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

Mount Sinai Health System

Status and phase: Completed; Phase 2

Conditions: Dystonia

Treatments: Drug: Placebo; Drug: incobotulinumtoxin A

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02107261

GCO 13-1679

Details and patient eligibility

About

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.

Enrollment: 21 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Double Blind

---

21 participants in 2 patient groups

Placebo Then Botulinum Toxin

Active Comparator group

Description:

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.

Treatment:

Drug: Placebo

Drug: incobotulinumtoxin A

Incobotulinumtoxin A Then Placebo

Active Comparator group

Description:

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.

Treatment:

Drug: Placebo

Drug: incobotulinumtoxin A