Continued Access Protocol: ExAblate Transcranial MR Guided Focused Ultrasound for the Treatment of Essential Tremors

A Continued Access Study to Evaluate the Effectiveness and Safety of ExAblate Transcranial MRgFUS Thalamotomy Treatment of Medication Refractory Essential Tremor Subjects

The objective of this prospective, multi site, single-arm study is to capture the efficacy of treatment using the ExAblate Transcranial System and to further demonstrate safety in medication-refractory tremor in patients with essential tremor (ET).

Study Overview

• Status: Completed
• Conditions: Essential Tremor
• Intervention / Treatment: Device: Transcranial ExAblate

Detailed Description

This study is evaluating a new technique for performing Thalamotomy for tremor control. While current techniques have possible invasive or radiation effects, the use of ExAblate if totally non-invasive and without any radiation. After informed consent and screening, eligible subjects will be undergo an ExAblate treatment. All subjects will be followed at 1 day, 1 week, 1, 3, 6 and 12 months and for up to 5 years as directed by their doctor.

• Study Type: Interventional
• Enrollment (Actual): 61
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • California
    • Stanford, California, United States, 94305
      • Stanford University Medical Center
  • Maryland
    • Baltimore, Maryland, United States, 21201
      • University of Maryland Medical System
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Brigham and Women's Hospital
  • Ohio
    • Columbus, Ohio, United States, 43210
      • The Ohio State Wexner Medical Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19106
      • University of Pennsylvania
  • Virginia
    • Charlottesville, Virginia, United States, 22908
      • University of Virginia
  • Washington
    • Seattle, Washington, United States, 98122
      • Swedish Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Men and women, age 22 years and older
• Subjects who are able and willing to give informed consent and able to attend all study visits
• Subjects with a diagnosis of Essential Tremor as confirmed from clinical history and examination by a neurologist or neurosurgeon specialized in movement disorder
• Subject exhibits a significant disability from their ET despite medical treatment
• Subjects should be on a stable dose of all ET medications for 30 days prior to study entry
• Subject is able to communicate sensations during the ExAblate Transcranial procedure

Exclusion Criteria:

• Subjects with unstable cardiac status
• Severe hypertension
• Subjects with standard contraindications for MR imaging such as non-MRI compatible implanted metallic devices including cardiac pacemakers, size limitations, etc.
• Known intolerance or allergies to the MRI contrast agent including advanced kidney disease or severely impaired renal function
• Significant claustrophobia that cannot be managed with mild medication
• Current medical condition resulting in abnormal bleeding and/or coagulopathy
• Receiving anticoagulant (e.g. warfarin) or antiplatelet (e.g. aspirin) therapy within one week of focused ultrasound procedure or drugs known to increase risk or hemorrhage
• History of intracranial hemorrhage
• History of multiple strokes, or a stroke within past 6 months
• Subjects who are not able or willing to tolerate the required prolonged stationary supine position during treatment
• Are participating or have participated in another clinical trial in the last 30 days
• Subjects unable to communicate with the investigator and staff
• Subjects with a history of seizures within the past year
• Subjects with brain tumors

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Transcranial ExAblate	Device: Transcranial ExAblate; Transcranial ExAblate; Other Names: Thalamotomy; ExAblate; TcMRgFUS

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Device and Procedure Related Adverse Events.	The cumulative sum of adverse events was followed through Year 5 of the study.	5 Years post treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Tremor Motor Score - Clinical Rating Scale for Tremor (CRST) Part A (Upper Extremity) + Part B Scores Summed	Sum of baseline and follow-up in upper extremity Tremor-Motor scores for the treated side is a sub-scale of Clinical Rating Scale for Tremor (CRST) Part A and Part B sum that was used to measure treated-side upper extremity tremor changes over time. Tremor-motor scores range from 0-32 points. Individual subject's scores at Baseline and Follow Up were calculated at baseline and follow-up visits averaged across subjects. Low scores are better (show improvement).	Baseline, 3 Months, 6 Months, 12 Months, 2, 3, 4, 5 Years post treatment
Clinical Rating Scale for Tremor (CRST Part A) Posture Score Change From Baseline	The Posture Score is a sub-Score of the Clinical Rating Scale for Tremor CRST Part A - Posture (Treated Side Upper Extremity Posture Score). The minimum-maximum average score range is 0-4 and higher scores indicate worse outcomes.	Baseline, 3 Months, 6 Months, 12 Months, 2, 3, 4, 5 Years post treatment
Clinical Rating Scale for Tremor Part C - Activities of Daily Living.	Subject daily functionalities as measured by Clinical Rating Scale for Tremor Part C, CRST Part-C (subscales). CRST Part-C is an 8-item score with a minimum-maximum range of 0-32. Higher scores mean worse outcomes.	Baseline, 3 Months, 6 Months, 12 Months, 2, 3, 4, 5 Years post treatment
Quality of Life in Essential Tremor Total (QUEST)	The QUEST Summary Total was used to assess quality of life changes over time in tremor patients. The QUEST consists of five dimensions for each subject converted to a percent of the total (0-100%). The summary total is the average of the five dimensions. Low Total scores are better.	Baseline, 3 Months, 6 Months, 12 Months, 2, 3, 4, 5 Years post treatment

Collaborators and Investigators

• Sponsor: InSightec

Study record dates

Study Major Dates

• Study Start (Actual): April 1, 2015
• Primary Completion (Actual): February 18, 2022
• Study Completion (Actual): March 10, 2023

Study Registration Dates

• First Submitted: November 10, 2014
• First Submitted That Met QC Criteria: November 12, 2014
• First Posted (Estimated): November 13, 2014

Study Record Updates

• Last Update Posted (Actual): July 3, 2024
• Last Update Submitted That Met QC Criteria: June 10, 2024
• Last Verified: June 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Movement Disorders; Dyskinesias; Tremor; Essential Tremor
• Other Study ID Numbers: ET002CA

Plan for Individual participant data (IPD)

Study Data/Documents

1. Clinical Study Report