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| Name | Class |
|---|---|
| Merz North America, Inc. | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo Then Botulinum Toxin | Active Comparator | 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. |
|
| Incobotulinumtoxin A Then Placebo | Active Comparator | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| incobotulinumtoxin A | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Physician Global Perception of Change - Blinded Rater 1 | 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 | 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 | 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 | 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 | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Perceived Stress Scale (PSS) | 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Steven Frucht, MD | NYU Langone Health | Principal Investigator |
| David M Simpson, MD | Icahn School of Medicine at Mount Sinai | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| NYU Langone Health | New York | New York | 10017 | United States | ||
| Icahn School of Medicine at Mount Sinai |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 18785647 | Background | Jankovic J, Ashoori A. Movement disorders in musicians. Mov Disord. 2008 Oct 30;23(14):1957-65. doi: 10.1002/mds.22255. | |
| 15668411 | Background | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| FG001 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Cycle 1: Week 1-4 |
| |||||||||||||
| Washout: Weakness Back to Baseline |
| |||||||||||||
| Cycle 2: Week 12-15 |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| BG001 | Botulinum Toxin Then Placebo |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Physician Global Perception of Change - Blinded Rater 1 | 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. | Data not collected for one participant. Data results for Week 8 before crossover treatment. | Posted | Count of Participants | Participants | at visit 4 (week 8) |
|
24 weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | matching placebo injection | 0 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Weakness | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| David Simpson, M.D. | Icahn School of Medicine at Mount Sinai | 2122418748 | david.simpson@mssm.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Oct 22, 2019 | Jun 3, 2022 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D004421 | Dystonia |
| C566973 | Dystonia, Focal, Task-Specific |
| ID | Term |
|---|---|
| D020820 | Dyskinesias |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
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| ID | Term |
|---|---|
| C545476 | incobotulinumtoxinA |
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|
|
| Placebo | Drug | matching placebo |
|
| Blinded High Speed Video Analysis Comparison | 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 |
| baseline and at 8 weeks |
| Change in Medical Research Council (MRC) Scale | 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 | 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 |
| New York |
| New York |
| 10029 |
| United States |
| 12945651 | Background | 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. |
| 11746620 | Background | 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. |
| 7569982 | Background | 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. |
| 12042882 | Background | 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. |
| 22524368 | Background | 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. |
| 16924027 | Background | 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. |
| 20795373 | Background | Altenmuller E, Jabusch HC. Focal dystonia in musicians: phenomenology, pathophysiology, triggering factors, and treatment. Med Probl Perform Art. 2010 Mar;25(1):3-9. |
| 15668436 | Background | 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. |
| 8929169 | Background | 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. |
| 23884039 | Background | 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. |
| 23814536 | Background | Chang FC, Frucht SJ. Motor and Sensory Dysfunction in Musician's Dystonia. Curr Neuropharmacol. 2013 Jan;11(1):41-7. doi: 10.2174/157015913804999531. |
| NOT COMPLETED |
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| NOT COMPLETED |
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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.
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
|
|
| Primary | Physician Global Perception of Change - Blinded Rater 2 | 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. | Data not collected for one participant. Data results for Week 8 before crossover treatment. | Posted | Count of Participants | Participants | at visit 4 (week 8) |
|
|
|
| Primary | Rating of Overall Musical Performance - Blinded Rater 1 | 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. | Data not collected for one participant. Data results for Week 8 before crossover treatment. | Posted | Count of Participants | Participants | baseline and at 8 weeks |
|
|
|
| Primary | Rating of Overall Musical Performance - Blinded Rater 2 | 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. | Data not collected for one participant. Data results for Week 8 before crossover treatment. | Posted | Count of Participants | Participants | baseline and week 8 |
|
|
|
| Primary | Quantitative MIDI Analysis Comparison | 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. | data not collected at 24 weeks | Posted | baseline at 24 weeks |
|
|
| Primary | Blinded High Speed Video Analysis Comparison | 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. | data not collected at 24 weeks | Posted | baseline and at 24 weeks |
|
|
| Secondary | Change in Perceived Stress Scale (PSS) | 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 | Data results for Week 8 before crossover treatment. | Posted | Least Squares Mean | Standard Error | score on a scale | baseline and at 8 weeks |
|
|
|
| Secondary | Change in Medical Research Council (MRC) Scale | 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. | Data results for Week 8 before crossover treatment. | Posted | Least Squares Mean | Standard Error | score on a scale | baseline and at 8 weeks |
|
|
|
| Secondary | Change in Motor Strength Comparison | 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. | Data results for Week 8 before crossover treatment. | Posted | Least Squares Mean | Standard Error | lbs of force exert | baseline and at 8 weeks |
|
|
|
| 20 |
| 0 |
| 20 |
| 12 |
| 20 |
| EG001 | Incobotulinum Toxin Injection | Incobotulinumtoxin A used on average doses between 10U and 30 Units per subject, but at no time did any subject receive over 100 Units. Each study visit, the assessing physician selected the muscles and the amounts of medication to be injected. | 0 | 21 | 0 | 21 | 7 | 21 |
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| D013568 |
| Pathological Conditions, Signs and Symptoms |
| Minimal Improved |
|
| No Change |
|
| Minimal Worse |
|
| +1 Minimally improved |
|
| 0 No change |
|
| -1 Minimal worse |
|
| -2 Much worse |
|
| -3 Very much worse |
|
| +1 Minimally Improved |
|
| 0 No Change |
|
| -1 Minimally Worse |
|
| -2 Much worse |
|
| -3 Very much worse |
|
| DYN pnch 3 max |
|
| DYN pnch 3 Avg |
|
| Grip max |
|
| Grip avg |
|
| FF D2-3 |
|