Essential TremorPhase 2/3BEST-GKNCT04748640
Bilateral Essential Tremor Treatment With Gamma Knife
A Phase 2/3 study testing Gamma Knife VIM thalamotomy in people with Essential Tremor, run by Université de Sherbrooke at 2 sites in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Essential Tremor
- What is being tested?
- Gamma Knife VIM thalamotomy
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 2 sitesCanada
- Study dates
- 8 Jun 2021to Oct 2026
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
Canada2 sites, 2 recruiting
- Recruiting
CIUSSS de l'Estrie-CHUS - Hôpital Fleurimont
Sherbrooke, Quebec
Christian Iorio-Morin, 819-346-1110, christian.iorio-morin@usherbrooke.ca
- Recruiting
Toronto Western Hospital, University Health Network
Toronto, Ontario, M5T 2S8
Martha Lenis, BHA, +14166035800 ext. 2797, martha.lenis@uhnresearch.ca
About this study
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.
- Conditions
- Essential Tremor
- Being tested
- Gamma Knife VIM thalamotomy
- Keywords
- Gamma Knife Thalamotomy, Tremor, Essential Tremor, Dyskinesias, Neurologic Manifestations, Nervous System Diseases, Movement Disorders, Central Nervous System Diseases
- Medical subject headings
- Essential Tremor, Tremor, Dyskinesias, Neurologic Manifestations, Nervous System Diseases, Movement Disorders, Central Nervous System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gamma Knife VIM thalamotomyProcedure or surgery
Ablation of contralateral VIM nucleus of thalamus with GK using a frame-based, Gamma Knife Perfexion or Icon unit (Elekta, Stockholm, Sweden).
| Study group | Role | What they receive |
|---|---|---|
| Treatment group | Experimental | 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). Receives: Procedure: Gamma Knife VIM thalamotomy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 12 months
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?".
Measured: 12 months
Secondary outcomes (11)
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)
Measured: Baseline, post operative month 3, 6, 12, 24, 36
Gait Assessment (missteps)
Number of missteps in a 6 m standardized tandem walk
Measured: Baseline, post operative month 3, 6, 12, 24, 36
Falls
Number of falls in last month
Measured: Baseline, post operative month 3, 6, 12, 24, 36
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)
Measured: Baseline, post operative month 3, 6, 12, 24, 36
Numbness
Incidence of new-onset numbness
Measured: Baseline, post operative month 3, 6, 12, 24, 36
Dysgeusia
Incidence of taste alterations
Measured: Baseline, post operative month 3, 6, 12, 24, 36
Other adverse events
Incidence of other adverse events as classified in the CTCAE
Measured: Baseline, post operative month 3, 6, 12, 24, 36
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).
Measured: Baseline, post operative month 3, 6, 12, 24, 36
Quality of life (QUEST)
QUality of life in ESsential Tremor (QUEST) questionnaire (0-100 percent, higher scores correspond to a worse quality of life)
Measured: Baseline, post operative month 3, 6, 12, 24, 36
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)
Measured: Baseline, post operative month 3, 6, 12, 24, 36
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.
Measured: Baseline, post operative month 3, 6, 12, 24, 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2021
- Study start8 Jun 2021
- Primary completionFeb 2026 (estimated)
- Study completionOct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Essential TremorStudies of the same condition, open and under way.
- More trials run by Université de SherbrookeThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Université de Sherbrooke
- Sponsor type
- University, hospital or other
- Collaborators
- University Health Network, Toronto
- Responsible party
- Principal investigator, Christian Iorio-Morin, Université de Sherbrooke
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04748640
- Sponsor's protocol ID
- 20-5172
- Acronym
- BEST-GK
- Status last verified
- Nov 2021
- First submitted
- 5 Feb 2021
- Last updated
- 17 Nov 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Bilateral Essential Tremor Treatment With Gamma Knife
- ClinicalTrials.gov ID
- NCT04748640
- Acronym / sponsor ID
- BEST-GK / 20-5172
- Conditions
- Essential Tremor
- Keywords
- Gamma Knife Thalamotomy, Tremor, Essential Tremor, Dyskinesias, Neurologic Manifestations, Nervous System Diseases, Movement Disorders, Central Nervous System Diseases
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Université de SherbrookeUniversity, hospital or other
- Collaborators
- University Health Network, Toronto
- Study start
- 8 Jun 2021 (actual)
- Primary completion
- Feb 2026 (estimated)
- Study completion
- Oct 2026 (estimated)
- First posted
- 10 Feb 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment group | Experimental | Procedure: Gamma Knife VIM thalamotomyParticipants 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). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Gamma Knife VIM thalamotomyAblation of contralateral VIM nucleus of thalamus with GK using a frame-based, Gamma Knife Perfexion or Icon unit (Elekta, Stockholm, Sweden). | Procedure or surgery | Treatment group |
Primary outcome
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?".
11 secondary outcomes
Gait Assessment (NRS-11)
Time frame: Baseline, post operative month 3, 6, 12, 24, 36
Gait Assessment (missteps)
Time frame: Baseline, post operative month 3, 6, 12, 24, 36
Falls
Time frame: Baseline, post operative month 3, 6, 12, 24, 36
Speech Assessment (NRS-11)
Time frame: Baseline, post operative month 3, 6, 12, 24, 36
Numbness
Time frame: Baseline, post operative month 3, 6, 12, 24, 36
Dysgeusia
Time frame: Baseline, post operative month 3, 6, 12, 24, 36
Other adverse events
Time frame: Baseline, post operative month 3, 6, 12, 24, 36
Tremor (CRST)
Time frame: Baseline, post operative month 3, 6, 12, 24, 36
Quality of life (QUEST)
Time frame: Baseline, post operative month 3, 6, 12, 24, 36
Quality of life (CRST part C)
Time frame: Baseline, post operative month 3, 6, 12, 24, 36
Health Utility (EQ-5D-5L)
Time frame: Baseline, post operative month 3, 6, 12, 24, 36
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Nov 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Gamma Knife VIM thalamotomy in people with Essential Tremor, run by Université de Sherbrooke at 2 sites in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 17 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Christian Iorio-Morin (819-346-1110, christian.iorio-morin@usherbrooke.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in QUEST Score (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Nov 2021.