Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Bilateral Essential Tremor Treatment With Gamma Knife (BEST-GK)

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: NCT04748640
Recruitment Status : Not yet recruiting
First Posted : February 10, 2021
Last Update Posted : February 16, 2021
Sponsor:
Collaborator:
University Health Network, Toronto
Information provided by (Responsible Party):
Christian Iorio-Morin, Université de Sherbrooke

Brief Summary:

Combined Phase II/III, multi-center, prospective, single-blinded trial.

Ten (10) patients with essential tremor who previously underwent successful and uncomplicated GK thalamotomy for essential tremor will undergo a contralateral treatment. The incidence of side effects will be determined at 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 at baseline, as well as 3, 6, 12, 24 and 36 months post-operatively.


Condition or disease Intervention/treatment Phase
Essential Tremor Procedure: Gamma Knife VIM thalamotomy Phase 2 Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Masking Description: All assessments filmed on video and scored by a blinded evaluator.
Primary Purpose: Treatment
Official Title: Bilateral Essential Tremor Treatment With Gamma Knife
Estimated Study Start Date : February 2021
Estimated Primary Completion Date : February 2026
Estimated Study Completion Date : October 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Tremor

Arm Intervention/treatment
Experimental: Treatment group
Participants will undergo a thalamotomy contralateral to their previous treatment with Gamma Knife using a frame-based, Gamma Knife Perfexion or Icon unit (Elekta, Stockholm, Sweden).
Procedure: Gamma Knife VIM thalamotomy
Ablation of contralateral VIM nucleus of thalamus with GK using a frame-based, Gamma Knife Perfexion or Icon unit (Elekta, Stockholm, Sweden).




Primary Outcome Measures :
  1. 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

  2. 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?".


Secondary Outcome Measures :
  1. Gait Assessment (NRS-11) [ Time Frame: Baseline, post operative month 3, 6, 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)

  2. Gait Assessment (missteps) [ Time Frame: Baseline, post operative month 3, 6, 12, 24, 36 ]
    Number of missteps in a 6 m standardized tandem walk

  3. Falls [ Time Frame: Baseline, post operative month 3, 6, 12, 24, 36 ]
    Number of falls in last month

  4. Speech Assessment (NRS-11) [ Time Frame: Baseline, post operative month 3, 6, 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)

  5. Numbness [ Time Frame: Baseline, post operative month 3, 6, 12, 24, 36 ]
    Incidence of new-onset numbness

  6. Dysgeusia [ Time Frame: Baseline, post operative month 3, 6, 12, 24, 36 ]
    Incidence of taste alterations

  7. Other adverse events [ Time Frame: Baseline, post operative month 3, 6, 12, 24, 36 ]
    Incidence of other adverse events as classified in the CTCAE

  8. Tremor (CRST) [ Time Frame: Baseline, post operative month 3, 6, 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).

  9. Quality of life (QUEST) [ Time Frame: Baseline, post operative month 3, 6, 12, 24, 36 ]
    QUality of life in ESsential Tremor (QUEST) questionnaire (0-100 percent, higher scores correspond to a worse quality of life)

  10. Quality of life (CRST part C) [ Time Frame: Baseline, post operative month 3, 6, 12, 24, 36 ]
    Part C of the Clinical Rating Scale for Tremor (CRST) (0-32, higher scores correspond to a worse quality of life)

  11. Health Utility (EQ-5D-5L) [ Time Frame: Baseline, post operative month 3, 6, 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.



Information from the National Library of Medicine

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.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 GK thalamotomy (> 12 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.

Information from the National Library of Medicine

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): NCT04748640


Contacts
Layout table for location contacts
Contact: Christian Iorio-Morin 819-346-1110 christian.iorio-morin@usherbrooke.ca

Locations
Layout table for location information
Canada, Ontario
Toronto Western Hospital, University Health Network
Toronto, Ontario, Canada, M5T 2S8
Contact: Martha Lenis, BHA    +14166035800 ext 2797    martha.lenis@uhnresearch.ca   
Canada, Quebec
CIUSSS de l'Estrie-CHUS - Hôpital Fleurimont
Sherbrooke, Quebec, Canada
Contact: Christian Iorio-Morin    819-346-1110    christian.iorio-morin@usherbrooke.ca   
Sponsors and Collaborators
Université de Sherbrooke
University Health Network, Toronto
Layout table for additonal information
Responsible Party: Christian Iorio-Morin, Assistant professor of neurosurgery, Université de Sherbrooke
ClinicalTrials.gov Identifier: NCT04748640    
Other Study ID Numbers: 20-5172
First Posted: February 10, 2021    Key Record Dates
Last Update Posted: February 16, 2021
Last Verified: February 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Christian Iorio-Morin, Université de Sherbrooke:
Gamma Knife Thalamotomy
Tremor
Essential Tremor
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Movement Disorders
Central Nervous System Diseases
Additional relevant MeSH terms:
Layout table for MeSH terms
Tremor
Essential Tremor
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Movement Disorders
Central Nervous System Diseases