Trial Outcomes & Findings for Incobotulinum Toxin A (Xeomin®) As A Treatment For Focal Task-Specific Dystonia Of The Musician's Hand (NCT NCT02107261)
NCT ID: NCT02107261
Last Updated: 2023-05-11
Results Overview
The principal outcome measure was improvement in musical performance, measured by blinded rater, using physician global perception of change at post treatment compared to at 8 weeks. Categories are very much improved, much improved, minimal improved, no change, and minimal worse.
COMPLETED
PHASE2
21 participants
at visit 4 (week 8)
2023-05-11
Participant Flow
Participant milestones
| Measure |
Placebo Then Botulinum Toxin
Placebo dose first does then boosters at week 2 and week 4 then 4 weeks washout, then Incobotulinumtoxin A first dose, with boosters at 2 week and week 4.
|
Incobotulinumtoxin A Then Placebo
Incobotulinumtoxin A dose first does then boosters at week 2 and week 4 then 4 weeks washout, then Placebo first dose, with boosters at 2 week and week 4.
|
|---|---|---|
|
Cycle 1: Week 1-4
STARTED
|
11
|
10
|
|
Cycle 1: Week 1-4
COMPLETED
|
11
|
10
|
|
Cycle 1: Week 1-4
NOT COMPLETED
|
0
|
0
|
|
Washout: Weakness Back to Baseline
STARTED
|
11
|
10
|
|
Washout: Weakness Back to Baseline
COMPLETED
|
10
|
10
|
|
Washout: Weakness Back to Baseline
NOT COMPLETED
|
1
|
0
|
|
Cycle 2: Week 12-15
STARTED
|
10
|
10
|
|
Cycle 2: Week 12-15
COMPLETED
|
10
|
9
|
|
Cycle 2: Week 12-15
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Placebo Then Botulinum Toxin
Placebo dose first does then boosters at week 2 and week 4 then 4 weeks washout, then Incobotulinumtoxin A first dose, with boosters at 2 week and week 4.
|
Incobotulinumtoxin A Then Placebo
Incobotulinumtoxin A dose first does then boosters at week 2 and week 4 then 4 weeks washout, then Placebo first dose, with boosters at 2 week and week 4.
|
|---|---|---|
|
Washout: Weakness Back to Baseline
Lost to Follow-up
|
1
|
0
|
|
Cycle 2: Week 12-15
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
Incobotulinum Toxin A (Xeomin®) As A Treatment For Focal Task-Specific Dystonia Of The Musician's Hand
Baseline characteristics by cohort
| Measure |
Placebo Then Botulinum Toxin
n=11 Participants
Placebo dose first does then boosters at week 2 and week 4 then 4 weeks washout, then Incobotulinumtoxin A first dose, with boosters at 2 week and week 4.
|
Botulinum Toxin Then Placebo
n=10 Participants
Incobotulinumtoxin A dose first does then boosters at week 2 and week 4 then 4 weeks washout, then Placebo first dose, with boosters at 2 week and week 4.
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
52.68 years
n=99 Participants
|
49.66 years
n=107 Participants
|
50.2 years
n=206 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
19 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
19 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: at visit 4 (week 8)Population: Data not collected for one participant. Data results for Week 8 before crossover treatment.
The principal outcome measure was improvement in musical performance, measured by blinded rater, using physician global perception of change at post treatment compared to at 8 weeks. Categories are very much improved, much improved, minimal improved, no change, and minimal worse.
Outcome measures
| Measure |
Placebo
n=11 Participants
matching placebo injection
|
Incobotulinum Toxin Injection
n=9 Participants
Incobotulinumtoxin A used on average doses between 10U and 30 Units per subject, but at no time did any subject receive over 100 Units. Each study visit, the assessing physician selected the muscles and the amounts of medication to be injected.
|
|---|---|---|
|
Physician Global Perception of Change - Blinded Rater 1
Much Improved
|
1 Participants
|
4 Participants
|
|
Physician Global Perception of Change - Blinded Rater 1
Minimally Worse
|
3 Participants
|
1 Participants
|
|
Physician Global Perception of Change - Blinded Rater 1
Very much improved
|
1 Participants
|
1 Participants
|
|
Physician Global Perception of Change - Blinded Rater 1
Minimally Improved
|
3 Participants
|
3 Participants
|
|
Physician Global Perception of Change - Blinded Rater 1
No Change
|
3 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: at visit 4 (week 8)Population: Data not collected for one participant. Data results for Week 8 before crossover treatment.
The principal outcome measure was improvement in musical performance, measured by blinded rater, using physician global perception of change at post treatment compared to at 8 weeks. Categories are very much improved, much improved, minimal improved, no change, and minimal worse.
