Trial Outcomes & Findings for Brain Stimulation for the Treatment of Tourette Syndrome (NCT NCT01329198)
NCT ID: NCT01329198
Last Updated: 2018-06-08
Results Overview
The Yale Global Tic Severity Scale (YGTSS) is a semistructured clinician-rated instrument that assesses the severity and frequency of motor and phonic tics over the previous week. Five index scores are obtained during the assessment, where higher scores indicate greater frequency or severity. These indices are: * Total Motor Tic Score (0-25) * Total Phonic Tic Score (0-25 * Total Tic Score (0-50) * Overall Impairment Rating (0-50) * Global Severity Score (0-7) The YGTSS Total Score is obtained by adding the Total Tic Score to the Overall Impairment Rating. The efficacy of the intervention will be assessed by comparing each subject's 6-month YGTSS Total Score to the pre-operative value for the same patient. Efficacy is considered 50% or greater reduction in this score.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
5 participants
Primary outcome timeframe
Baseline to 6 Months
Results posted on
2018-06-08
Participant Flow
Participant milestones
| Measure |
Delayed Activation DBS
Sham stimulation in which no electrical charge is delivered through the Neuropace RNS system. By Day 60, all subjects will be programmed to receive active stimulation.
* There will be a one month post-operative period during which stimulation is not turned on.
* One month post-implant, subjects will be randomized one to one in a blinded fashion to receive active or sham stimulation.
* By Day 60, all subjects will be programmed to receive active stimulation. Physiological data will be collected for Specific Aim 2 of the study.
* Both the sham and the active groups (will undergo identical programming procedures at 30 and 60 days.
|
Immediate Activation DBS
Active stimulation through the Neuropace RNS system at settings to maximally reduce tic frequency \& severity, while limiting potential stimulation-induced side-effects
* There will be a one month post-operative period during which stimulation is not turned on.
* One month post-implant, subjects will be randomized one to one in a blinded fashion to receive active or sham stimulation.
* By Day 60, all subjects will be programmed to receive active stimulation. Physiological data will be collected for Specific Aim 2 of the study.
* Both the sham and the active groups (3 subjects in each group) will undergo identical programming procedures at 30 and 60 days.
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
2
|
|
Overall Study
COMPLETED
|
3
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Brain Stimulation for the Treatment of Tourette Syndrome
Baseline characteristics by cohort
| Measure |
Delayed Activation DBS
n=3 Participants
Sham stimulation in which no electrical charge is delivered through the Neuropace RNS system. By Day 60, all subjects will be programmed to receive active stimulation.
* There will be a one month post-operative period during which stimulation is not turned on.
* One month post-implant, subjects will be randomized one to one in a blinded fashion to receive active or sham stimulation.
* By Day 60, all subjects will be programmed to receive active stimulation. Physiological data will be collected for Specific Aim 2 of the study.
* Both the sham and the active groups (3 subjects in each group) will undergo identical programming procedures at 30 and 60 days.
|
Immediate Activation DBS
n=2 Participants
Active stimulation through the Neuropace RNS system at settings to maximally reduce tic frequency \& severity, while limiting potential stimulation-induced side-effects
* There will be a one month post-operative period during which stimulation is not turned on.
* One month post-implant, subjects will be randomized one to one in a blinded fashion to receive active or sham stimulation.
* By Day 60, all subjects will be programmed to receive active stimulation. Physiological data will be collected for Specific Aim 2 of the study.
* Both the sham and the active groups (3 subjects in each group) will undergo identical programming procedures at 30 and 60 days.
|
Total
n=5 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
2 participants
n=7 Participants
|
5 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to 6 MonthsThe Yale Global Tic Severity Scale (YGTSS) is a semistructured clinician-rated instrument that assesses the severity and frequency of motor and phonic tics over the previous week. Five index scores are obtained during the assessment, where higher scores indicate greater frequency or severity. These indices are: * Total Motor Tic Score (0-25) * Total Phonic Tic Score (0-25 * Total Tic Score (0-50) * Overall Impairment Rating (0-50) * Global Severity Score (0-7) The YGTSS Total Score is obtained by adding the Total Tic Score to the Overall Impairment Rating. The efficacy of the intervention will be assessed by comparing each subject's 6-month YGTSS Total Score to the pre-operative value for the same patient. Efficacy is considered 50% or greater reduction in this score.
