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

Study Overview
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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.

Official Title
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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

Conditions
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Tremor, Limb

Intervention / Treatment
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* Drug: Abobotulinum toxin A
* Other: Placebo


Participation Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Interventional Model Description: 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
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Abobotulinum toxin A<br> | Drug: Abobotulinum toxin A<br>* Dosing will be determined at each study visit based upon muscles involved<br>|
| Placebo Comparator: Placebo<br> | Other: Placebo<br>* Dosing will be determined at each study visit based upon muscles involved<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Upper Limb, Tremor, task-dependent