Trial Outcomes & Findings for ExAblate (MRgFUS) Treatment of Metastatic Bone Tumors for the Palliation of Pain (NCT NCT00656305)

Last Updated: 2020-02-05

Results Overview

Using the Numerical Rating Score (NRS) for pain (0 being no pain and 10 being worst imaginable pain), each subject was rated as a Responder or Non-responder. A responder is defined as a subject with a reduction in NRS worst score from baseline of two (2) or more points, and no increase in pain medication use.

Recruitment status

COMPLETED

Study phase

Target enrollment

147 participants

Primary outcome timeframe

3 months post treatment

Results posted on

2020-02-05

Participant Flow

Of the 147 subjects enrolled (signed consent), 23 subjects failed additional study requirements and 9 subjects exited after randomization but prior to treatment

Participant milestones

Participant milestones
Measure	ExAblate Treatment Arm ExAblate 2000: MR guided focused ultrasound	ExAblate Sham Control Arm ExAblate 2000: MR guided focused ultrasound sham
Overall Study STARTED	87	28
Overall Study COMPLETED	65	21
Overall Study NOT COMPLETED	22	7

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

ExAblate (MRgFUS) Treatment of Metastatic Bone Tumors for the Palliation of Pain

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	ExAblate Treatment Arm n=87 Participants ExAblate 2000: MR guided focused ultrasound	ExAblate Sham Control Arm n=28 Participants ExAblate 2000: MR guided focused ultrasound sham	Total n=115 Participants Total of all reporting groups
Age, Continuous	58.7 years STANDARD_DEVIATION 13.3 • n=5 Participants	56.8 years STANDARD_DEVIATION 10.5 • n=7 Participants	57.8 years STANDARD_DEVIATION 11.9 • n=5 Participants
Sex: Female, Male Female	49 Participants n=5 Participants	22 Participants n=7 Participants	71 Participants n=5 Participants
Sex: Female, Male Male	38 Participants n=5 Participants	6 Participants n=7 Participants	44 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	81 Participants n=5 Participants	26 Participants n=7 Participants	107 Participants n=5 Participants
Race (NIH/OMB) More than one race	4 Participants n=5 Participants	0 Participants n=7 Participants	4 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment Canada	6 participants n=5 Participants	1 participants n=7 Participants	7 participants n=5 Participants
Region of Enrollment United States	30 participants n=5 Participants	11 participants n=7 Participants	41 participants n=5 Participants
Region of Enrollment Italy	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Region of Enrollment Israel	17 participants n=5 Participants	5 participants n=7 Participants	22 participants n=5 Participants
Region of Enrollment Russia	34 participants n=5 Participants	10 participants n=7 Participants	44 participants n=5 Participants

PRIMARY outcome

Timeframe: 3 months post treatment

Population: 115 subjects were underwent treatment. Of these, 4 subjects terminated treatment prior to full completion. These 4 subjects are excluded from the efficacy analysis. An additional 4 subjects were found to have been enrolled in the study more than once. In a pre-PMA meeting with FDA, it was agreed upon to exclude these 4 subjects from the analysis.

Outcome measures

Outcome measures
Measure	ExAblate Treatment Arm n=81 Participants ExAblate 2000: MR guided focused ultrasound	ExAblate Sham Control Arm n=26 Participants ExAblate 2000: MR guided focused ultrasound sham
Number of Responders	54 Participants	6 Participants

SECONDARY outcome

Timeframe: 3 months post treatment

Population: Only 26 of the 81 subjects in ExAblate treatment arm and 6 of 26 in the sham arm were taking non-opioid medication at baseline.

Medication Use change reported from baseline until of study. Medication use is quantified by "morphine equivalent usage" (measured separately from Responder/Non-responder definition for the primary endpoint)

Outcome measures

Outcome measures
Measure	ExAblate Treatment Arm n=26 Participants ExAblate 2000: MR guided focused ultrasound	ExAblate Sham Control Arm n=6 Participants ExAblate 2000: MR guided focused ultrasound sham
Number of Participants With a Change in Medication Use	15 Participants	2 Participants

SECONDARY outcome

Timeframe: 3 months post treatment

The BPI-QOL questionnaire is designed to show the severity and interference of pain in the lives of patients. This is a 7-item questionnaire that asks respondents the extent to which pain interferes with their general activity, mood, walking ability, normal work, relations with other people, sleep and enjoyment of life using a 0-10 numerical rating scale in which 0 represents 'does not interfere' and 10 indicates 'completely interferes'. Responses to the 7 items are averaged to form the pain interference scale score. Lower scores are better, showing less interference in daily activities while higher scores show more interference and hence worse outcomes.

Outcome measures

Outcome measures
Measure	ExAblate Treatment Arm n=81 Participants ExAblate 2000: MR guided focused ultrasound	ExAblate Sham Control Arm n=26 Participants ExAblate 2000: MR guided focused ultrasound sham
Quality of Life (QOL) as Measured by Change in Bodily Pain Inventory (BPI) From Baseline	2.5 score on a scale Standard Deviation 2.8	.3 score on a scale Standard Deviation 2.3

Adverse Events

ExAblate Treatment Arm

Serious events: 8 serious events

Other events: 49 other events

Deaths: 8 deaths

ExAblate Sham Control Arm

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	ExAblate Treatment Arm n=87 participants at risk ExAblate 2000: MR guided focused ultrasound	ExAblate Sham Control Arm n=28 participants at risk ExAblate 2000: MR guided focused ultrasound sham
Immune system disorders Death	9.2% 8/87	0.00% 0/28
Surgical and medical procedures Hospitalization	1.1% 1/87	0.00% 0/28

Other adverse events

Other adverse events
Measure	ExAblate Treatment Arm n=87 participants at risk ExAblate 2000: MR guided focused ultrasound	ExAblate Sham Control Arm n=28 participants at risk ExAblate 2000: MR guided focused ultrasound sham
Cardiac disorders Deep Vein Thrombosis	1.1% 1/87 • Number of events 1	0.00% 0/28
Injury, poisoning and procedural complications Skin burn	2.3% 2/87 • Number of events 2	0.00% 0/28
Nervous system disorders Numbness	1.1% 1/87 • Number of events 1	0.00% 0/28
Nervous system disorders Neuropathy/leg pain	2.3% 2/87 • Number of events 2	0.00% 0/28
Injury, poisoning and procedural complications Pain/discomfort	52.9% 46/87 • Number of events 48	3.6% 1/28 • Number of events 1
Respiratory, thoracic and mediastinal disorders Apnea	1.1% 1/87 • Number of events 1	0.00% 0/28
Surgical and medical procedures Fracture	1.1% 1/87 • Number of events 1	0.00% 0/28
General disorders Fatigue	2.3% 2/87 • Number of events 2	0.00% 0/28
Infections and infestations Fever	1.1% 1/87 • Number of events 1	0.00% 0/28

Additional Information

Nadir Alikacem

InSightec

Phone: 214-630-2000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place