Outcome measures
| Measure |
Delayed Activation DBS
n=3 Participants
Sham stimulation in which no electrical charge is delivered through the Neuropace RNS system. By Day 60, all subjects will be programmed to receive active stimulation.
* There will be a one month post-operative period during which stimulation is not turned on.
* One month post-implant, subjects will be randomized one to one in a blinded fashion to receive active or sham stimulation.
* By Day 60, all subjects will be programmed to receive active stimulation. Physiological data will be collected for Specific Aim 2 of the study.
* Both the sham and the active groups (3 subjects in each group) will undergo identical programming procedures at 30 and 60 days.
|
Immediate Activation DBS
n=2 Participants
Active stimulation through the Neuropace RNS system at settings to maximally reduce tic frequency \& severity, while limiting potential stimulation-induced side-effects
* There will be a one month post-operative period during which stimulation is not turned on.
* One month post-implant, subjects will be randomized one to one in a blinded fashion to receive active or sham stimulation.
* By Day 60, all subjects will be programmed to receive active stimulation. Physiological data will be collected for Specific Aim 2 of the study.
* Both the sham and the active groups (3 subjects in each group) will undergo identical programming procedures at 30 and 60 days.
|
Subject 3
|
Subject 4
|
Subject 5
|
|---|---|---|---|---|---|
|
Mean Change in Yale Global Tic Severity Scale (YGTSS) Scores From Baseline to 6 Months Across All Study Participants Presented
Change in YGTSS Baseline to 6 months
|
-20.33 units on a scale
Interval -41.46 to 0.79
|
-14.05 units on a scale
Interval -127.77 to 99.67
|
—
|
—
|
—
|
|
Mean Change in Yale Global Tic Severity Scale (YGTSS) Scores From Baseline to 6 Months Across All Study Participants Presented
YGTSS Baseline Score
|
91 units on a scale
Interval 79.47 to 102.53
|
92.5 units on a scale
Interval 86.14 to 98.86
|
—
|
—
|
—
|
|
Mean Change in Yale Global Tic Severity Scale (YGTSS) Scores From Baseline to 6 Months Across All Study Participants Presented
YGTSS 6 Month Score
|
70.67 units on a scale
Interval 63.65 to 77.69
|
78.45 units on a scale
Interval 59.43 to 97.47
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline to 6 MonthsElectrical recordings of electroencephalography activity were taken from each subject's implanted leads at each visit from baseline to 6 months. At baseline, the recordings were taken with the device in the off state (not stimulating), while at the 6 month visits the recordings were taken with the subject's device set to optimal parameters for tic control. Using Pearson's correlation coefficient, the variations in frequency and power which were observed were correlated with the Yale Global Tic Severity (YGTSS) scores obtained during primary outcome testing.
Outcome measures
| Measure |
Delayed Activation DBS
n=1 Participants
Sham stimulation in which no electrical charge is delivered through the Neuropace RNS system. By Day 60, all subjects will be programmed to receive active stimulation.
* There will be a one month post-operative period during which stimulation is not turned on.
* One month post-implant, subjects will be randomized one to one in a blinded fashion to receive active or sham stimulation.
* By Day 60, all subjects will be programmed to receive active stimulation. Physiological data will be collected for Specific Aim 2 of the study.
* Both the sham and the active groups (3 subjects in each group) will undergo identical programming procedures at 30 and 60 days.
|
Immediate Activation DBS
n=1 Participants
Active stimulation through the Neuropace RNS system at settings to maximally reduce tic frequency \& severity, while limiting potential stimulation-induced side-effects
* There will be a one month post-operative period during which stimulation is not turned on.
* One month post-implant, subjects will be randomized one to one in a blinded fashion to receive active or sham stimulation.
* By Day 60, all subjects will be programmed to receive active stimulation. Physiological data will be collected for Specific Aim 2 of the study.
