Clinical trial NCT04501484: Bilateral Essential Tremor Treatment With FUS
Official title: Bilateral Essential Tremor Treatment With Magnetic Resonance-guided Focused Ultrasound
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Essential Tremor
Keywords: High-Intensity Focused Ultrasound Ablation
Lead sponsor: University Health Network, Toronto
Sponsor class: OTHER
Collaborators: name: Université de Sherbrooke
First posted: 2020-08-06
Enrollment: 19
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Change in QUEST Score
timeFrame: 12 months
measure: Patient-based Assessment of Utility
timeFrame: 12 months
Secondary outcomes:
measure: Gait Assessment (NRS-11)
timeFrame: Immediately after surgery, post operative month 1, 3, 12, 24, 36
measure: Gait Assessment (missteps)
timeFrame: Immediately after surgery, post operative month 1, 3, 12, 24, 36
measure: Falls
timeFrame: Post operative month 1, 3, 12, 24, 36
measure: Speech Assessment (NRS-11)
timeFrame: Immediately after surgery, post operative month 1, 3, 12, 24, 36
measure: Numbness
timeFrame: Immediately after surgery, post operative month 1, 3, 12, 24, 36
measure: Dysgeusia
timeFrame: Immediately after surgery, post operative month 1, 3, 12, 24, 36
measure: Other adverse events
timeFrame: Immediately after surgery, post operative month 1, 3, 12, 24, 36
measure: Tremor (CRST)
timeFrame: Immediately after surgery, post operative month 1, 3, 12, 24, 36
measure: Quality of life (QUEST)
timeFrame: Post operative month 1, 3, 12, 24, 36
measure: Quality of life (CRST part C)
timeFrame: Post operative month 1, 3, 12, 24, 36
measure: Health Utility (EQ-5D-5L)
timeFrame: Post operative month 1, 3, 12, 24, 36
Other outcomes:
Locations:
city: Toronto
country: Canada
facility: Toronto Western Hospital, University Health Network
state: Ontario
Study officials:
affiliation: University of Toronto
name: Andres Lozano, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: