Trial Outcomes & Findings for Thalamic Deep Brain Stimulation for Secondary Dystonia in Children and Young Adults (NCT NCT03078816)
NCT ID: NCT03078816
Last Updated: 2020-10-08
Results Overview
Rating scale that measures movement and disability related to dystonia, range 0-120 motor, 0-30 disability , higher number indicates more severe dystonia Change from Baseline in Burke-Fahn-Marsden Dystonia Rating Scale
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
5 participants
Primary outcome timeframe
Change from baseline to 12 months postoperatively
Results posted on
2020-10-08
Participant Flow
Participant milestones
| Measure |
DBS Active
Participants will have DBS placement and active stimulation.The following components will be used:
* Activa PC Primary Cell Neurostimulator(Model 37601)
* Activa RC Rechargeable Neurostimulator(Model 37612)
* Activa SC Single Cell Neurostimulator(Models 37602 and 37603)
* DBS Lead(Model 3387)
* DBS Extension(Models 37085/6)
* Patient Programmer(Model 37642)
* Test Stimulator(Model 3625)
* N'Vision Clinician Programmer(Model 8840)
* N'Vision Software Application Card(Model 8870) Activa PC Primary Cell Neurostimulator(Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
|
|---|---|
|
Overall Study
STARTED
|
5
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
DBS Active
Participants will have DBS placement and active stimulation.The following components will be used:
* Activa PC Primary Cell Neurostimulator(Model 37601)
* Activa RC Rechargeable Neurostimulator(Model 37612)
* Activa SC Single Cell Neurostimulator(Models 37602 and 37603)
* DBS Lead(Model 3387)
* DBS Extension(Models 37085/6)
* Patient Programmer(Model 37642)
* Test Stimulator(Model 3625)
* N'Vision Clinician Programmer(Model 8840)
* N'Vision Software Application Card(Model 8870) Activa PC Primary Cell Neurostimulator(Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
Thalamic Deep Brain Stimulation for Secondary Dystonia in Children and Young Adults
Baseline characteristics by cohort
| Measure |
DBS Active
n=4 Participants
All participants will be enrolled in DBS placement and active stimulation. The following components will be used:
* Activa PC Primary Cell Neurostimulator - (Model 37601)
* Activa RC Rechargeable Neurostimulator - (Model 37612)
* Activa SC Single Cell Neurostimulator (Models 37602 and 37603)
* DBS Lead - (Model 3387)
* DBS Extension - (Models 37085/6)
* Patient Programmer - (Model 37642)
* Test Stimulator - (Model 3625)
* N'Vision Clinician Programmer - (Model 8840)
* N'Vision Software Application Card - (Model 8870)
Activa PC Primary Cell Neurostimulator - (Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
|
|---|---|
|
Age, Categorical
<=18 years
|
3 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
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1 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
14.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White/ not Hispanic
|
2 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White/ hispanic
|
1 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
other/not hispanic
|
1 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Change from baseline to 12 months postoperativelyRating scale that measures movement and disability related to dystonia, range 0-120 motor, 0-30 disability , higher number indicates more severe dystonia Change from Baseline in Burke-Fahn-Marsden Dystonia Rating Scale
Outcome measures
| Measure |
DBS Active
n=4 Participants
Participants will have DBS placement and active stimulation.The following components will be used:
* Activa PC Primary Cell Neurostimulator(Model 37601)
* Activa RC Rechargeable Neurostimulator(Model 37612)
* Activa SC Single Cell Neurostimulator(Models 37602 and 37603)
* DBS Lead(Model 3387)
* DBS Extension(Models 37085/6)
* Patient Programmer(Model 37642)
* Test Stimulator(Model 3625)
* N'Vision Clinician Programmer(Model 8840)
* N'Vision Software Application Card(Model 8870) Activa PC Primary Cell Neurostimulator(Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
|
|---|---|
|
Change From Baseline in Burke-Fahn-Marsden Dystonia Rating Scale
