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

Tremor, Limb Clinical Trial

Official title:

A Placebo-controlled, Double-blind, Randomized, Cross Over Pilot Study of the Efficacy and Tolerability of Abobotulinum Toxin A (Dysport) as a Treatment for Task-dependent Upper Limb Tremor

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03136341
• Other study ID #: GCO 17-0592
• Status: Recruiting
• Phase: Phase 4
• Start date: December 4, 2018
• Est. completion date: December 2022

Study information

• Verified date: February 2021
• Source: Icahn School of Medicine at Mount Sinai
• Contact: Mary-Catherine George
• Phone: 212-241-0784
• Email: Mary-Catherine.George[@]mssm.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 25
• Est. completion date: December 2022
• Est. primary completion date: December 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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.

Related Conditions & MeSH terms

• Tremor
• Tremor, Limb

Intervention

Drug:
• Abobotulinum toxin A
Dosing will be determined at each study visit based upon muscles involved

Other:
• Placebo
Dosing will be determined at each study visit based upon muscles involved

Locations

Country	Name	City	State
United States	Icahn School of Medicine at Mount Sinai	New York	New York

Sponsors (2)

Lead Sponsor	Collaborator
David M. Simpson	Ipsen

Country where clinical trial is conducted

United States, 

References & Publications (14)

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

Altenmüller E. Focal dystonia: advances in brain imaging and understanding of fine motor control in musicians. Hand Clin. 2003 Aug;19(3):523-38, xi. Review. — View Citation

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. — View Citation

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. Review. — View Citation

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. — View Citation

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. — View Citation

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

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. — View Citation

Münte TF, Altenmüller E, Jäncke L. The musician's brain as a model of neuroplasticity. Nat Rev Neurosci. 2002 Jun;3(6):473-8. Review. — View Citation

Pavicic T, Prager W, Klöppel 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. — View Citation

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. — View Citation

Pullman SL, Hristova AH. Musician's dystonia. Neurology. 2005 Jan 25;64(2):186-7. — View Citation

Schuele S, Jabusch HC, Lederman RJ, Altenmüller E. Botulinum toxin injections in the treatment of musician's dystonia. Neurology. 2005 Jan 25;64(2):341-3. — View Citation

Tröster 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. — View Citation

* Note: There are 14 references in all — Click here to view all references

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	Physician Global Impression of Change	Physician global impression of change is measured by using a visual analog scale. The CGI using a VAS is rated on a 100-point scale, with the severity of illness scale using a range of responses from 0 (no improvement) through to 100 (normal use).illness scale using a range of responses from 1 (normal) through to 7 (amongst the most severely ill patients).	Week 6