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Completed

NCT Number: NCT02107261

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

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.

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Key information

Conditions

Dystonia Dyskinesias Dystonia, Focal, Task-Specific Nervous System Diseases Neurologic Manifestations Pathological Conditions, Signs and Symptoms Signs and Symptoms

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

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

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

incobotulinumtoxin A

Drug

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.

Other names: Xeomin

Placebo

Drug

matching placebo

Primary outcomes

  1. Physician Global Perception of Change - Blinded Rater 1

    Time frame: 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.

  2. Physician Global Perception of Change - Blinded Rater 2

    Time frame: 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.

  3. Rating of Overall Musical Performance - Blinded Rater 1

    Time frame: 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.

  4. Rating of Overall Musical Performance - Blinded Rater 2

    Time frame: 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.

  5. Quantitative MIDI Analysis Comparison

    Time frame: 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.

  6. Blinded High Speed Video Analysis Comparison

    Time frame: 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 outcomes

  1. Change in Perceived Stress Scale (PSS)

    Time frame: 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

  2. Change in Medical Research Council (MRC) Scale

    Time frame: 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.

  3. Change in Motor Strength Comparison

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Icahn School of Medicine at Mount Sinai

Other

Collaborators

  • Merz North America, Inc.

Registry information

Official study title

A Placebo-Controlled, Double-Blind, Randomized, Cross Over Pilot Study Of The Efficacy And Tolerability Of Incobotulinum Toxin A (Xeomin®) As A Treatment For Focal Task-Specific Dystonia Of The Musician's Hand

Important dates

Study start
2014
Primary completion
2020
Study completion
2020
First posted
Apr 8, 2014
Registry last updated
May 11, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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