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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official 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 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 Summary:
Sponsor: Icahn School of Medicine at Mount Sinai

Current Primary Outcome:

  • Patient and Physician Global Perception of change [ Time Frame: 4 weeks 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 at 4 weeks.
  • Quantitative MIDI Analysis comparison [ Time Frame: baseline and at 4 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 4 weeks.
  • Blinded high speed video analysis comparison [ Time Frame: baseline at 4 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 4 weeks.
  • 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.
  • Blinded high speed video analysis comparison [ Time Frame: baseline and at 24 weeks ]
    The principal outcome measure will be improvement in musical pe

    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    • Perceived Stress Scale [ Time Frame: baseline and at 24 weeks ]
      Patient reported outcomes will be measured by questionnaires that address adverse events and the impact of quality of life using the perceived stress scale.
    • Motor Strength comparison [ Time Frame: baseline and at 4 weeks ]
      Motor strength will be tested using a dynamometer to document any weakness the treatment may produce as compared to the baseline visit and 4 weeks.
    • Motor Strength comparison [ Time Frame: baseline and at 24 weeks ]
      Motor strength will be tested using a dynamometer to document any weakness the treatment may produce as compared to the baseline visit and 24 weeks.


    Original Secondary Outcome: Same as current

    Information By: Icahn School of Medicine at Mount Sinai

    Dates:
    Date Received: March 26, 2014
    Date Started: March 2014
    Date Completion: June 2018
    Last Updated: September 14, 2016
    Last Verified: September 2016