Trial Outcomes & Findings for ExAblate Transcranial MR Guided Focused Ultrasound for the Treatment of Parkinson's Disease (NCT NCT01772693)
NCT ID: NCT01772693
Last Updated: 2020-10-09
Results Overview
Evaluate of adverse events (AEs) associated with the ExAblate Transcranial thalamotomy of medication-refractory, tremor-dominant PD during the ExAblate treatment. Primary safety analyses are reported in the adverse events module.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
27 participants
Primary outcome timeframe
Month 3
Results posted on
2020-10-09
Participant Flow
Participant milestones
| Measure |
ExAblate Transcranial MRgFUS
ExAblate Transcranial MR guided Focused Ultrasound
ExAblate Transcranial MRgFUS: ExAblate Transcranial MR Guided Focused Ultrasound
|
Sham ExAblate Transcranial MRgFUS
Sham treatment with ExAblate MR guided Focused Ultrasound
Sham ExAblate Transcranial MRgFUS: Sham ExAblate Transcranial MR Guided Focused Ultrasound
|
|---|---|---|
|
Overall Study
STARTED
|
20
|
7
|
|
Overall Study
COMPLETED
|
14
|
6
|
|
Overall Study
NOT COMPLETED
|
6
|
1
|
Reasons for withdrawal
| Measure |
ExAblate Transcranial MRgFUS
ExAblate Transcranial MR guided Focused Ultrasound
ExAblate Transcranial MRgFUS: ExAblate Transcranial MR Guided Focused Ultrasound
|
Sham ExAblate Transcranial MRgFUS
Sham treatment with ExAblate MR guided Focused Ultrasound
Sham ExAblate Transcranial MRgFUS: Sham ExAblate Transcranial MR Guided Focused Ultrasound
|
|---|---|---|
|
Overall Study
Health Concerns Unrelated to Study
|
2
|
1
|
|
Overall Study
Withdrawal by Subject
|
2
|
0
|
|
Overall Study
Alternative Treatment
|
2
|
0
|
Baseline Characteristics
ExAblate Transcranial MR Guided Focused Ultrasound for the Treatment of Parkinson's Disease
Baseline characteristics by cohort
| Measure |
ExAblate Transcranial MRgFUS
n=20 Participants
ExAblate Transcranial MR guided Focused Ultrasound
ExAblate Transcranial MRgFUS: ExAblate Transcranial MR Guided Focused Ultrasound
|
Sham ExAblate Transcranial MRgFUS
n=7 Participants
Sham treatment with ExAblate MR guided Focused Ultrasound
Sham ExAblate Transcranial MRgFUS: Sham ExAblate Transcranial MR Guided Focused Ultrasound
|
Total
n=27 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
67.9 years
STANDARD_DEVIATION 9.6 • n=5 Participants
|
62.9 years
STANDARD_DEVIATION 11.3 • n=7 Participants
|
66.6 years
STANDARD_DEVIATION 10.1 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
19 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Caucasian
|
18 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
24 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Black
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Other
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
20 participants
n=5 Participants
|
7 participants
n=7 Participants
|
27 participants
n=5 Participants
|
|
Clinical Rating Scale for Tremor - Treated Side
|
19.0 units on a scale
STANDARD_DEVIATION 8.7 • n=5 Participants
|
20.1 units on a scale
STANDARD_DEVIATION 7.4 • n=7 Participants
|
19.3 units on a scale
STANDARD_DEVIATION 8.3 • n=5 Participants
|
|
Mean Unified Parkinson's Disease Rating Scale (UPDRS) #20
|
3.7 units on a scale
STANDARD_DEVIATION 1.0 • n=5 Participants
|
3.4 units on a scale
STANDARD_DEVIATION 1.5 • n=7 Participants
|
3.6 units on a scale
STANDARD_DEVIATION 1.1 • n=5 Participants
|
|
Mean Unified Parkinson's Disease Rating Scale (UPDRS) #21
|
3.2 units on a scale
STANDARD_DEVIATION 1.3 • n=5 Participants
|
3.6 units on a scale
STANDARD_DEVIATION 1.1 • n=7 Participants
|
3.3 units on a scale
STANDARD_DEVIATION 1.2 • n=5 Participants
|
PRIMARY outcome
Timeframe: Month 3Population: All Adverse Events are reported in the Adverse Events module.
Evaluate of adverse events (AEs) associated with the ExAblate Transcranial thalamotomy of medication-refractory, tremor-dominant PD during the ExAblate treatment. Primary safety analyses are reported in the adverse events module.
Outcome measures
| Measure |
ExAblate Transcranial MRgFUS
n=20 Participants
ExAblate Transcranial MR guided Focused Ultrasound
ExAblate Transcranial MRgFUS: ExAblate Transcranial MR Guided Focused Ultrasound
|
Sham ExAblate Transcranial MRgFUS
n=7 Participants
Sham treatment with ExAblate MR guided Focused Ultrasound
Sham ExAblate Transcranial MRgFUS: Sham ExAblate Transcranial MR Guided Focused Ultrasound
|
|---|---|---|
|
Number of Adverse Events
|
100 Number of Adverse Events
|
10 Number of Adverse Events
|
SECONDARY outcome
Timeframe: Baseline, Month 3The percent change from baseline to Month 3 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 3 Months were used to calculate percent change from baseline and averaged across subjects. High percent change from baseline is better (shows improvement).
