Essential TremorPhase 1NCT02037217
ExAblate Transcranial MR Guided Focused Ultrasound in the Treatment of Essential Tremor
A Phase 1 study testing ExAblate Transcranial MRgFUS System in people with Essential Tremor, run by InSightec at 1 site in South Korea.
Trial summary
A feasibility Study to Evaluate Safety and Initial Effectiveness of ExAblate Transcranial MRI-guided focused ultrasound (MRgFUS) treatment of patients with medication-refractory movement disorders, namely Essential Tremor (ET). This study is designed as a prospective, single site, single arm, nonrandomized study. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Essential Tremor
- What is being tested?
- ExAblate Transcranial MRgFUS System
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteSouth Korea
- Study dates
- Feb 2011to Jan 2014
- Phase
- Phase 1
- 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Men and women, between 18 and 80 years, inclusive
- Patients who are able and willing to give consent and able to attend all study visits
- A diagnosis of ET as confirmed from clinical history and examination by a movement disorder neurologist
- Tremor refractory to adequate trials of at least two medications, one of which should be either propranolol or primidone. An adequate medication trial is defined as a therapeutic dose of each medication or the development of side effects as the medication dose is titrated.
- Vim nucleus of thalamus can be target by the ExAblate device. The Vim region of the thalamus must be apparent on MRI such that targeting can be performed with either direct visualization or by measurement from a line connecting the anterior and posterior commissures of the brain.
- Able to communicate sensations during the ExAblate MRgFUS treatment
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- Postural or intention tremor severity score of greater than or equal to 2 in the dominant hand/arm as measured by the CRST rating scale.
- Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
- May have bilateral appendicular tremor
- Significant disability due to essential tremor despite medical treatment (CRST score of 2 or above in any one of the items 16-23 from the Disability subsection of the CRST: [speaking, feeding other than liquids, bringing liquids to mouth, hygiene, dressing, writing, working, and social activities])
- Inclusion and exclusion criteria have been agreed upon by two members of the medical team.
Who cannot join
- Patients with unstable cardiac status including:
- 1.1 Unstable angina pectoris on medication 1.2 Patients with documented myocardial infarction within six months of protocol entry 1.3 Congestive heart failure requiring medication (other than diuretic) 1.4 Patients on anti-arrhythmic drugs
- Patients exhibiting any behavior(s) consistent with ethanol or substance abuse as defined by the criteria outlined in the DSM-IV as manifested by one (or more) of the following occurring within a 12 month period:
- Recurrent substance use resulting in a failure to fulfill major role obligations at work, school, or home (such as repeated absences or poor work performance related to substance use; substance-related absences, suspensions, or expulsions from school; or neglect of children or household).
- Recurrent substance use in situations in which it is physically hazardous (such as driving an automobile or operating a machine when impaired by substance use)
- Recurrent substance-related legal problems (such as arrests for substance related disorderly conduct)
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- Continued substance use despite having persistent or recurrent social or interpersonal problems caused or exacerbated by the effects of the substance (for example, arguments with spouse about consequences of intoxication and physical fights).
- Severe hypertension (diastolic BP > 100 on medication)
- Patients 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 (e.g. Gadolinium or Magnevist) including advanced kidney disease
- Severely impaired renal function (estimated glomerular filtration rate < 45ml/min/1.73 m2) or receiving dialysis
- History of 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 (e.g. Avastin) within one month of focused ultrasound procedure
- Active or suspected acute or chronic uncontrolled infection
- History of intracranial hemorrhage
- Cerebrovascular disease (multiple CVA or CVA within 6 months)
- Individuals who are not able or willing to tolerate the required prolonged stationary supine position during treatment (can be up to 4 hrs of total table time.)
- Symptoms and signs of increased intracranial pressue (e.g. headache, nausea, vomiting, lethargy, and papilledema)
- Are participating or have participated in another clinical trial in the last 30 days
- Patients unable to communicate with the investigator and staff.
- Presence of any other neurodegenerative disease like parkinson-plus syndromes suspected on neurological examination. These include: multisystem atrophy, progressive supranuclear palsy, dementia with Lewy bodies, and Alzheimer"s disease.
- Anyone suspected to have the diagnosis of idiopathic Parkinson"s disease. This includes excluding anyone with the presence of parkinsonian features including bradykinesia rigidity, or postural instability. Subjects who exhibit only mild resting tremor but no other symptoms or signs of PD may be included.
