Trial Outcomes & Findings for ExAblate Transcranial MRgFUS of the Globus Pallidum for Treatment of Parkinson's Disease (NCT NCT02263885)

NCT ID: NCT02263885

Last Updated: 2022-01-19

Results Overview

The primary outcome of this feasibility trial was safety. Adverse Events were categorized by physicians as mild, moderate, or severe. Events resulting in impairment, requiring intervention to preclude impairment, or that were life threatening, and deaths were reported by physicians as Serious Adverse Events. Outcomes are entered in the adverse events module.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 20 participants
• Primary outcome timeframe: Month 24
• Results posted on: 2022-01-19

Participant Flow

Participant milestones

Measure	ExAblate Transcranial System Test Arm Transcranial ExAblate MRgFUS Transcranial MRgFUS lesioning of the Globus Pallidum
Overall Study STARTED	20
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	10

Reasons for withdrawal

Measure	ExAblate Transcranial System Test Arm Transcranial ExAblate MRgFUS Transcranial MRgFUS lesioning of the Globus Pallidum
Overall Study Withdrawal by Subject	7
Overall Study Alternative Treatment	3

Baseline Characteristics

ExAblate Transcranial MRgFUS of the Globus Pallidum for Treatment of Parkinson's Disease

Baseline characteristics by cohort

Measure	ExAblate Transcranial System Test Arm n=20 Participants Transcranial ExAblate MRgFUS Transcranial MRgFUS lesioning of the Globus Pallidum
Age, Continuous	56.4 years STANDARD_DEVIATION 11.3 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Race/Ethnicity, Customized Race · White	18 Participants n=5 Participants
Race/Ethnicity, Customized Race · Black	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · Hispanic	2 Participants n=5 Participants
Race/Ethnicity, Customized Race · Other	0 Participants n=5 Participants
Region of Enrollment United States	20 participants n=5 Participants

PRIMARY outcome

Timeframe: Month 24

The primary outcome of this feasibility trial was safety. Adverse Events were categorized by physicians as mild, moderate, or severe. Events resulting in impairment, requiring intervention to preclude impairment, or that were life threatening, and deaths were reported by physicians as Serious Adverse Events. Outcomes are entered in the adverse events module.

Outcome measures

Measure	ExAblate Transcranial System Test Arm n=20 Participants Transcranial ExAblate MRgFUS Transcranial MRgFUS lesioning of the Globus Pallidum
Adverse Events	36 Number of Adverse Events

SECONDARY outcome

Timeframe: Screening, Month 3, Month 6, Month 12, Month 24

The Unified Dyskinesia Rating Scale Total score assesses overall involuntary movements associated with the treatment of Parkinson's disease. The minimum score possible is 0. The maximum total score possible is 104. High scores show worse symptoms.

Outcome measures

Measure	ExAblate Transcranial System Test Arm n=20 Participants Transcranial ExAblate MRgFUS Transcranial MRgFUS lesioning of the Globus Pallidum
Unified Dyskinesia Rating Scale Total Score. Screening	36.1 score on a scale Interval 31.2 to 40.9
Unified Dyskinesia Rating Scale Total Score. Month 3	14.9 score on a scale Interval 9.3 to 20.4
Unified Dyskinesia Rating Scale Total Score. Month 6	16.9 score on a scale Interval 10.1 to 23.7
Unified Dyskinesia Rating Scale Total Score. Month 12	19.5 score on a scale Interval 11.9 to 27.1
Unified Dyskinesia Rating Scale Total Score. Month 24	23.1 score on a scale Interval 12.4 to 33.8

SECONDARY outcome

Timeframe: Screening, Month 3, Month 6, Month 12, Month 24

The Unified Dyskinesia Ratient Scale (UDysRS) was developed to assess involuntary movements often associated with the treatment of Parkinson's disease. Part III evaluates the intensity of impairment of dyskinesia with respect to the performance of 7 tasks on a scale of 0 to 4. The sum of the Part III score ranges 0 to 28 and high scores show worse symptoms.

Outcome measures

Measure	ExAblate Transcranial System Test Arm n=20 Participants Transcranial ExAblate MRgFUS Transcranial MRgFUS lesioning of the Globus Pallidum
Unified Dyskinesia Rating Scale (UDysRS), Part III Screening	10.2 score on a scale Interval 7.5 to 12.9
Unified Dyskinesia Rating Scale (UDysRS), Part III Month 3	4.5 score on a scale Interval 2.4 to 6.6
Unified Dyskinesia Rating Scale (UDysRS), Part III Month 6	6.0 score on a scale Interval 2.7 to 9.4
Unified Dyskinesia Rating Scale (UDysRS), Part III Month 12	4.1 score on a scale Interval 1.7 to 6.7
Unified Dyskinesia Rating Scale (UDysRS), Part III Month 24	5.8 score on a scale Interval 2.3 to 9.2

SECONDARY outcome

Timeframe: Screening, Month 3, Month 6, Month 12, Month 24

The Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS UPDRSP Part III motor exam focuses specifically on the treated side tremor and motor fluctuations. Each of 11 items are scored from 0 to 4 for a total score minimum of 0 and a maximum of 44 points. High scores show worse symptoms.