Outcome measures
| Measure |
ExAblate Transcranial MRgFUS
n=20 Participants
ExAblate Transcranial MR guided Focused Ultrasound
ExAblate Transcranial MRgFUS: ExAblate Transcranial MR Guided Focused Ultrasound
|
Sham ExAblate Transcranial MRgFUS
n=7 Participants
Sham treatment with ExAblate MR guided Focused Ultrasound
Sham ExAblate Transcranial MRgFUS: Sham ExAblate Transcranial MR Guided Focused Ultrasound
|
|---|---|---|
|
Tremor Motor Score Percent Change From Baseline.
|
51.9 percent change from baseline
Standard Deviation 33.4
|
12.7 percent change from baseline
Standard Deviation 25.7
|
SECONDARY outcome
Timeframe: Baseline, Month 3, Month 12Upper extremity Tremor-Motor score for the treated side is a sub-scale of Clinical Rating Scale for Tremor (CRST) Part A and Part B sum that measures treated-side upper extremity tremor changes over time. Tremor-motor scores range from 0-32 points for each side with lower scores being a better outcome. Subject's Tremor-Motor scores for the test and sham control groups were averaged at each study visit.
Outcome measures
| Measure |
ExAblate Transcranial MRgFUS
n=20 Participants
ExAblate Transcranial MR guided Focused Ultrasound
ExAblate Transcranial MRgFUS: ExAblate Transcranial MR Guided Focused Ultrasound
|
Sham ExAblate Transcranial MRgFUS
n=7 Participants
Sham treatment with ExAblate MR guided Focused Ultrasound
Sham ExAblate Transcranial MRgFUS: Sham ExAblate Transcranial MR Guided Focused Ultrasound
|
|---|---|---|
|
Tremor Motor Scores - Clinical Rating Scale for Tremor - Treated Side Upper Extremity Parts A & B.
Baseline
|
19.0 score on a scale
Standard Deviation 8.7
|
20.1 score on a scale
Standard Deviation 7.4
|
|
Tremor Motor Scores - Clinical Rating Scale for Tremor - Treated Side Upper Extremity Parts A & B.
Month 3
|
9.6 score on a scale
Standard Deviation 9.7
|
17.4 score on a scale
Standard Deviation 7.8
|
|
Tremor Motor Scores - Clinical Rating Scale for Tremor - Treated Side Upper Extremity Parts A & B.
Month 12
|
10.5 score on a scale
Standard Deviation 10.2
|
NA score on a scale
Standard Deviation NA
The sham ExAblate subjects exited the study at Month 3. The Exablate test subjects continued through Month 12.
|
SECONDARY outcome
Timeframe: Baseline, Month 3, Month 12Population: The Exablate sham subjects exited the study at the Month 3 visit. The Exablate test subjects continued on through Month 12.
The Clinical Rating Scale for Tremor (CRST) Part C is a measure of functional disability due to tremor. The Clinical Rating Scale for Tremor Part C consists of 8 items each scored from 0 to 4. Thus, the total score summed ranges from 0 to 32 and provides an overall assessment of activities of daily living. Low scores on the Clinical Rating Scale for Tremor (CRST) Part C are better. Low scores show improvement in functional disabilities compared to higher scores.
Outcome measures
| Measure |
ExAblate Transcranial MRgFUS
n=20 Participants
ExAblate Transcranial MR guided Focused Ultrasound
ExAblate Transcranial MRgFUS: ExAblate Transcranial MR Guided Focused Ultrasound
|
Sham ExAblate Transcranial MRgFUS
n=7 Participants
Sham treatment with ExAblate MR guided Focused Ultrasound
Sham ExAblate Transcranial MRgFUS: Sham ExAblate Transcranial MR Guided Focused Ultrasound
|
|---|---|---|
|
Functional Disabilities - Clinical Rating Scale for Tremor Part C - Functional Disabilities
Baseline
|
14.1 score on a scale
Standard Deviation 5.7
|
16.7 score on a scale
Standard Deviation 3.9
|
|
Functional Disabilities - Clinical Rating Scale for Tremor Part C - Functional Disabilities
Month 3
|
6.4 score on a scale
Standard Deviation 6.6
|
14.6 score on a scale
Standard Deviation 4.2
|
|
Functional Disabilities - Clinical Rating Scale for Tremor Part C - Functional Disabilities
Month 12
|
6.9 score on a scale
Standard Deviation 7.2
|
NA score on a scale
Standard Deviation NA
The Exablate sham subjects exited the study at the Month 3 study visit.