Outcome measures
| Measure |
Placebo
n=11 Participants
matching placebo injection
|
Incobotulinum Toxin Injection
n=9 Participants
Incobotulinumtoxin A used on average doses between 10U and 30 Units per subject, but at no time did any subject receive over 100 Units. Each study visit, the assessing physician selected the muscles and the amounts of medication to be injected.
|
|---|---|---|
|
Physician Global Perception of Change - Blinded Rater 2
Very much improved
|
0 Participants
|
0 Participants
|
|
Physician Global Perception of Change - Blinded Rater 2
Minimal Improved
|
4 Participants
|
5 Participants
|
|
Physician Global Perception of Change - Blinded Rater 2
Much Improved
|
1 Participants
|
2 Participants
|
|
Physician Global Perception of Change - Blinded Rater 2
No Change
|
3 Participants
|
1 Participants
|
|
Physician Global Perception of Change - Blinded Rater 2
Minimal Worse
|
3 Participants
|
1 Participants
|
PRIMARY outcome
Timeframe: baseline and at 8 weeksPopulation: Data not collected for one participant. Data results for Week 8 before crossover treatment.
Rating of overall musical performance based on comparison to baseline video at week 8. Rating from +3, very much improved to -3, very much worse. Higher score indicates more improvement.
Outcome measures
| Measure |
Placebo
n=11 Participants
matching placebo injection
|
Incobotulinum Toxin Injection
n=9 Participants
Incobotulinumtoxin A used on average doses between 10U and 30 Units per subject, but at no time did any subject receive over 100 Units. Each study visit, the assessing physician selected the muscles and the amounts of medication to be injected.
|
|---|---|---|
|
Rating of Overall Musical Performance - Blinded Rater 1
+3 Very much improved
|
0 Participants
|
1 Participants
|
|
Rating of Overall Musical Performance - Blinded Rater 1
+2 Much improved
|
2 Participants
|
2 Participants
|
|
Rating of Overall Musical Performance - Blinded Rater 1
+1 Minimally improved
|
0 Participants
|
3 Participants
|
|
Rating of Overall Musical Performance - Blinded Rater 1
0 No change
|
5 Participants
|
2 Participants
|
|
Rating of Overall Musical Performance - Blinded Rater 1
-1 Minimal worse
|
4 Participants
|
1 Participants
|
|
Rating of Overall Musical Performance - Blinded Rater 1
-2 Much worse
|
0 Participants
|
0 Participants
|
|
Rating of Overall Musical Performance - Blinded Rater 1
-3 Very much worse
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: baseline and week 8Population: Data not collected for one participant. Data results for Week 8 before crossover treatment.
Rating of overall musical performance based on comparison to baseline video at week 8. Best Overall Musical Performance on 7 point scale from +3 Very much improved to -3 Very much worse. Higher score indicates more improvement.
Outcome measures
| Measure |
Placebo
n=11 Participants
matching placebo injection
|
Incobotulinum Toxin Injection
n=9 Participants
Incobotulinumtoxin A used on average doses between 10U and 30 Units per subject, but at no time did any subject receive over 100 Units. Each study visit, the assessing physician selected the muscles and the amounts of medication to be injected.
|
|---|---|---|
|
Rating of Overall Musical Performance - Blinded Rater 2
+3 Very much improved
|
0 Participants
|
0 Participants
|
|
Rating of Overall Musical Performance - Blinded Rater 2
+2 Much Improved
|
3 Participants
|
2 Participants
|
|
Rating of Overall Musical Performance - Blinded Rater 2
+1 Minimally Improved
|
0 Participants
|
4 Participants
|
|
Rating of Overall Musical Performance - Blinded Rater 2
0 No Change
|
5 Participants
|
2 Participants
|
|
Rating of Overall Musical Performance - Blinded Rater 2
-1 Minimally Worse
|
3 Participants
|
1 Participants
|
|
Rating of Overall Musical Performance - Blinded Rater 2
-2 Much worse
|
0 Participants
|
0 Participants
|
|
Rating of Overall Musical Performance - Blinded Rater 2
-3 Very much worse
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: baseline at 24 weeksPopulation: data not collected at 24 weeks
The principal outcome measure will be improvement in musical performance, measured by self-rated questionnaire, quantitative Musical Instrument Digital Interface (MIDI) analysis and blinded high speed video analysis post treatment compared to baseline and 24 weeks.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: baseline and at 24 weeksPopulation: data not collected at 24 weeks
The principal outcome measure will be improvement in musical performance, measured by self-rated questionnaire, quantitative MIDI analysis and blinded high speed video analysis post treatment compared to baseline and 24 weeks.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: baseline and at 8 weeksPopulation: Data results for Week 8 before crossover treatment.
