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

Primary Purpose

Tremor, Limb

Status

Recruiting

Phase

Phase 4

Locations

United States

Study Type

Interventional

Intervention

Abobotulinum toxin A

Placebo

About this trial

This is an interventional treatment trial for Tremor, Limb focused on measuring Upper Limb, Tremor, task-dependent

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Icahn School of Medicine at Mount SinaiRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Abobotulinum toxin A

Placebo

Arm Description

Outcomes

Primary Outcome Measures

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).

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).

Secondary Outcome Measures

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).

Full Information

NCT ID

NCT03136341

First Posted

April 21, 2017

Last Updated

February 23, 2021

Sponsor

David M. Simpson

Collaborators

Ipsen

1. Study Identification

Unique Protocol Identification Number

NCT03136341

Brief Title

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

2. Study Status

Record Verification Date

February 2021

Overall Recruitment Status

Recruiting

Study Start Date

December 4, 2018 (Actual)

Primary Completion Date

December 2022 (Anticipated)

Study Completion Date

December 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

David M. Simpson

Collaborators

Ipsen

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Tremor, Limb

Keywords

Upper Limb, Tremor, task-dependent

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Crossover Assignment

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

ParticipantInvestigatorOutcomes Assessor

Masking Description

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

Allocation

Randomized

Enrollment

25 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Abobotulinum toxin A

Arm Type

Active Comparator

Arm Title

Placebo

Arm Type

Placebo Comparator

Intervention Type

Drug

Intervention Name(s)

Abobotulinum toxin A

Intervention Description

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

Intervention Type

Other

Intervention Name(s)

Placebo

Intervention Description

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

Primary Outcome Measure Information:

Title

Patient's Impression of Change

Description

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).

Time Frame

Week 6

Title

Patient's Impression of Change

Description

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).

Time Frame

Week 18

Secondary Outcome Measure Information:

Title

Physician Global Impression of Change

Description

Time Frame

Week 6

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

80 Years

Accepts Healthy Volunteers

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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Mary-Catherine George

Phone

212-241-0784

Mary-Catherine.George@mssm.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

David M Simpson, MD

Organizational Affiliation

Icahn School of Medicine at Mount Sinai

Official's Role

Principal Investigator

Facility Information:

Facility Name

Icahn School of Medicine at Mount Sinai

City

New York

State/Province

New York

ZIP/Postal Code

10029

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Mary-Catherine George

Phone

212-824-0784

mary-catherine.george@mssm.edu

First Name & Middle Initial & Last Name & Degree

David M Simpson, MD

Phone

212-241-8748

david.simpson@mssm.edu

First Name & Middle Initial & Last Name & Degree

David M Simpson, MD

12. IPD Sharing Statement

Plan to Share IPD

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18785647

Citation

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Citation

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Citation

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

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