Bilateral Essential Tremor Treatment With FUS (BEST-FUS)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04501484|
Recruitment Status : Recruiting
First Posted : August 6, 2020
Last Update Posted : May 17, 2022
- Study Details
- Tabular View
- No Results Posted
- How to Read a Study Record
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.
|Condition or disease||Intervention/treatment||Phase|
|Essential Tremor||Procedure: Magnetic Resonance-guided Focused Ultrasound VIM Thalamotomy||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||50 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Masking Description:||All assessments filmed on video and scored by a blinded evaluator.|
|Official Title:||Bilateral Essential Tremor Treatment With Magnetic Resonance-guided Focused Ultrasound|
|Actual Study Start Date :||July 8, 2020|
|Estimated Primary Completion Date :||October 2024|
|Estimated Study Completion Date :||October 2024|
Experimental: Treatment Group
Participants will undergo a thalamotomy contralateral to their previous treatment with MRgFUS using ExAblate Neuro 4000 device (InSightec Ltd, Tirat Carmel, Israel).
Procedure: Magnetic Resonance-guided Focused Ultrasound VIM Thalamotomy
Ablation of contralateral VIM nucleus of thalamus with MRgFUS using ExAblate Neuro 4000 Device (InSightec Ltd, Tirat Carmel, Israel)
- Change in QUEST Score [ Time Frame: 12 months ]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 [ Time Frame: 12 months ]A patient-reported assessment of utility after 12 months using the question "Knowing what you know now, would you do this treatment again?".
- Gait Assessment (NRS-11) [ Time Frame: Immediately after surgery, post operative month 1, 3, 12, 24, 36 ]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) [ Time Frame: Immediately after surgery, post operative month 1, 3, 12, 24, 36 ]Number of missteps in a 6 m standardized tandem walk
- Falls [ Time Frame: Post operative month 1, 3, 12, 24, 36 ]Number of falls in last month
- Speech Assessment (NRS-11) [ Time Frame: Immediately after surgery, post operative month 1, 3, 12, 24, 36 ]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 [ Time Frame: Immediately after surgery, post operative month 1, 3, 12, 24, 36 ]Incidence of new-onset numbness
- Dysgeusia [ Time Frame: Immediately after surgery, post operative month 1, 3, 12, 24, 36 ]Incidence of taste alterations
- Other adverse events [ Time Frame: Immediately after surgery, post operative month 1, 3, 12, 24, 36 ]Incidence of other adverse events as classified in the CTCAE
- Tremor (CRST) [ Time Frame: Immediately after surgery, post operative month 1, 3, 12, 24, 36 ]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) [ Time Frame: Post operative month 1, 3, 12, 24, 36 ]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) [ Time Frame: Post operative month 1, 3, 12, 24, 36 ]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) [ Time Frame: Post operative month 1, 3, 12, 24, 36 ]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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04501484
|Contact: Martha Lenis, BHA||+14166035800 ext email@example.com|
|Contact: Christian Iorio-Morin, MD, PhDfirstname.lastname@example.org|
|Toronto Western Hospital, University Health Network||Recruiting|
|Toronto, Ontario, Canada, M5T 2S8|
|Contact: Martha Lenis, BHA +14166035800 ext 2797 email@example.com|
|Principal Investigator:||Andres Lozano, MD, PhD||University of Toronto|
|Responsible Party:||Andres M. Lozano, Neurosurgeon, University Health Network, Toronto|
|Other Study ID Numbers:||
|First Posted:||August 6, 2020 Key Record Dates|
|Last Update Posted:||May 17, 2022|
|Last Verified:||May 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|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|
High-Intensity Focused Ultrasound Ablation
Nervous System Diseases
Central Nervous System Diseases