Bilateral Essential Tremor Treatment With FUS (BEST-FUS)
Primary Purpose
Essential Tremor
Status
Recruiting
Phase
Not Applicable
Locations
Canada
Study Type
Interventional
Intervention
Magnetic Resonance-guided Focused Ultrasound VIM Thalamotomy
Sponsored by
About this trial
This is an interventional treatment trial for Essential Tremor focused on measuring High-Intensity Focused Ultrasound Ablation
Eligibility Criteria
Inclusion Criteria:
- Patients must be ≥ 18 years
- The patient has a primary diagnosis of ET or ET plus confirmed by a movement disorder neurologist;
- The patient previously underwent a MRgFUS (> 6 months ago);
- The tremor on the untreated side negatively impacts the patients' quality of life;
- The patient wants treatment of the contralateral side.
Exclusion Criteria:
- Clinically relevant gait and/or balance impairment (e.g. wide-based, more than 2 falls/months, wheelchair, walker, cane use)
- Clinically relevant speech impairment (e.g. impairment of intelligibility)
- Inability to comply with the follow-up schedule;
- Refusal of the treating physician.
Sites / Locations
- Toronto Western Hospital, University Health NetworkRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Treatment Group
Arm Description
Participants will undergo a thalamotomy contralateral to their previous treatment with MRgFUS using ExAblate Neuro 4000 device (InSightec Ltd, Tirat Carmel, Israel).
Outcomes
Primary Outcome Measures
Change in QUEST Score
Difference in QUEST score between baseline and the 12-month follow-up, with statistical significance defined as p = 0.05 and a minimum clinically important difference (MCID) defined as 5
Patient-based Assessment of Utility
A patient-reported assessment of utility after 12 months using the question "Knowing what you know now, would you do this treatment again?".
Secondary Outcome Measures
Gait Assessment (NRS-11)
Patient-reported and blinded observer rating of the patient's ability to walk (Numerical rating scale (NRS-11) from 0 to 10, with 10 being best)
Gait Assessment (missteps)
Number of missteps in a 6 m standardized tandem walk
Falls
Number of falls in last month
Speech Assessment (NRS-11)
Patient-reported and blinded observer rating of the patient's ability to speak (Numerical rating scale (NRS-11) from 0 to 10, with 10 being best)
Numbness
Incidence of new-onset numbness
Dysgeusia
Incidence of taste alterations
Other adverse events
Incidence of other adverse events as classified in the CTCAE
Tremor (CRST)
Assessment of tremor using the Clinical Rating Scale for Tremor (CRST) parts A (0-88) and B (0-36), higher scores correspond to a worse tremor).
Quality of life (QUEST)
QUality of life in ESsential Tremor (QUEST) questionnaire (0-100%, higher scores correspond to a worse quality of life)
Quality of life (CRST part C)
Part C of the Clinical Rating Scale for Tremor (CRST) (0-32, higher scores correspond to a worse quality of life)
Health Utility (EQ-5D-5L)
Health Utility measured using the EQ-5D-5L and computed using the Canadian preference weights. Values range from 0 to 1, with 1 representing a perfect health.
Full Information
NCT ID
NCT04501484
First Posted
July 29, 2020
Last Updated
May 16, 2022
Sponsor
University Health Network, Toronto
Collaborators
Université de Sherbrooke
1. Study Identification
Unique Protocol Identification Number
NCT04501484
Brief Title
Bilateral Essential Tremor Treatment With FUS
Acronym
BEST-FUS
Official Title
Bilateral Essential Tremor Treatment With Magnetic Resonance-guided Focused Ultrasound
Study Type
Interventional
2. Study Status
Record Verification Date
May 2022
Overall Recruitment Status
Recruiting
Study Start Date
July 8, 2020 (Actual)
Primary Completion Date
October 2024 (Anticipated)
Study Completion Date
October 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University Health Network, Toronto
Collaborators
Université de Sherbrooke
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Combined Phase II/III, multi-center, prospective, single-blinded trial.
Ten (10) patients with who previously underwent successful and uncomplicated MRgFUS thalamotomy for essential tremor will undergo a contralateral treatment. The incidence of side effects will be determined at 1 and 3 months postoperatively, graded per the CTCAE v5 and analyzed by a data safety monitoring board. Upon successful review, this Phase II trial will be converted to a Phase III trial of utility that will enrol 40 additional patients. The primary outcome will be the change in QUEST score at 12 months postoperatively, as well as a patient-reported assessment of Health Utility. Secondary outcomes will include objective tremor, gait and speech assessments (filmed and scored by blinded evaluators), as well as quality of life questionnaires and adverse events questionnaires. Outcomes will be assessed immediately after the procedure, as well as 1, 3, 12, 24 and 36 months post-operatively.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Essential Tremor
Keywords
High-Intensity Focused Ultrasound Ablation
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Masking Description
All assessments filmed on video and scored by a blinded evaluator.