- Presence of significant cognitive impairment as determined with a score ≤ 24 on the Mini Mental Status Examination (MMSE)
- History of immunocompromise, including patient who is HIV positive
- Known life-threatening systemic disease
- Patients with a history of seizures within the past year
- Patients with current or a prior history of any psychiatric illness will be excluded. Any presence or history of psychosis will be excluded. Patients with mood disorders including depression will be excluded. For the purpose of this study, we consider a significant mood disorder to include any patient who has:
- been under the care of a psychiatrist for over 3 months
- taken antidepressant medications for greater than 6 months
- has participated in cognitive-behavioral therapy
- been hospitalized for the treatment of a psychiatric illness
- received transcranial magnetic stimulation
- received electroconvulsive therapy
- Patients with risk factors for intraoperative or postoperative bleeding (platelet count less than 100,000 per cubic millimeter, PT > 14, PTT > 36 or INR > 1.3) or a documented coagulopathy
- Patients with brain tumors
- Any illness that in the investigator's opinion preclude participation in this study.
- Pregnancy or lactation.
- Legal incapacity or limited legal capacity.
- Patients who have had deep brain stimulation or a prior stereotactic ablation of the basal ganglia
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Feasibility Study to Evaluate Safety and Initial Effectiveness of ExAblate Transcranial MR Guided Focused Ultrasound for Unilateral Thalamotomy in the Treatment of Essential Tremor
A feasibility Study to Evaluate Safety and Initial Effectiveness of ExAblate Transcranial MRI-guided focused ultrasound (MRgFUS) treatment of patients with medication-refractory movement disorders, namely Essential Tremor (ET).
This study is designed as a prospective, single site, single arm, nonrandomized study. Assessments will be made before and three months after MRgFUS for clinical symptom relief, quality of life (QoL) improvements, and safety of MRgFUS in the treatment of ET. Similarly, QoL measures will be obtained using Quality of Life in Essential Tremor (QUEST) questionnaire. Relative Safety will be evaluated using a common description of Significant Clinical Complications for patients treated in this study. This study will be performed on the 3T MR scanners.
Read more
The ExAblate system is a medical device that involves a focused ultrasound system and an MRI scanner. ExAblate delivers a pulse of focused ultrasound energy, or sonication, to the targeted tissue. In this particular study the targeted tissue is a unilateral thermal lesion created in the ventralis intermedius nucleus of the thalamus.
The treatment begins with a series of standard diagnostic MR images to identify the location and shape of tumor to be treated. The ExAblate computer uses the physician's designation of the target volume to plan the best way to cover the target volume with small spots called "sonications". These treatment spots are cylinder shaped. Their size depends on sonication power and duration. During the treatment, a specific MR scan, which can be processed to identify changes in tissue temperature, provides a thermal map of the treatment volume to confirm the therapeutic effect. The thermal map is used to monitor the treatment in progress, and confirm that the ablation is proceeding according to plan, thus closing the therapy loop.
The ExAblate transcranial operates a helmet-shaped transducer (currently utilizing 1000-element phased array transducer) positioned above the subject head. The ExAblate transcranial system also includes means to immobilize the subject head, cool the interface water, and software for CT analysis and phase correction computation.
The ExAblate transcranial system is an experimental device and is being investigated in this study.
- Conditions
- Essential Tremor
- Being tested
- ExAblate Transcranial MRgFUS System
- Medical subject headings
- Essential Tremor
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ExAblate Transcranial MRgFUS SystemDevice
| Study group | Role | What they receive |
|---|---|---|
| ExAblate Treatment | Experimental | Not described Receives: Device: ExAblate Transcranial MRgFUS System |
What is being measured
The primary outcome is the main question the study is designed to answer.
Device or procedure related Adverse Events reported
Safety of the ExAblate transcranial treatment will be determined by an evaluation of the incidence and severity of device and procedure related complications from the first / treatment day visit through the 3-Months post-treatment time point. All AEs will be reported and categorized by investigators as definitely, probably, possibly, or unrelated to the device, procedure, or post-surgical changes in neurological status. Alternative treatments resulting from post-surgical changes in neurological status will be reported.
Measured: 3 Months
Secondary outcomes (1)
Tremor rating scales: the Clinical Rating Scale for Tremors
Primary effectiveness will be evaluated using validated, tremor rating scales: the Clinical Rating Scale for Tremors (CRST) for ET patients, based upon patients in whom unilateral ExAblate lesioning is attempted (i.e., Intent-to-Treat analysis). Efficacy is defined as a reduction in contralateral symptoms at 3-months post-treatment.
Measured: 3 Month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2014 (estimated)
- Study startFeb 2011
- Primary completionJan 2014
- Study completionJan 2014
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 InSightecThe 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- InSightec
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jin Woo Chang, Prof., Principal investigator, Yonsei University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02037217
- Sponsor's protocol ID
- ET001K
- Status last verified
- Mar 2016
- First submitted
- 14 Jan 2014
- Last updated
- 8 Mar 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Mar 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Feasibility Study to Evaluate Safety and Initial Effectiveness of ExAblate Transcranial MR Guided Focused Ultrasound for Unilateral Thalamotomy in the Treatment of Essential Tremor
- ClinicalTrials.gov ID
- NCT02037217
- Acronym / sponsor ID
- ET001K
- Conditions
- Essential Tremor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- InSightecIndustry (pharma, biotech, devices)
- Study start
- Feb 2011
- Primary completion
- Jan 2014 (actual)
- Study completion
- Jan 2014 (actual)
- First posted
- 15 Jan 2014 (estimated)
- 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.
