A Cross Over Pilot Study of Abobotulinum Toxin A (Dysport) as a Treatment for Task-dependent Upper Limb Tremor

NCT ID: NCT03136341

Last Updated: 2021-02-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE4

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-12-04

Study Completion Date

2022-12-31

Brief Summary

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Task-dependent upper limb tremor is a disorder that frequently impairs patients' function and quality of life. Few studies exist that quantify the improvements in motor performance and disability with botulinum toxin (injections). The study team aims to perform an exploratory pilot trial using toxin Type A (Dysport) in 25 patients with task-dependent upper limb tremor, to refine quantitative measures of performance using blinded video analysis, demonstrate efficacy and tolerability of injections, and provide power calculations for a potential future multi-centered double blinded clinical trial.

Detailed Description

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Conditions

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Tremor, Limb

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Participants are randomized to receive either study drug or placebo at the initial phase and cross over 12 weeks later and receive the alternative treatment
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors
Videos will be randomly provided to an outside assessor to determine benefit as a secondary outcome measure

Study Groups

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Abobotulinum toxin A

Group Type ACTIVE_COMPARATOR

Abobotulinum toxin A

Intervention Type DRUG

Dosing will be determined at each study visit based upon muscles involved

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Dosing will be determined at each study visit based upon muscles involved

Interventions

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Abobotulinum toxin A

Dosing will be determined at each study visit based upon muscles involved

Intervention Type DRUG

Placebo

Dosing will be determined at each study visit based upon muscles involved

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Patients with task-dependent essential tremor in upper limb. Tremor is present during at least one of the study functional tasks i. pointing task (touch between nose and a pen held 2 ft. in front. ii. pouring task (cup: pouring water out/in). iii. feeding task (weighted fork from plate on table to mouth). iv. string instrument bowing or holding instrument in static position. v. computer mouse (simple vertical or horizontal movements).
* Patients must be capable of performing informed consent.
* Patients on stable medications for treatment of task-dependent essential tremor for 1 month prior to study entry and maintain them throughout the study.
* Patients previously treated with BoNT of any type must have occurred no sooner than 12 weeks prior to study entry.

Exclusion Criteria

* Patients with no presence of tremor during study functional tasks.
* Presence of tremor during the functional task is below minimum amplitude required to be considered for injection based upon investigator's clinical judgment.
* Patients with unstable medical conditions or psychiatric conditions.
* Patients with a medical condition that precludes them from receiving BoNT injections.
* Pregnant women, or premenopausal women not willing to use contraceptive measures throughout the duration of the study.
* Lithium, Valproic acid, Amiodarone, typical and atypical neuroleptics. Exposure to other than the listed tremorogenic or potentially tremorogenic drugs is allowed only if, in the opinion of the investigator, this will not interfere with the study drug evaluations.
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ipsen

INDUSTRY

Sponsor Role collaborator

David M. Simpson

OTHER

Sponsor Role lead

Responsible Party

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David M. Simpson

Professor

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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David M Simpson, MD

Role: PRINCIPAL_INVESTIGATOR

Icahn School of Medicine at Mount Sinai

Locations

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Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Mary-Catherine George

Role: CONTACT

212-241-0784

Facility Contacts

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Mary-Catherine George

Role: primary

212-824-0784

David M Simpson, MD

Role: backup

212-241-8748

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, 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)

Carruthers J, Fournier N, Kerscher M, Ruiz-Avila J, Trindade de Almeida AR, Kaeuper G. The convergence of medicine and neurotoxins: a focus on botulinum toxin type A and its application in aesthetic medicine--a global, evidence-based botulinum toxin consensus education initiative: part II: incorporating botulinum toxin into aesthetic clinical practice. Dermatol Surg. 2013 Mar;39(3 Pt 2):510-25. doi: 10.1111/dsu.12148.

Reference Type BACKGROUND
PMID: 23458295 (View on PubMed)

Pavicic T, Prager W, Kloppel M, Ravichandran S, Galatoire O. IncobotulinumtoxinA use in aesthetic indications in daily practice: a European multicenter, noninterventional, retrospective study. Clin Cosmet Investig Dermatol. 2015 Mar 5;8:135-42. doi: 10.2147/CCID.S74519. eCollection 2015.

Reference Type BACKGROUND
PMID: 25834463 (View on PubMed)

Brin MF, Lyons KE, Doucette J, Adler CH, Caviness JN, Comella CL, Dubinsky RM, Friedman JH, Manyam BV, Matsumoto JY, Pullman SL, Rajput AH, Sethi KD, Tanner C, Koller WC. A randomized, double masked, controlled trial of botulinum toxin type A in essential hand tremor. Neurology. 2001 Jun 12;56(11):1523-8. doi: 10.1212/wnl.56.11.1523.

Reference Type BACKGROUND
PMID: 11402109 (View on PubMed)

Louis ED, Barnes L, Wendt KJ, Ford B, Sangiorgio M, Tabbal S, Lewis L, Kaufmann P, Moskowitz C, Comella CL, Goetz CC, Lang AE. A teaching videotape for the assessment of essential tremor. Mov Disord. 2001 Jan;16(1):89-93. doi: 10.1002/1531-8257(200101)16:13.0.co;2-l.

Reference Type BACKGROUND
PMID: 11215599 (View on PubMed)

Troster AI, Pahwa R, Fields JA, Tanner CM, Lyons KE. Quality of life in Essential Tremor Questionnaire (QUEST): development and initial validation. Parkinsonism Relat Disord. 2005 Sep;11(6):367-73. doi: 10.1016/j.parkreldis.2005.05.009.

Reference Type BACKGROUND
PMID: 16103000 (View on PubMed)

Other Identifiers

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GCO 17-0592

Identifier Type: -

Identifier Source: org_study_id

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