|
14.7 change in points on a scale
Standard Error 11.8
|
PRIMARY outcome
Timeframe: baseline to 12 months postoperativelyQuality of life measure, scored 0-100, larger scores indicate greater hinderance (ie. lower quality of life)
Outcome measures
| Measure |
DBS Active
n=4 Participants
Participants will have DBS placement and active stimulation.The following components will be used:
* Activa PC Primary Cell Neurostimulator(Model 37601)
* Activa RC Rechargeable Neurostimulator(Model 37612)
* Activa SC Single Cell Neurostimulator(Models 37602 and 37603)
* DBS Lead(Model 3387)
* DBS Extension(Models 37085/6)
* Patient Programmer(Model 37642)
* Test Stimulator(Model 3625)
* N'Vision Clinician Programmer(Model 8840)
* N'Vision Software Application Card(Model 8870) Activa PC Primary Cell Neurostimulator(Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
|
|---|---|
|
Percent Change in Pediatric Quality of Life Inventory (PedsQL)
|
27 percent change
Interval 0.0 to 54.8
|
PRIMARY outcome
Timeframe: Change from baseline to 12 months postoperativelySeverity scale for secondary dystonia, range 0-32, higher scores indicates more severe dystonia
Outcome measures
| Measure |
DBS Active
n=4 Participants
Participants will have DBS placement and active stimulation.The following components will be used:
* Activa PC Primary Cell Neurostimulator(Model 37601)
* Activa RC Rechargeable Neurostimulator(Model 37612)
* Activa SC Single Cell Neurostimulator(Models 37602 and 37603)
* DBS Lead(Model 3387)
* DBS Extension(Models 37085/6)
* Patient Programmer(Model 37642)
* Test Stimulator(Model 3625)
* N'Vision Clinician Programmer(Model 8840)
* N'Vision Software Application Card(Model 8870) Activa PC Primary Cell Neurostimulator(Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
|
|---|---|
|
Change in Barry Albright Dystonia Rating Scale
|
0.25 average change of points on a scale
Standard Error 3.2
|
PRIMARY outcome
Timeframe: change from baseline to 12 months postoperativelyRating scale that measures movement and disability related to dystonia, range 0-120 motor, 0-30 disability , higher number indicates more severe dystonia. These ratings were carried out retroactively by a neurologist who was unfamiliar with the four study participants and who had no knowledge of their unblinded scores.
Outcome measures
| Measure |
DBS Active
n=4 Participants
Participants will have DBS placement and active stimulation.The following components will be used:
* Activa PC Primary Cell Neurostimulator(Model 37601)
* Activa RC Rechargeable Neurostimulator(Model 37612)
* Activa SC Single Cell Neurostimulator(Models 37602 and 37603)
* DBS Lead(Model 3387)
* DBS Extension(Models 37085/6)
* Patient Programmer(Model 37642)
* Test Stimulator(Model 3625)
* N'Vision Clinician Programmer(Model 8840)
* N'Vision Software Application Card(Model 8870) Activa PC Primary Cell Neurostimulator(Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
|
|---|---|
|
Change in Blinded Burke-Fahn-Marsden Dystonia Rating Scale
|
4.12 change in points on a scale
Standard Error 3.96
|
SECONDARY outcome
Timeframe: Change from baseline to 12 months postoperativelyMeasure of spasticity, range 0-32, higher values indicate more spasticity
Outcome measures
| Measure |
DBS Active
n=4 Participants
Participants will have DBS placement and active stimulation.The following components will be used:
* Activa PC Primary Cell Neurostimulator(Model 37601)
* Activa RC Rechargeable Neurostimulator(Model 37612)
* Activa SC Single Cell Neurostimulator(Models 37602 and 37603)
* DBS Lead(Model 3387)
* DBS Extension(Models 37085/6)
* Patient Programmer(Model 37642)
* Test Stimulator(Model 3625)
* N'Vision Clinician Programmer(Model 8840)
* N'Vision Software Application Card(Model 8870) Activa PC Primary Cell Neurostimulator(Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
|
|---|---|
|
Change in Modified Ashworth Scale - Upper Limbs
|
0.29 percentage change in points on a scale
Interval 0.0 to 80.3
|
SECONDARY outcome
Timeframe: Change from baseline to 12 months postoperativelyPopulation: Syllable rates collected from 2 out of the 4 participants as reported here.