Outcome measures

Measure	ExAblate Transcranial System Test Arm n=20 Participants Transcranial ExAblate MRgFUS Transcranial MRgFUS lesioning of the Globus Pallidum
Movement Disorders Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS), Part III Motor Exam Screening	20.0 score on a scale Interval 17.5 to 22.4
Movement Disorders Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS), Part III Motor Exam Month 3	10.6 score on a scale Interval 8.7 to 12.5
Movement Disorders Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS), Part III Motor Exam Month 6	11.1 score on a scale Interval 9.6 to 12.6
Movement Disorders Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS), Part III Motor Exam Month 12	10.5 score on a scale Interval 8.2 to 12.7
Movement Disorders Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS), Part III Motor Exam Month 24	12.3 score on a scale Interval 9.8 to 14.9

SECONDARY outcome

Timeframe: Screening, Month 3, Month 6, Month 12, Month 24

.Part II of the Mood Disorders Society Unified Parkinson's Disease Rating Scale focuses on the impact of symptoms on motor aspects of daily living. Part II consists of 13 items scored from 0 to 4 for a maximum score of 52. High Part II scores indicate worse (greater) impact.

Outcome measures

Measure	ExAblate Transcranial System Test Arm n=20 Participants Transcranial ExAblate MRgFUS Transcranial MRgFUS lesioning of the Globus Pallidum
Mood Disorders Society - Unified Parkinson's Disease Rating Scale Part II Screening	14.0 score on a scale Interval 10.7 to 17.3
Mood Disorders Society - Unified Parkinson's Disease Rating Scale Part II Month 3	7.4 score on a scale Interval 5.14 to 9.6
Mood Disorders Society - Unified Parkinson's Disease Rating Scale Part II Month 6	8.7 score on a scale Interval 5.1 to 10.8
Mood Disorders Society - Unified Parkinson's Disease Rating Scale Part II Month 12	11.4 score on a scale Interval 8.6 to 14.2
Mood Disorders Society - Unified Parkinson's Disease Rating Scale Part II Month 24	13.8 score on a scale Interval 9.4 to 18.2

SECONDARY outcome

Timeframe: Screening, Month 3, Month 6, Month 12, Month 24

Part IV of the Mood Disorder Society Unified Parkinson's Disease Rating Scale assesses dyskinesias that include OFF-state dystonia; items include time spent with dyskinesia, functional impact of dyskinesia, time spent in the OFF state, functional impact of fluctuations, complexity of motor fluctuations and painful OFF state dystonia. Six items are scored 0 to 4 with a maximum total score of 24. High scores indicate worse symptoms.

Outcome measures

Measure	ExAblate Transcranial System Test Arm n=20 Participants Transcranial ExAblate MRgFUS Transcranial MRgFUS lesioning of the Globus Pallidum
Mood Disorder Society Unified Parkinson's Disease Rating Scale Part IV Screening	11.1 score on a scale Interval 8.6 to 13.6
Mood Disorder Society Unified Parkinson's Disease Rating Scale Part IV Month 3	6.4 score on a scale Interval 4.5 to 8.2
Mood Disorder Society Unified Parkinson's Disease Rating Scale Part IV Month 6	6.0 score on a scale Interval 4.4 to 7.6
Mood Disorder Society Unified Parkinson's Disease Rating Scale Part IV Month 12	5.9 score on a scale Interval 4.0 to 7.8
Mood Disorder Society Unified Parkinson's Disease Rating Scale Part IV Month 24	8.7 score on a scale Interval 5.8 to 11.5

Adverse Events

ExAblate Transcranial System Test Arm

Serious events: 1 serious events

Other events: 15 other events

Deaths: 0 deaths

Serious adverse events

Measure	ExAblate Transcranial System Test Arm n=20 participants at risk Transcranial ExAblate MRgFUS Transcranial MRgFUS lesioning of the Globus Pallidum
Nervous system disorders Stroke	5.0% 1/20 • Number of events 1 • Adverse event data was collected for 24 months.

Other adverse events

Measure	ExAblate Transcranial System Test Arm n=20 participants at risk Transcranial ExAblate MRgFUS Transcranial MRgFUS lesioning of the Globus Pallidum
Nervous system disorders Fine Motor Problems	10.0% 2/20 • Number of events 2 • Adverse event data was collected for 24 months.
Skin and subcutaneous tissue disorders Bruise	10.0% 2/20 • Number of events 2 • Adverse event data was collected for 24 months.
Skin and subcutaneous tissue disorders Facial Edema	15.0% 3/20 • Number of events 3 • Adverse event data was collected for 24 months.
General disorders Drooling	10.0% 2/20 • Number of events 2 • Adverse event data was collected for 24 months.
Nervous system disorders Dyskinesia	10.0% 2/20 • Number of events 2 • Adverse event data was collected for 24 months.
Gastrointestinal disorders Nausea/Vomiting	15.0% 3/20 • Number of events 3 • Adverse event data was collected for 24 months.
General disorders Headache	25.0% 5/20 • Number of events 5 • Adverse event data was collected for 24 months.
General disorders Sonication Related Head Pain	35.0% 7/20 • Number of events 7 • Adverse event data was collected for 24 months.
Psychiatric disorders Anxiety	10.0% 2/20 • Number of events 2 • Adverse event data was collected for 24 months.
Nervous system disorders Dysarthria	20.0% 4/20 • Number of events 4 • Adverse event data was collected for 24 months.

Additional Information

Nadir Alikacem, PhD

InSightec

Phone: 214-630-2000

Email: nadira@insightec.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place