|
Adverse Events
ExAblate Transcranial MRgFUS
Serious events: 3 serious events
Other events: 20 other events
Deaths: 0 deaths
Sham ExAblate Transcranial MRgFUS
Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
ExAblate Transcranial MRgFUS
n=20 participants at risk
ExAblate Transcranial MR guided Focused Ultrasound
ExAblate Transcranial MRgFUS: ExAblate Transcranial MR Guided Focused Ultrasound
|
Sham ExAblate Transcranial MRgFUS
n=7 participants at risk
Sham treatment with ExAblate MR guided Focused Ultrasound
Sham ExAblate Transcranial MRgFUS: Sham ExAblate Transcranial MR Guided Focused Ultrasound
|
|---|---|---|
|
Nervous system disorders
Hemiparesis
|
10.0%
2/20 • Number of events 2 • 1 year
|
0.00%
0/7 • 1 year
|
|
Nervous system disorders
Ataxia
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Gastrointestinal disorders
Cholecystitis
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Degenerative knee disease
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Psychiatric disorders
Worsening depression
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Vascular disorders
Transient ischemic attack
|
0.00%
0/20 • 1 year
|
14.3%
1/7 • Number of events 1 • 1 year
|
Other adverse events
| Measure |
ExAblate Transcranial MRgFUS
n=20 participants at risk
ExAblate Transcranial MR guided Focused Ultrasound
ExAblate Transcranial MRgFUS: ExAblate Transcranial MR Guided Focused Ultrasound
|
Sham ExAblate Transcranial MRgFUS
n=7 participants at risk
Sham treatment with ExAblate MR guided Focused Ultrasound
Sham ExAblate Transcranial MRgFUS: Sham ExAblate Transcranial MR Guided Focused Ultrasound
|
|---|---|---|
|
General disorders
Sleep Disorder
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Nervous system disorders
Dysgnosia
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Nervous system disorders
Tremor worsened
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
General disorders
Fatique
|
10.0%
2/20 • Number of events 2 • 1 year
|
0.00%
0/7 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal weakness
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Ear and labyrinth disorders
Dizziness
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Dysmetria
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Gait disturbance
|
10.0%
2/20 • Number of events 2 • 1 year
|
0.00%
0/7 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Hemiparesis
|
10.0%
2/20 • Number of events 2 • 1 year
|
0.00%
0/7 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Imbalance
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Nervous system disorders
Dysmetria
|
10.0%
2/20 • Number of events 2 • 1 year
|
0.00%
0/7 • 1 year
|
|
Nervous system disorders
Ataxia
|
10.0%
2/20 • Number of events 2 • 1 year
|
0.00%
0/7 • 1 year
|
|
Nervous system disorders
Numbness/tingling
|
25.0%
5/20 • Number of events 7 • 1 year
|
0.00%
0/7 • 1 year
|
|
Nervous system disorders
Unsteady
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Vascular disorders
Hypertension
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Vascular disorders
Syncope
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Skin and subcutaneous tissue disorders
Sonication related flushing
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Eye disorders
Visual field defect
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Gastrointestinal disorders
Nausea/Vomiting
|
25.0%
5/20 • Number of events 5 • 1 year
|
14.3%
1/7 • Number of events 1 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Positional pain
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Nervous system disorders
Imbalance
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Psychiatric disorders
Anxiety
|
10.0%
2/20 • Number of events 2 • 1 year
|
0.00%
0/7 • 1 year
|
|
Psychiatric disorders
Dysgnosia
|
10.0%
2/20 • Number of events 2 • 1 year
|
0.00%
0/7 • 1 year
|
|
General disorders
Headache
|
55.0%
11/20 • Number of events 11 • 1 year
|
28.6%
2/7 • Number of events 2 • 1 year
|
|
Skin and subcutaneous tissue disorders
Sonication related scalp pain
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
General disorders
Sonication related head pain
|
25.0%
5/20 • Number of events 6 • 1 year
|
14.3%
1/7 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Pin Site Pain
|
0.00%
0/20 • 1 year
|
14.3%
1/7 • Number of events 1 • 1 year
|
|
Eye disorders
Pigment change in eye
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Eye disorders
Vision change
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Gastrointestinal disorders
Cholecystitis
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Gastrointestinal disorders
Stomach pain
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
General disorders
Vocal change
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Psychiatric disorders
Worsening depression
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
5.0%
1/20 • Number of events 2 • 1 year
|
0.00%
0/7 • 1 year
|
|
Vascular disorders
Cerebelar infarct
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Vascular disorders
Transient Ischemic Attack
|
0.00%
0/20 • 1 year
|
14.3%
1/7 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Facial edema
|
15.0%
3/20 • Number of events 3 • 1 year
|
0.00%
0/7 • 1 year
|
|
Skin and subcutaneous tissue disorders
Pin site numbness/tingling
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
|
Skin and subcutaneous tissue disorders
Pin site pain
|
15.0%
3/20 • Number of events 3 • 1 year
|
14.3%
1/7 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Stereotactic frame bruising
|
5.0%
1/20 • Number of events 1 • 1 year
|
0.00%
0/7 • 1 year
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place