Allocation
N/A
Enrollment
50 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Treatment Group
Arm Type
Experimental
Arm Description
Participants will undergo a thalamotomy contralateral to their previous treatment with MRgFUS using ExAblate Neuro 4000 device (InSightec Ltd, Tirat Carmel, Israel).
Intervention Type
Procedure
Intervention Name(s)
Magnetic Resonance-guided Focused Ultrasound VIM Thalamotomy
Intervention Description
Ablation of contralateral VIM nucleus of thalamus with MRgFUS using ExAblate Neuro 4000 Device (InSightec Ltd, Tirat Carmel, Israel)
Primary Outcome Measure Information:
Title
Change in QUEST Score
Description
Difference in QUEST score between baseline and the 12-month follow-up, with statistical significance defined as p = 0.05 and a minimum clinically important difference (MCID) defined as 5
Time Frame
12 months
Title
Patient-based Assessment of Utility
Description
A patient-reported assessment of utility after 12 months using the question "Knowing what you know now, would you do this treatment again?".
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Gait Assessment (NRS-11)
Description
Patient-reported and blinded observer rating of the patient's ability to walk (Numerical rating scale (NRS-11) from 0 to 10, with 10 being best)
Time Frame
Immediately after surgery, post operative month 1, 3, 12, 24, 36
Title
Gait Assessment (missteps)
Description
Number of missteps in a 6 m standardized tandem walk
Time Frame
Immediately after surgery, post operative month 1, 3, 12, 24, 36
Title
Falls
Description
Number of falls in last month
Time Frame
Post operative month 1, 3, 12, 24, 36
Title
Speech Assessment (NRS-11)
Description
Patient-reported and blinded observer rating of the patient's ability to speak (Numerical rating scale (NRS-11) from 0 to 10, with 10 being best)
Time Frame
Immediately after surgery, post operative month 1, 3, 12, 24, 36
Title
Numbness
Description
Incidence of new-onset numbness
Time Frame
Immediately after surgery, post operative month 1, 3, 12, 24, 36
Title
Dysgeusia
Description
Incidence of taste alterations
Time Frame
Immediately after surgery, post operative month 1, 3, 12, 24, 36
Title
Other adverse events
Description
Incidence of other adverse events as classified in the CTCAE
Time Frame
Immediately after surgery, post operative month 1, 3, 12, 24, 36
Title
Tremor (CRST)
Description
Assessment of tremor using the Clinical Rating Scale for Tremor (CRST) parts A (0-88) and B (0-36), higher scores correspond to a worse tremor).
Time Frame
Immediately after surgery, post operative month 1, 3, 12, 24, 36
Title
Quality of life (QUEST)
Description
QUality of life in ESsential Tremor (QUEST) questionnaire (0-100%, higher scores correspond to a worse quality of life)
Time Frame
Post operative month 1, 3, 12, 24, 36
Title
Quality of life (CRST part C)
Description
Part C of the Clinical Rating Scale for Tremor (CRST) (0-32, higher scores correspond to a worse quality of life)
Time Frame
Post operative month 1, 3, 12, 24, 36
Title
Health Utility (EQ-5D-5L)
Description
Health Utility measured using the EQ-5D-5L and computed using the Canadian preference weights. Values range from 0 to 1, with 1 representing a perfect health.
Time Frame
Post operative month 1, 3, 12, 24, 36
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients must be ≥ 18 years
The patient has a primary diagnosis of ET or ET plus confirmed by a movement disorder neurologist;
The patient previously underwent a MRgFUS (> 6 months ago);
The tremor on the untreated side negatively impacts the patients' quality of life;
The patient wants treatment of the contralateral side.
Exclusion Criteria:
Clinically relevant gait and/or balance impairment (e.g. wide-based, more than 2 falls/months, wheelchair, walker, cane use)
Clinically relevant speech impairment (e.g. impairment of intelligibility)
Inability to comply with the follow-up schedule;
Refusal of the treating physician.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Martha Lenis, BHA
Phone
+14166035800
Ext
2797
Email
martha.lenis@uhnresearch.ca
First Name & Middle Initial & Last Name or Official Title & Degree
Christian Iorio-Morin, MD, PhD
Email
christian.iorio-morin@usherbrooke.ca
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Andres Lozano, MD, PhD
Organizational Affiliation
University of Toronto
Official's Role
Principal Investigator
Facility Information:
Facility Name
Toronto Western Hospital, University Health Network
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5T 2S8
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Martha Lenis, BHA
Phone
+14166035800
Ext
2797
Email
martha.lenis@uhnresearch.ca
12. IPD Sharing Statement
Plan to Share IPD
No
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Bilateral Essential Tremor Treatment With FUS
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