A feasibility Study to Evaluate Safety and Initial Effectiveness of ExAblate Transcranial MRI-guided focused ultrasound (MRgFUS) treatment of patients with medication-refractory movement disorders, namely Essential Tremor (ET).
This study is designed as a prospective, single site, single arm, nonrandomized study. Assessments will be made before and three months after MRgFUS for clinical symptom relief, quality of life (QoL) improvements, and safety of MRgFUS in the treatment of ET. Similarly, QoL measures will be obtained using Quality of Life in Essential Tremor (QUEST) questionnaire. Relative Safety will be evaluated using a common description of Significant Clinical Complications for patients treated in this study. This study will be performed on the 3T MR scanners.
The ExAblate system is a medical device that involves a focused ultrasound system and an MRI scanner. ExAblate delivers a pulse of focused ultrasound energy, or sonication, to the targeted tissue. In this particular study the targeted tissue is a unilateral thermal lesion created in the ventralis intermedius nucleus of the thalamus.
The treatment begins with a series of standard diagnostic MR images to identify the location and shape of tumor to be treated. The ExAblate computer uses the physician's designation of the target volume to plan the best way to cover the target volume with small spots called "sonications". These treatment spots are cylinder shaped. Their size depends on sonication power and duration. During the treatment, a specific MR scan, which can be processed to identify changes in tissue temperature, provides a thermal map of the treatment volume to confirm the therapeutic effect. The thermal map is used to monitor the treatment in progress, and confirm that the ablation is proceeding according to plan, thus closing the therapy loop.
The ExAblate transcranial operates a helmet-shaped transducer (currently utilizing 1000-element phased array transducer) positioned above the subject head. The ExAblate transcranial system also includes means to immobilize the subject head, cool the interface water, and software for CT analysis and phase correction computation.
The ExAblate transcranial system is an experimental device and is being investigated in this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ExAblate Treatment | Experimental | Device: ExAblate Transcranial MRgFUS System |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Men and women, between 18 and 80 years, inclusive
- Patients who are able and willing to give consent and able to attend all study visits
- A diagnosis of ET as confirmed from clinical history and examination by a movement disorder neurologist
- Tremor refractory to adequate trials of at least two medications, one of which should be either propranolol or primidone. An adequate medication trial is defined as a therapeutic dose of each medication or the development of side effects as the medication dose is titrated.
- Vim nucleus of thalamus can be target by the ExAblate device. The Vim region of the thalamus must be apparent on MRI such that targeting can be performed with either direct visualization or by measurement from a line connecting the anterior and posterior commissures of the brain.
Key exclusion criteria (39 listed)
- Patients with unstable cardiac status including:
- 1.1 Unstable angina pectoris on medication 1.2 Patients with documented myocardial infarction within six months of protocol entry 1.3 Congestive heart failure requiring medication (other than diuretic) 1.4 Patients on anti-arrhythmic drugs
- Patients exhibiting any behavior(s) consistent with ethanol or substance abuse as defined by the criteria outlined in the DSM-IV as manifested by one (or more) of the following occurring within a 12 month period:
- Severe hypertension (diastolic BP > 100 on medication)
- Patients with standard contraindications for MR imaging such as non-MRI compatible implanted metallic devices including cardiac pacemakers, size limitations, etc.
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 |
|---|---|---|
| ExAblate Transcranial MRgFUS System | Device | ExAblate Treatment |
Primary outcome
Device or procedure related Adverse Events reported
Time frame: 3 Months
Safety of the ExAblate transcranial treatment will be determined by an evaluation of the incidence and severity of device and procedure related complications from the first / treatment day visit through the 3-Months post-treatment time point. All AEs will be reported and categorized by investigators as definitely, probably, possibly, or unrelated to the device, procedure, or post-surgical changes in neurological status. Alternative treatments resulting from post-surgical changes in neurological status will be reported.
1 secondary outcome
Tremor rating scales: the Clinical Rating Scale for Tremors
Time frame: 3 Month
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Mar 2016.
- Sponsor websiteLink provided by the sponsor.
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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.
Jin Woo Chang, Prof. principal investigator
Yonsei University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ExAblate Transcranial MRgFUS System in people with Essential Tremor, run by InSightec at 1 site in South Korea.
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 11 inclusion and 39 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: finished, as of the sponsor's last update on 8 Mar 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. 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 phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Device or procedure related Adverse Events reported (time frame: 3 Months). 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 8 Mar 2016.