Clinical trial NCT03136341: A Cross Over Pilot Study of Abobotulinum Toxin A (Dysport) as a Treatment for Task-dependent Upper Limb Tremor
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Tremor, Limb
Keywords: Upper Limb
Tremor
task-dependent
Lead sponsor: Icahn School of Medicine at Mount Sinai
Sponsor class: OTHER
Collaborators: name: Ipsen
First posted: 2017-05-02
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention 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
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Patient's Impression of Change
timeFrame: Week 6
measure: Patient's Impression of Change
timeFrame: Week 18
Secondary outcomes:
measure: Physician Global Impression of Change
timeFrame: Week 6
Other outcomes:
Locations:
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
Study officials:
affiliation: Icahn School of Medicine at Mount Sinai
name: David M Simpson, MD
role: PRINCIPAL_INVESTIGATOR
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