Patient reported outcomes measured by questionnaires that address adverse events and the impact of quality of life using the perceived stress scale. A 10-item questionnaire, each item scored 0 (never) to 4 (very often), full scale from 0-40, with higher score indicating higher perceived stress. the more often the person perceives stress Change in PSS at week 8 compared to baseline
Outcome measures
| Measure |
Placebo
n=11 Participants
matching placebo injection
|
Incobotulinum Toxin Injection
n=10 Participants
Incobotulinumtoxin A used on average doses between 10U and 30 Units per subject, but at no time did any subject receive over 100 Units. Each study visit, the assessing physician selected the muscles and the amounts of medication to be injected.
|
|---|---|---|
|
Change in Perceived Stress Scale (PSS)
|
-1.429 score on a scale
Standard Error 0.864
|
-0.5031 score on a scale
Standard Error 0.946
|
SECONDARY outcome
Timeframe: baseline and at 8 weeksPopulation: Data results for Week 8 before crossover treatment.
Motor strength using a dynamometer of the finger/wrist/elbow flexors to document any treatment induced weakness. Scale from 0-4, higher score indicates poorer health outcomes.
Outcome measures
| Measure |
Placebo
n=11 Participants
matching placebo injection
|
Incobotulinum Toxin Injection
n=10 Participants
Incobotulinumtoxin A used on average doses between 10U and 30 Units per subject, but at no time did any subject receive over 100 Units. Each study visit, the assessing physician selected the muscles and the amounts of medication to be injected.
|
|---|---|---|
|
Change in Medical Research Council (MRC) Scale
Right FDP dig 2
|
0.07 score on a scale
Standard Error 0.0655
|
-0.087 score on a scale
Standard Error 0.0654
|
|
Change in Medical Research Council (MRC) Scale
R Lumb dig 4
|
-0.013 score on a scale
Standard Error 0.0493
|
-0.1190 score on a scale
Standard Error 0.0492
|
SECONDARY outcome
Timeframe: baseline and at 8 weeksPopulation: Data results for Week 8 before crossover treatment.
Motor strength tested using a dynamometer to document any weakness the treatment may produce as compared to the baseline visit and 8 weeks. Dynamometer (DYN) - is a mechanical device that measures the pounds of force that a muscle exerts, Grip - a device that measures grip strength, and finger flexors digit 2 and 3 (FF d2-3). These measures are the amount of lbs of force exert. The higher number is the highest exerted out of 3 tries.
Outcome measures
| Measure |
Placebo
n=11 Participants
matching placebo injection
|
Incobotulinum Toxin Injection
n=10 Participants
Incobotulinumtoxin A used on average doses between 10U and 30 Units per subject, but at no time did any subject receive over 100 Units. Each study visit, the assessing physician selected the muscles and the amounts of medication to be injected.
|
|---|---|---|
|
Change in Motor Strength Comparison
DYN pnch 4 max
|
0.679 lbs of force exert
Standard Error 0.5954
|
-3.037 lbs of force exert
Standard Error 0.629
|
|
Change in Motor Strength Comparison
DYN pnch 4 avg
|
1.065 lbs of force exert
Standard Error 0.5688
|
-2.323 lbs of force exert
Standard Error 0.5951
|
|
Change in Motor Strength Comparison
DYN pnch 3 max
|
0.5570 lbs of force exert
Standard Error 0.7079
|
-2.905 lbs of force exert
Standard Error 0.7522
|
|
Change in Motor Strength Comparison
DYN pnch 3 Avg
|
1.1440 lbs of force exert
Standard Error 0.6354
|
-2.2 lbs of force exert
Standard Error 0.6762
|
|
Change in Motor Strength Comparison
Grip max
|
3.084 lbs of force exert
Standard Error 4.6889
|
-25.04 lbs of force exert
Standard Error 4.9933
|
|
Change in Motor Strength Comparison
Grip avg
|
4.944 lbs of force exert
Standard Error 4.721
|
-22.172 lbs of force exert
Standard Error 4.9974
|
|
Change in Motor Strength Comparison
FF D2-3
|
19.526 lbs of force exert
Standard Error 7.1284
|
-4.423 lbs of force exert
Standard Error 6.5695
|
Adverse Events
Placebo
Incobotulinum Toxin Injection
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Placebo
n=20 participants at risk
matching placebo injection
|
Incobotulinum Toxin Injection
n=21 participants at risk
Incobotulinumtoxin A used on average doses between 10U and 30 Units per subject, but at no time did any subject receive over 100 Units. Each study visit, the assessing physician selected the muscles and the amounts of medication to be injected.
|
|---|---|---|
|
Musculoskeletal and connective tissue disorders
Weakness
|
60.0%
12/20 • 24 weeks
|
33.3%
7/21 • 24 weeks
|
Additional Information
David Simpson, M.D.
Icahn School of Medicine at Mount Sinai
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place