
Results posted on
2017-11-22
Participant Flow
75 subjects were consented, some did not meet criteria, and 53 were enrolled into the study.
Participant milestones
| Measure |
Spasmodic Dysphonia
Participants with Spasmodic Dysphonia (SD) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
Spasmodic Dysphonia/Voice Tremor
Participants with Spasmodic Dysphonia/Voice Tremor (SD/VT) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
|---|---|---|
|
Overall Study
STARTED
|
30
|
23
|
|
Overall Study
COMPLETED
|
28
|
22
|
|
Overall Study
NOT COMPLETED
|
2
|
1
|
Reasons for withdrawal
| Measure |
Spasmodic Dysphonia
Participants with Spasmodic Dysphonia (SD) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
Spasmodic Dysphonia/Voice Tremor
Participants with Spasmodic Dysphonia/Voice Tremor (SD/VT) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
|---|---|---|
|
Overall Study
unconfirmed diagnosis of SD
|
2
|
1
|
Baseline Characteristics
Only participants with voice tremor (VT)
Baseline characteristics by cohort
| Measure |
Spasmodic Dysphonia
n=28 Participants
Participants with Spasmodic Dysphonia (SD) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
Spasmodic Dysphonia/Voice Tremor
n=22 Participants
Participants with Spasmodic Dysphonia/Voice Tremor (SD/VT) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
Total
n=50 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
50.7 years
STANDARD_DEVIATION 11.3 • n=28 Participants
|
58.0 years
STANDARD_DEVIATION 12.2 • n=22 Participants
|
54.4 years
STANDARD_DEVIATION 11.8 • n=50 Participants
|
|
Sex: Female, Male
Female
|
18 Participants
n=28 Participants
|
19 Participants
n=22 Participants
|
37 Participants
n=50 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=28 Participants
|
3 Participants
n=22 Participants
|
13 Participants
n=50 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=28 Participants
|
0 Participants
n=22 Participants
|
0 Participants
n=50 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=28 Participants
|
0 Participants
n=22 Participants
|
0 Participants
n=50 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=28 Participants
|
0 Participants
n=22 Participants
|
0 Participants
n=50 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=28 Participants
|
2 Participants
n=22 Participants
|
3 Participants
n=50 Participants
|
|
Race (NIH/OMB)
White
|
27 Participants
n=28 Participants
|
20 Participants
n=22 Participants
|
47 Participants
n=50 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=28 Participants
|
0 Participants
n=22 Participants
|
0 Participants
n=50 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=28 Participants
|
0 Participants
n=22 Participants
|
0 Participants
n=50 Participants
|
|
Dystonia subtype
ADSD
|
16 Participants
n=28 Participants
|
0 Participants
n=22 Participants
|
16 Participants
n=50 Participants
|
|
Dystonia subtype
ABSD
|
12 Participants
n=28 Participants
|
0 Participants
n=22 Participants
|
12 Participants
n=50 Participants
|
|
Dystonia subtype
ADSD/VT
|
0 Participants
n=28 Participants
|
15 Participants
n=22 Participants
|
15 Participants
n=50 Participants
|
|
Dystonia subtype
ABSD/VT
|
0 Participants
n=28 Participants
|
7 Participants
n=22 Participants
|
7 Participants
n=50 Participants
|
|
Age of onset of dysphonia
|
37.7 years
STANDARD_DEVIATION 11.0 • n=28 Participants
|
44.1 years
STANDARD_DEVIATION 14.2 • n=22 Participants
|
40.9 years
STANDARD_DEVIATION 12.6 • n=50 Participants
|
|
Duration of symptom of dysphonia
|
13.4 years
STANDARD_DEVIATION 11.5 • n=28 Participants
|
14.7 years
STANDARD_DEVIATION 12.7 • n=22 Participants
|
14.1 years
STANDARD_DEVIATION 12.1 • n=50 Participants
|
|
Spasmodic dysphonia severity
|
25.6 years
STANDARD_DEVIATION 16.3 • n=28 Participants
|
30.3 years
STANDARD_DEVIATION 16.8 • n=22 Participants
|
28.0 years
STANDARD_DEVIATION 16.6 • n=50 Participants
|
|
Voice Tremor severity
|
—
|
49.3 units on a scale
STANDARD_DEVIATION 24.7 • n=22 Participants • Only participants with voice tremor (VT)
|
49.3 units on a scale
STANDARD_DEVIATION 24.7 • n=22 Participants • Only participants with voice tremor (VT)
|
PRIMARY outcome
Timeframe: Day 1Population: Only alcohol-responsive participants were in this analysis which is 23 from the SD group and 22 from the SD/VT group.
