Incobotulinum Toxin A (Xeomin®) As A Treatment For Focal Task-Specific Dystonia Of The Musician's Hand

Phase 2

Completed

• Conditions: Dystonia
• Interventions: Drug: incobotulinumtoxin A; Drug: Placebo

• Registration Number: NCT02107261
• Lead Sponsor: Icahn School of Medicine at Mount Sinai

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-03
• Study First Submitted: 2014-03-26
• Study First Submitted QC: 2014-04-04
• Study First Posted: 2014-04-08
• Primary Completion: 2020-01-27
• Study Completion: 2020-01-27
• Results First Submitted: 2022-06-08
• Status Verified: 2023-04
• Results First Submitted QC: 2023-04-19
• Last Update Submitted: 2023-04-19
• Results First Posted: 2023-05-11
• Last Update Posted: 2023-05-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 21

Inclusion Criteria

• Patients with focal task-specific dystonia of one or both hands, selectively triggered by performance on a musical instrument.
• Patients must have been evaluated by Dr. Frucht at the Movement Disorders Division of Mount Sinai Medical Center as part of their clinical care.
• Patients whose performance on an instrument is directly linked to their occupation.
• Patients must be between the ages of 18 and 80.
• Impairment in musical performance must be visible and demonstrable.

Exclusion Criteria

• Patients whose dystonia is not severe enough to interfere with musical performance in the opinion of a skilled examiner.
• Patients with unstable medical conditions or psychiatric conditions.
• Patients with a medical condition that precludes them from receiving BoNT injections.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Incobotulinumtoxin A Then Placebo	incobotulinumtoxin A	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.
Placebo Then Botulinum Toxin	Placebo	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.
Placebo Then Botulinum Toxin	incobotulinumtoxin A	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	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.

Primary Outcome Measures

Name	Time	Method
Physician Global Perception of Change - Blinded Rater 1	at visit 4 (week 8)	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.
Physician Global Perception of Change - Blinded Rater 2	at visit 4 (week 8)	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.
Rating of Overall Musical Performance - Blinded Rater 1	baseline and at 8 weeks	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.
Rating of Overall Musical Performance - Blinded Rater 2	baseline and week 8	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.
Quantitative MIDI Analysis Comparison	baseline 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.
Blinded High Speed Video Analysis Comparison	baseline and 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.

Secondary Outcome Measures

Name	Time	Method
Change in Perceived Stress Scale (PSS)	baseline and at 8 weeks	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
Change in Medical Research Council (MRC) Scale	baseline and at 8 weeks	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.
Change in Motor Strength Comparison	baseline and at 8 weeks	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.

Trial Locations

Locations (2)

NYU Langone Health; 🇺🇸 New York, New York, United States

Icahn School of Medicine at Mount Sinai; 🇺🇸 New York, New York, United States