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

April 19, 2023 updated by: David M. Simpson, Icahn School of Medicine at Mount Sinai

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

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 Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

21

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New York
      • New York, New York, United States, 10029
        • Icahn School of Medicine at Mount Sinai
      • New York, New York, United States, 10017
        • NYU Langone Health

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: 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.
Drug: Placebo
matching placebo
Drug: incobotulinumtoxin A
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
Active Comparator: 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.
Drug: Placebo
matching placebo
Drug: incobotulinumtoxin A
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
at visit 4 (week 8)
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.
at visit 4 (week 8)
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.
baseline and at 8 weeks
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.
baseline and week 8
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.
baseline at 24 weeks
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.
baseline and at 24 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
baseline and at 8 weeks
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.
baseline and at 8 weeks
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.
baseline and at 8 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Icahn School of Medicine at Mount Sinai

Collaborators

Merz North America, Inc.

Investigators

  • Principal Investigator: David M Simpson, MD, Icahn School of Medicine at Mount Sinai
  • Principal Investigator: Steven Frucht, MD, NYU Langone Health

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2014

Primary Completion (Actual)

January 27, 2020

Study Completion (Actual)

January 27, 2020

Study Registration Dates

First Submitted

March 26, 2014

First Submitted That Met QC Criteria

April 4, 2014

First Posted (Estimate)

April 8, 2014

Study Record Updates

Last Update Posted (Estimate)

May 11, 2023

Last Update Submitted That Met QC Criteria

April 19, 2023

Last Verified

April 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GCO 13-1679

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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