|
General |
Study Status |
Delayed |
Application Number / Requirement Number |
P150038 S006/ PAS001 |
Date Original Protocol Accepted |
11/06/2020
|
Date Current Protocol Accepted |
11/11/2022
|
Study Name |
PAS Registry Study for ExAblate 4000
|
Device Name |
ExAblate
|
General Study Protocol Parameters |
Study Design |
Comprehensive/Linked/RegistryBased Surveillance
|
Data Source |
Sponsor Registry
|
Comparison Group |
No Control
|
Analysis Type |
Descriptive
|
Study Population |
Adult: >21
|
Detailed Study Protocol Parameters |
Study Objectives |
Prospective, multicenter, international single arm, observational post-approval registry study
|
Study Population |
Single-arm study of male and female patients at least 30 years old who plan to be treated for TDPD using the Exablate Neuro System and who meet the specific enrollment criteria
|
Sample Size |
50 patients are planned for this registry study. From the protocol: A total of 50 subjects will be enrolled. A t-test graph demonstrated that the power will be sufficient to determine any significant differences in long-term effectiveness at a=0.05. The graph showed that at 25 subjects, the number of subjects needed had plateaued. With an approximate average of an 8% dropout per year through Year 5, the remaining population is estimated to be approximately 30 subjects at year 5, which shows full power.
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Key Study Endpoints |
From the protocol: Safety Analysis Safety will be evaluated through descriptive analysis of the incidence and severity of adverse events. Adverse events will be recorded and categorized according to severity, expectedness, and relationship to Exablate Neuro system thalamotomy procedure and/or disease progression. Results will be tabulated and reported as follows: Post thalamotomy incidence and severity of adverse events (AEs). Comparison of post thalamotomy incidence and severity adverse event rates to the prior Exablate study for ET (PMA #P150038), and TDPD earlier study (P150038/S006). Comparison of post thalamotomy incidence and severity adverse events rates to prior Deep Brain Stimulation (DBS) rates Effectiveness Analysis Effectiveness will be evaluated through descriptive analysis of ON medication CRST scores for tremor/motor, posture/rest, and level of disability. Results will be presented as percent change from baseline to post thalamotomy study visits for all subjects where unilateral Exablate thalamotomy was attempted.
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Follow-up Visits and Length of Follow-up |
5 years
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Interim or Final Data Summary |
Interim Results |
Safety Results Table 5 below presents 13 events in seven of the 18 subjects. Eleven subjects had no adverse events (total subjects, 18). Three of the events were new this reporting period (bold highlight, insomnia and falls, which were related to Parkinson’s (insomnia and a transient PD related fall) and a Procedure Related fall per the investigator. Five events in four subjects were Mild in severity, five events were Moderate, three events in two subjects were Severe, all of which were reported in the previous semiannual reports, and no events were Life Threatening. There were no new Severe AEs in this reporting period. Seven out of 13 adverse events were either transient, resolving within three days or unrelated to the device or procedure. Three events were Thalamotomy related. The event of severe imbalance issues was reported five days post treatment and was reported in a previous semiannual report (as "Ambulatory Issues.")
Effectiveness Results Although the sample size is still small, the CRST generally improved from Screening baseline. The mean Tremor-Motor score improved from 13.1 to 2.2 at Month 3; percent change improved 85% (Table 6 and Figure 1). Tremor as measured by posture and rest components also showed improvement (Tables 7 & 8, and Figures 2 & 3). The Part C mean score improved from 10.0 to 4.0 at Month 3 (Table 9 and Figures 4)). Percent change at Month 6 was 63%.
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Actual Number of Patients Enrolled |
18
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Actual Number of Sites Enrolled |
8
|
Patient Follow-up Rate |
The expected subjects of Mo1, Mo3, Mo6 and Mo12 are 18, 15, 9 and 5, respectively. The actual who has completed the follow-up visit are 17, 12, 9, 5.
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