Number of participants who had reported positive effects of at least one alcohol drink on their voice symptoms
Outcome measures
| Measure |
Spasmodic Dysphonia
n=23 Participants
Participants with Spasmodic Dysphonia (SD) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
Spasmodic Dysphonia/Voice Tremor
n=22 Participants
Participants with Spasmodic Dysphonia/Voice Tremor (SD/VT) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
|---|---|---|
|
Number of Participants Who Reported Positive Effects.
|
17 Participants
|
20 Participants
|
SECONDARY outcome
Timeframe: baseline and Day 1Population: Only alcohol-responsive participants were included in analysis
The number of SD-characteristic voice breaks in each sentence at pre-drug and post-drug assessment
Outcome measures
| Measure |
Spasmodic Dysphonia
n=17 Participants
Participants with Spasmodic Dysphonia (SD) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
Spasmodic Dysphonia/Voice Tremor
n=20 Participants
Participants with Spasmodic Dysphonia/Voice Tremor (SD/VT) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
|---|---|---|
|
Number of Voice Breaks
Baseline - pre-treatment
|
3.6 voice breaks
Standard Deviation 1.6
|
4.1 voice breaks
Standard Deviation 2.5
|
|
Number of Voice Breaks
Day 1 - post treatment
|
2.6 voice breaks
Standard Deviation 1.8
|
2.8 voice breaks
Standard Deviation 2.0
|
SECONDARY outcome
Timeframe: baseline and Day 1Population: Only alcohol-responsive participants were included in analysis
Visual analog scale of severity (0 for none, 100 for most severe/profound)
Outcome measures
| Measure |
Spasmodic Dysphonia
n=17 Participants
Participants with Spasmodic Dysphonia (SD) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
Spasmodic Dysphonia/Voice Tremor
n=20 Participants
Participants with Spasmodic Dysphonia/Voice Tremor (SD/VT) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
|---|---|---|
|
Voice Harshness Severity
Day 1 - post treatment
|
42.8 units on a scale
Standard Deviation 23.6
|
31.6 units on a scale
Standard Deviation 17.8
|
|
Voice Harshness Severity
Baseline - pre treatment
|
48.0 units on a scale
Standard Deviation 25.3
|
58.3 units on a scale
Standard Deviation 23.1
|
SECONDARY outcome
Timeframe: baseline and Day 1Population: Only alcohol-responsive participants were included in analysis
Visual analog scale of severity (0 for none, 100 for most severe/profound)
Outcome measures
| Measure |
Spasmodic Dysphonia
n=17 Participants
Participants with Spasmodic Dysphonia (SD) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
Spasmodic Dysphonia/Voice Tremor
n=20 Participants
Participants with Spasmodic Dysphonia/Voice Tremor (SD/VT) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
|---|---|---|
|
Breathlessness Severity
Baseline - pre treatment
|
24.0 units on a scale
Standard Deviation 18.1
|
18.6 units on a scale
Standard Deviation 25.2
|
|
Breathlessness Severity
Day 1 - post treatment
|
20.0 units on a scale
Standard Deviation 19.5
|
14.8 units on a scale
Standard Deviation 19.2
|
SECONDARY outcome
Timeframe: baseline and Day 1Population: Only alcohol-responsive participants with voice tremors were included in analysis
Visual analog scale of severity (0 for none, 100 for most severe/profound)
Outcome measures
| Measure |
Spasmodic Dysphonia
Participants with Spasmodic Dysphonia (SD) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
Spasmodic Dysphonia/Voice Tremor
n=20 Participants
Participants with Spasmodic Dysphonia/Voice Tremor (SD/VT) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
|---|---|---|
|
Voice Tremor Severity
Baseline - pre treatment
|
—
|
51.2 units on a scale
Standard Deviation 23.6
|
|
Voice Tremor Severity
Day 1 - post treatment
|
—
|
31.4 units on a scale
Standard Deviation 18.5
|
Adverse Events
Spasmodic Dysphonia
Serious events: 0 serious events
Other events: 14 other events
Deaths: 0 deaths
Spasmodic Dysphonia/Voice Tremor
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Spasmodic Dysphonia
n=28 participants at risk
Participants with Spasmodic Dysphonia (SD) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
Spasmodic Dysphonia/Voice Tremor
n=22 participants at risk
Participants with Spasmodic Dysphonia/Voice Tremor (SD/VT) given oral administration of a single dose of sodium oxybate (1.0-1.5 gm)
|
|---|---|---|
|
Nervous system disorders
Slight lightheadedness
|
39.3%
11/28 • Up to 60 minutes after drug ingestion
side effects questionnaire, Epworth Sleepiness Scale
|
18.2%
4/22 • Up to 60 minutes after drug ingestion
side effects questionnaire, Epworth Sleepiness Scale
|
|
Nervous system disorders
Drowsiness
|
0.00%
0/28 • Up to 60 minutes after drug ingestion
side effects questionnaire, Epworth Sleepiness Scale
|
4.5%
1/22 • Up to 60 minutes after drug ingestion
side effects questionnaire, Epworth Sleepiness Scale
|
|
Nervous system disorders
Dizziness
|
17.9%
5/28 • Up to 60 minutes after drug ingestion
side effects questionnaire, Epworth Sleepiness Scale
|
22.7%
5/22 • Up to 60 minutes after drug ingestion
side effects questionnaire, Epworth Sleepiness Scale
|
|
Nervous system disorders
Headache
|
3.6%
1/28 • Up to 60 minutes after drug ingestion
side effects questionnaire, Epworth Sleepiness Scale
|
0.00%
0/22 • Up to 60 minutes after drug ingestion
side effects questionnaire, Epworth Sleepiness Scale
|
Additional Information
Dr. Kristina Simonyan
Icahn School of Medicine at Mount Sinai
Phone: 212-241-0656
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place