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

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by Mount Sinai School of Medicine
Sponsor:
Collaborator:
Merz Pharmaceuticals, LLC
Information provided by (Responsible Party):
David M. Simpson, Mount Sinai School of Medicine
ClinicalTrials.gov Identifier:
NCT02107261
First received: March 26, 2014
Last updated: April 4, 2014
Last verified: April 2014
  Purpose

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.


Condition Intervention Phase
Dystonia
Drug: incobotulinumtoxin A
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Mount Sinai School of Medicine:

Primary Outcome Measures:
  • Patient and Physician Global Perception of change [ Time Frame: 4 weeks and at 24 weeks ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 Outcome Measures:
  • Perceived Stress Scale [ Time Frame: baseline and at 24 weeks ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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.


Estimated Enrollment: 20
Study Start Date: March 2014
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Naive to botulinum toxin
Individuals with musicians dystonia who have not been treated previously with botulinum toxin.
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 Name: Xeomin
Active Comparator: Prior treatment with botulinum toxin
Individuals with musician's dystonia who have been previously treated with botulinum toxin.
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 Name: Xeomin

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02107261

Contacts
Contact: Mary Catherine George 212-241-0784 mary-catherine.george@mssm.edu

Locations
United States, New York
Icahn School of Medicine at Mount Sinai Recruiting
New York, New York, United States, 10029
Contact: Mary Catherine George    212-241-0784    mary-catherine.george@mssm.edu   
Principal Investigator: David M Simpson, MD         
Sponsors and Collaborators
Mount Sinai School of Medicine
Merz Pharmaceuticals, LLC
Investigators
Principal Investigator: Steven Frucht, MD Mount Sinai School of Medicine
Principal Investigator: David M Simpson, MD Mount Sinai School of Medicine
  More Information

Publications:

Responsible Party: David M. Simpson, Professor, Mount Sinai School of Medicine
ClinicalTrials.gov Identifier: NCT02107261     History of Changes
Other Study ID Numbers: GCO 13-1679
Study First Received: March 26, 2014
Last Updated: April 4, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Mount Sinai School of Medicine:
Musician's Dystonia

Additional relevant MeSH terms:
Dystonia
Dystonic Disorders
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Movement Disorders
Central Nervous System Diseases
Botulinum Toxins
Botulinum Toxins, Type A
Anti-Dyskinesia Agents
Central Nervous System Agents
Therapeutic Uses
Pharmacologic Actions
Neuromuscular Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on October 16, 2014