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

NCT ID: NCT02107261

Last Updated: 2023-05-11

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-03-31

Study Completion Date

2020-01-27

Brief Summary

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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 Description

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Conditions

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Dystonia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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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.

Group Type ACTIVE_COMPARATOR

incobotulinumtoxin A

Intervention Type DRUG

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.

Placebo

Intervention Type DRUG

matching placebo

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.

Group Type ACTIVE_COMPARATOR

incobotulinumtoxin A

Intervention Type DRUG

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.

Placebo

Intervention Type DRUG

matching placebo

Interventions

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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.

Intervention Type DRUG

Placebo

matching placebo

Intervention Type DRUG

Other Intervention Names

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Xeomin

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Merz North America, Inc.

INDUSTRY

Sponsor Role collaborator

Icahn School of Medicine at Mount Sinai

OTHER

Sponsor Role lead

Responsible Party

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David M. Simpson

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Steven Frucht, MD

Role: PRINCIPAL_INVESTIGATOR

NYU Langone Health

David M Simpson, MD

Role: PRINCIPAL_INVESTIGATOR

Icahn School of Medicine at Mount Sinai

Locations

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NYU Langone Health

New York, New York, United States

Site Status

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status

Countries

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United States

References

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Jankovic J, Ashoori A. Movement disorders in musicians. Mov Disord. 2008 Oct 30;23(14):1957-65. doi: 10.1002/mds.22255.

Reference Type BACKGROUND
PMID: 18785647 (View on PubMed)

Pullman SL, Hristova AH. Musician's dystonia. Neurology. 2005 Jan 25;64(2):186-7. doi: 10.1212/01.WNL.0000157497.08500.c1. No abstract available.

Reference Type BACKGROUND
PMID: 15668411 (View on PubMed)

Altenmuller E. Focal dystonia: advances in brain imaging and understanding of fine motor control in musicians. Hand Clin. 2003 Aug;19(3):523-38, xi. doi: 10.1016/s0749-0712(03)00043-x.

Reference Type BACKGROUND
PMID: 12945651 (View on PubMed)

Frucht SJ, Fahn S, Greene PE, O'Brien C, Gelb M, Truong DD, Welsh J, Factor S, Ford B. The natural history of embouchure dystonia. Mov Disord. 2001 Sep;16(5):899-906. doi: 10.1002/mds.1167.

Reference Type BACKGROUND
PMID: 11746620 (View on PubMed)

Elbert T, Pantev C, Wienbruch C, Rockstroh B, Taub E. Increased cortical representation of the fingers of the left hand in string players. Science. 1995 Oct 13;270(5234):305-7. doi: 10.1126/science.270.5234.305.

Reference Type BACKGROUND
PMID: 7569982 (View on PubMed)

Munte TF, Altenmuller E, Jancke L. The musician's brain as a model of neuroplasticity. Nat Rev Neurosci. 2002 Jun;3(6):473-8. doi: 10.1038/nrn843.

Reference Type BACKGROUND
PMID: 12042882 (View on PubMed)

Altenmuller E, Baur V, Hofmann A, Lim VK, Jabusch HC. Musician's cramp as manifestation of maladaptive brain plasticity: arguments from instrumental differences. Ann N Y Acad Sci. 2012 Apr;1252:259-65. doi: 10.1111/j.1749-6632.2012.06456.x.

Reference Type BACKGROUND
PMID: 22524368 (View on PubMed)

Schmidt A, Jabusch HC, Altenmuller E, Hagenah J, Bruggemann N, Hedrich K, Saunders-Pullman R, Bressman SB, Kramer PL, Klein C. Dominantly transmitted focal dystonia in families of patients with musician's cramp. Neurology. 2006 Aug 22;67(4):691-3. doi: 10.1212/01.wnl.0000230148.00035.f9.

Reference Type BACKGROUND
PMID: 16924027 (View on PubMed)

Altenmuller E, Jabusch HC. Focal dystonia in musicians: phenomenology, pathophysiology, triggering factors, and treatment. Med Probl Perform Art. 2010 Mar;25(1):3-9.

Reference Type BACKGROUND
PMID: 20795373 (View on PubMed)

Schuele S, Jabusch HC, Lederman RJ, Altenmuller E. Botulinum toxin injections in the treatment of musician's dystonia. Neurology. 2005 Jan 25;64(2):341-3. doi: 10.1212/01.WNL.0000149768.36634.92.

Reference Type BACKGROUND
PMID: 15668436 (View on PubMed)

Pullman SL, Greene P, Fahn S, Pedersen SF. Approach to the treatment of limb disorders with botulinum toxin A. Experience with 187 patients. Arch Neurol. 1996 Jul;53(7):617-24. doi: 10.1001/archneur.1996.00550070055012.

Reference Type BACKGROUND
PMID: 8929169 (View on PubMed)

Peterson DA, Berque P, Jabusch HC, Altenmuller E, Frucht SJ. Rating scales for musician's dystonia: the state of the art. Neurology. 2013 Aug 6;81(6):589-98. doi: 10.1212/WNL.0b013e31829e6f72. Epub 2013 Jul 24.

Reference Type BACKGROUND
PMID: 23884039 (View on PubMed)

Chang FC, Frucht SJ. Motor and Sensory Dysfunction in Musician's Dystonia. Curr Neuropharmacol. 2013 Jan;11(1):41-7. doi: 10.2174/157015913804999531.

Reference Type BACKGROUND
PMID: 23814536 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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GCO 13-1679

Identifier Type: -

Identifier Source: org_study_id

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