Articulation, range (min 6- no upper limit), longer times indicate less articulation/more difficulty with speech
Outcome measures
| Measure |
DBS Active
n=2 Participants
Participants will have DBS placement and active stimulation.The following components will be used:
* Activa PC Primary Cell Neurostimulator(Model 37601)
* Activa RC Rechargeable Neurostimulator(Model 37612)
* Activa SC Single Cell Neurostimulator(Models 37602 and 37603)
* DBS Lead(Model 3387)
* DBS Extension(Models 37085/6)
* Patient Programmer(Model 37642)
* Test Stimulator(Model 3625)
* N'Vision Clinician Programmer(Model 8840)
* N'Vision Software Application Card(Model 8870) Activa PC Primary Cell Neurostimulator(Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
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|---|---|
|
Change in Diadochokinetic Syllable Rates
|
3.25 change in number utterances per 15 sec
Standard Deviation 4.75
|
SECONDARY outcome
Timeframe: Change from baseline to 12 months postoperativelyPopulation: The Children's Memory Scale was not administered.
Will include the following subtests: Memory for Faces, Dot Locations, and Digit Span
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Change from baseline to 12 months postoperativelyPopulation: Behavioral Assessment System was able to be completed by only one of the four participants. Categorical description of "atypicality" is all that was reported for the baseline and 12 month follow-up on this participant (participant 4), and no quantitative score was recorded.
Mood and behavior assessment, main use as a screening tool for depression.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Change from baseline to 12 months postoperativelyThis scale is used to measure spasticity, which is a velocity-dependent increase in muscle stretch reflexes associated with increased muscle tone as a component of upper motor neuron syndrome. It is scored 0-4 with higher scores indicating greater severity.
Outcome measures
| Measure |
DBS Active
n=4 Participants
Participants will have DBS placement and active stimulation.The following components will be used:
* Activa PC Primary Cell Neurostimulator(Model 37601)
* Activa RC Rechargeable Neurostimulator(Model 37612)
* Activa SC Single Cell Neurostimulator(Models 37602 and 37603)
* DBS Lead(Model 3387)
* DBS Extension(Models 37085/6)
* Patient Programmer(Model 37642)
* Test Stimulator(Model 3625)
* N'Vision Clinician Programmer(Model 8840)
* N'Vision Software Application Card(Model 8870) Activa PC Primary Cell Neurostimulator(Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
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|---|---|
|
Change in Modified Ashworth Scale Spasticity Ratings - Lower Limbs
|
0.26 percentage change in points on a scale
Interval -29.6 to 58.7
|
SECONDARY outcome
Timeframe: baseline to 12 months postoperativelyPopulation: The Kaufman Brief Intelligence Test was only administered to 3 of the 4 participants.
Kaufman Brief Intelligence Test Second Edition (KBIT-2) is a brief measure of verbal and nonverbal intelligence used with individuals ages 4 through 90 years, raw scores 0 - unlimited, with higher scores indicating higher ability.
Outcome measures
| Measure |
DBS Active
n=3 Participants
Participants will have DBS placement and active stimulation.The following components will be used:
* Activa PC Primary Cell Neurostimulator(Model 37601)
* Activa RC Rechargeable Neurostimulator(Model 37612)
* Activa SC Single Cell Neurostimulator(Models 37602 and 37603)
* DBS Lead(Model 3387)
* DBS Extension(Models 37085/6)
* Patient Programmer(Model 37642)
* Test Stimulator(Model 3625)
* N'Vision Clinician Programmer(Model 8840)
* N'Vision Software Application Card(Model 8870) Activa PC Primary Cell Neurostimulator(Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
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|---|---|
|
Change in Kaufman Brief Intelligence Test - Second Addition
|
6.67 change BL- 12 mo. raw score on a scale
Standard Deviation 4.16
|
SECONDARY outcome
Timeframe: baseline to 12 months postoperativelyThis scale is a measurement of quality of life related to dystonia, with lower scores indicating greater quality of life and high scores indicating more hinderance. It is scored 0-100.