* Both the sham and the active groups (3 subjects in each group) will undergo identical programming procedures at 30 and 60 days.
|
Subject 3
n=1 Participants
|
Subject 4
n=1 Participants
|
Subject 5
n=1 Participants
|
|---|---|---|---|---|---|
|
Correlation of Tics and Neural Physiology
|
-0.317 Pearson Correlation Coefficient
|
-0.950 Pearson Correlation Coefficient
|
-0.704 Pearson Correlation Coefficient
|
-0.855 Pearson Correlation Coefficient
|
-0.399 Pearson Correlation Coefficient
|
Adverse Events
Sham DBS
Serious events: 1 serious events
Other events: 3 other events
Deaths: 0 deaths
Active DBS
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Sham DBS
n=3 participants at risk
Sham stimulation in which no electrical charge is delivered through the Neuropace RNS system.
NeuroPace RNS® System Deep Brain Stimulator: • There will be a one month post-operative period during which stimulation is not turned on.
* One month post-implant, subjects will be randomized one to one in a blinded fashion to receive active or sham stimulation.
* By Day 60, all subjects will be programmed to receive active stimulation. Physiological data will be collected for Specific Aim 2 of the study.
* Both the sham and the active groups (3 subjects in each group) will undergo identical programming procedures at 30 and 60 days.
|
Active DBS
n=2 participants at risk
Active stimulation through the Neuropace RNS system at settings to maximally reduce tic frequency \& severity, while limiting potential stimulation-induced side-effects
NeuroPace RNS® System Deep Brain Stimulator: • There will be a one month post-operative period during which stimulation is not turned on.
* One month post-implant, subjects will be randomized one to one in a blinded fashion to receive active or sham stimulation.
* By Day 60, all subjects will be programmed to receive active stimulation. Physiological data will be collected for Specific Aim 2 of the study.
* Both the sham and the active groups (3 subjects in each group) will undergo identical programming procedures at 30 and 60 days.
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
33.3%
1/3 • Number of events 1 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
0.00%
0/2 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
|
Injury, poisoning and procedural complications
Attempted suicide
|
0.00%
0/3 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
Other adverse events
| Measure |
Sham DBS
n=3 participants at risk
Sham stimulation in which no electrical charge is delivered through the Neuropace RNS system.
NeuroPace RNS® System Deep Brain Stimulator: • There will be a one month post-operative period during which stimulation is not turned on.
* One month post-implant, subjects will be randomized one to one in a blinded fashion to receive active or sham stimulation.
* By Day 60, all subjects will be programmed to receive active stimulation. Physiological data will be collected for Specific Aim 2 of the study.
* Both the sham and the active groups (3 subjects in each group) will undergo identical programming procedures at 30 and 60 days.
|
Active DBS
n=2 participants at risk
Active stimulation through the Neuropace RNS system at settings to maximally reduce tic frequency \& severity, while limiting potential stimulation-induced side-effects
NeuroPace RNS® System Deep Brain Stimulator: • There will be a one month post-operative period during which stimulation is not turned on.
* One month post-implant, subjects will be randomized one to one in a blinded fashion to receive active or sham stimulation.
* By Day 60, all subjects will be programmed to receive active stimulation. Physiological data will be collected for Specific Aim 2 of the study.
* Both the sham and the active groups (3 subjects in each group) will undergo identical programming procedures at 30 and 60 days.
|
|---|---|---|
|
Nervous system disorders
Paresthesia
|
100.0%
3/3 • Number of events 25 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
100.0%
2/2 • Number of events 18 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
|
Nervous system disorders
Presyncope
|
100.0%
3/3 • Number of events 10 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
100.0%
2/2 • Number of events 4 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
|
Nervous system disorders
Movements Involuntary
|
100.0%
3/3 • Number of events 7 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
100.0%
2/2 • Number of events 2 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
|
Nervous system disorders
Dysarthria
|
33.3%
1/3 • Number of events 1 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
50.0%
1/2 • Number of events 2 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
|
Eye disorders
Blurred vision
|
100.0%
3/3 • Number of events 5 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
100.0%
2/2 • Number of events 5 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
|
Gastrointestinal disorders
Nausea
|
66.7%
2/3 • Number of events 4 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected starting the day of the screening visit and continuing through each subject's 2 year follow-up visit.
|
Additional Information
Assistant Director of Clinical Trials
University of Florida Center for Movement Disorders & Neurorestoration
Phone: 352-294-5000
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place