Clinical trial NCT02107261: Incobotulinum Toxin A (Xeomin®) As A Treatment For Focal Task-Specific Dystonia Of The Musician's Hand
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 Dystonia Of The Musician's Hand
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Dystonia
Keywords: Musician's Dystonia
Lead sponsor: Icahn School of Medicine at Mount Sinai
Sponsor class: OTHER
Collaborators: name: Merz North America, Inc.
First posted: 2014-04-08
Enrollment: 21
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Physician Global Perception of Change - Blinded Rater 1
timeFrame: at visit 4 (week 8)
measure: Physician Global Perception of Change - Blinded Rater 2
timeFrame: at visit 4 (week 8)
measure: Rating of Overall Musical Performance - Blinded Rater 1
timeFrame: baseline and at 8 weeks
measure: Rating of Overall Musical Performance - Blinded Rater 2
timeFrame: baseline and week 8
measure: Quantitative MIDI Analysis Comparison
timeFrame: baseline at 24 weeks
measure: Blinded High Speed Video Analysis Comparison
timeFrame: baseline and at 24 weeks
Secondary outcomes:
measure: Change in Perceived Stress Scale (PSS)
timeFrame: baseline and at 8 weeks
measure: Change in Medical Research Council (MRC) Scale
timeFrame: baseline and at 8 weeks
measure: Change in Motor Strength Comparison
timeFrame: baseline and at 8 weeks
Other outcomes:
Locations:
city: New York
country: United States
facility: NYU Langone Health
state: New York
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
Study officials:
affiliation: NYU Langone Health
name: Steven Frucht, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Icahn School of Medicine at Mount Sinai
name: David M Simpson, MD
role: PRINCIPAL_INVESTIGATOR
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