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

NCT03136341 | Recruiting Phase 4 FDA Drug

• 1 other identifier: GCO 17-0592
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Tremor, Limb

Keywords

Upper Limb; Tremor; task-dependent

Outcome Measures

Primary Outcomes (2)

• Patient's Impression of Change

  The principal outcome measure will be patient's impression of change using visual analog scale. Visual Analog Scale (VAS) 100 point scale from 0 (no improvement) to 100 (normal use).

  Week 6

• Patient's Impression of Change

  The principal outcome measure will be patient's impression of change using visual analog scale. Visual Analog Scale is a 100 point scale from 0 (no improvement) to 100 (normal use).

  Week 18

Secondary Outcomes (1)

• Physician Global Impression of Change

  Week 6

Study Arms (2)

Abobotulinum toxin A

ACTIVE COMPARATOR

Drug: Abobotulinum toxin A

Placebo

PLACEBO COMPARATOR

Other: Placebo

Interventions

Abobotulinum toxin A DRUG

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

Abobotulinum toxin A

Placebo OTHER

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

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• David M. Simpson: lead
• Ipsen: collaborator

Study Sites (1)

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

RECRUITING

Related Publications (14)

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• 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.

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• 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.

  PMID: 7569982 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.

  PMID: 12042882 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.

  PMID: 20795373 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.

  PMID: 15668436 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.

  PMID: 8929169 BACKGROUND

• 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.

  PMID: 23458295 BACKGROUND

• 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.

  PMID: 25834463 BACKGROUND

• 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.

  PMID: 11402109 BACKGROUND

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• 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.

  PMID: 16103000 BACKGROUND

MeSH Terms

Conditions

Tremor

Interventions

abobotulinumtoxinA

Condition Hierarchy (Ancestors)

Dyskinesias; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• David M Simpson, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mary-Catherine George

CONTACT

212-241-0784; Mary-Catherine.George@mssm.edu

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Videos will be randomly provided to an outside assessor to determine benefit as a secondary outcome measure
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Model Details: 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

Study Record Dates

• First Submitted: April 21, 2017
• First Posted: May 2, 2017
• Study Start: December 4, 2018
• Primary Completion: December 1, 2022
• Study Completion: December 1, 2022
• Last Updated: February 25, 2021

Record last verified: 2021-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)