The investigational drug being studied in this protocol is Incobotulinumtoxin A (Xeomin®). Botulinum toxin (BoNT) prevents the release of the acetylcholine from peripheral nerves, inhibiting muscle contractions. BoNT is effective in relaxing overactive muscles. In musician's dystonia, the ability to reduce abnormally overactive muscles in the hand can be critical for the musical professional to continue his or her career. With the use of EMG/electrical stimulation and/or ultrasound guidance, the injector can precisely localize the individual muscles that are affected in this condition with great accuracy. Prior studies have shown that BoNT injections produce beneficial effects in forearm muscles, and less effect in shoulder or proximal arm muscles. Possible risks in treating patients with BoNT include excessive weakness of the injected muscles. The drug may also affect non-targeted muscles. However these risks will be minimized during the screening period by carefully targeting the affected muscles and by administering low doses of BoNT. Small booster doses may be given at follow up visit (2, 4, 14 and 16-weeks after the primary injection date) if the initial injection was insufficient to produce sufficient efficacy in relief of the focal dystonia and did not produce excess weakness of the targeted muscle.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
21
Incobotulinumtoxin A is being used on average doses between 10U and 30 Units per subject, but at no time will any subject receive over 100 Units. Each study visit, the assessing physician will select the muscles and the amounts of medication to be injected.
matching placebo
NYU Langone Health
New York, New York, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Physician Global Perception of Change - Blinded Rater 1
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.
Time frame: at visit 4 (week 8)
Physician Global Perception of Change - Blinded Rater 2
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.
Time frame: at visit 4 (week 8)
Rating of Overall Musical Performance - Blinded Rater 1
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.
Time frame: baseline and at 8 weeks
Rating of Overall Musical Performance - Blinded Rater 2
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.
Time frame: baseline and week 8
Quantitative MIDI Analysis Comparison
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.
Time frame: baseline at 24 weeks
Blinded High Speed Video Analysis Comparison
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.
Time frame: baseline and at 24 weeks
Change in Perceived Stress Scale (PSS)
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
Time frame: baseline and at 8 weeks
Change in Medical Research Council (MRC) Scale
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.
Time frame: baseline and at 8 weeks
Change in Motor Strength Comparison
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.
Time frame: baseline and at 8 weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.