Outcome measures
| Measure |
DBS Active
n=4 Participants
Participants will have DBS placement and active stimulation.The following components will be used:
* Activa PC Primary Cell Neurostimulator(Model 37601)
* Activa RC Rechargeable Neurostimulator(Model 37612)
* Activa SC Single Cell Neurostimulator(Models 37602 and 37603)
* DBS Lead(Model 3387)
* DBS Extension(Models 37085/6)
* Patient Programmer(Model 37642)
* Test Stimulator(Model 3625)
* N'Vision Clinician Programmer(Model 8840)
* N'Vision Software Application Card(Model 8870) Activa PC Primary Cell Neurostimulator(Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
|
|---|---|
|
Change in Burke-Fahn-Marsden Dystonia Disability Subscale
|
15.7 percentage change in score on a scale
Interval -9.1 to 53.8
|
SECONDARY outcome
Timeframe: baseline to 12 months posoperativelyPopulation: Outcome data for this scale is only available for two of the four participants because the scale was not administered during the 12 month follow up visit for these two patients.
This scale is a measurement of quality of life related to dystonia, with lower scores indicating greater quality of life and high scores indicating more hinderance. It is scored 0-199
Outcome measures
| Measure |
DBS Active
n=2 Participants
Participants will have DBS placement and active stimulation.The following components will be used:
* Activa PC Primary Cell Neurostimulator(Model 37601)
* Activa RC Rechargeable Neurostimulator(Model 37612)
* Activa SC Single Cell Neurostimulator(Models 37602 and 37603)
* DBS Lead(Model 3387)
* DBS Extension(Models 37085/6)
* Patient Programmer(Model 37642)
* Test Stimulator(Model 3625)
* N'Vision Clinician Programmer(Model 8840)
* N'Vision Software Application Card(Model 8870) Activa PC Primary Cell Neurostimulator(Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
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|---|---|
|
Change in Modified Unified Parkinson's Disease Rating Scale - Second Edition
|
19.25 percentage change in points on a scale
Interval 15.0 to 23.5
|
Adverse Events
DBS Active
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
DBS Active
n=4 participants at risk
All participants will be enrolled in DBS placement and active stimulation. The following components will be used:
* Activa PC Primary Cell Neurostimulator - (Model 37601)
* Activa RC Rechargeable Neurostimulator - (Model 37612)
* Activa SC Single Cell Neurostimulator (Models 37602 and 37603)
* DBS Lead - (Model 3387)
* DBS Extension - (Models 37085/6)
* Patient Programmer - (Model 37642)
* Test Stimulator - (Model 3625)
* N'Vision Clinician Programmer - (Model 8840)
* N'Vision Software Application Card - (Model 8870)
Activa PC Primary Cell Neurostimulator - (Model 37601): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques. The device will deliver constant stimulation to the thalamus using settings programmed by study team.
Activa RC Rechargeable Neurostimulator - (Model 37612): Deep Brain Stimulator system will be implanted using standard neurosurgical techniques.
|
|---|---|
|
Gastrointestinal disorders
Diarrhea
|
25.0%
1/4 • Number of events 1 • Patients were monitored for two years following surgery.
|
|
General disorders
teeth hurt
|
25.0%
1/4 • Number of events 1 • Patients were monitored for two years following surgery.
|
|
General disorders
worsening hand movements
|
25.0%
1/4 • Patients were monitored for two years following surgery.
|
|
General disorders
drooling in sleep
|
25.0%
1/4 • Number of events 2 • Patients were monitored for two years following surgery.
|
|
General disorders
full body jerks
|
25.0%
1/4 • Number of events 1 • Patients were monitored for two years following surgery.
|
|
General disorders
fever or flu
|
25.0%
1/4 • Number of events 1 • Patients were monitored for two years following surgery.
|
|
General disorders
attitude
|
25.0%
1/4 • Number of events 1 • Patients were monitored for two years following surgery.
|
|
General disorders
viral illness
|
25.0%
1/4 • Number of events 1 • Patients were monitored for two years following surgery.
|
|
General disorders
Weight Gain
|
25.0%
1/4 • Number of events 1 • Patients were monitored for two years following surgery.
|
|
General disorders
stool incontinence
|
25.0%
1/4 • Number of events 1 • Patients were monitored for two years following surgery.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place