Trial Outcomes & Findings for Kinematic-guided BoNT-A Treatment for ET and PD Tremor (NCT NCT02427646)
NCT ID: NCT02427646
Last Updated: 2019-08-20
Results Overview
Improvement in hand tremor as determined by a reduction of \>8 points on a standardized clinical assessment tool (Fahn-Tolosa-Marin Tremor Assessment Scale) pre and post Xeomin® injection using kinematic guided injection parameters for both IPD and ET. Lower scores indicate a better outcome. Means and standard deviations are provided in the data tables. FTM minimum and maximum scores range from 0 to 92 FTM points.
Recruitment status
UNKNOWN
Study phase
PHASE2
Target enrollment
54 participants
Primary outcome timeframe
0 to 96 weeks
Results posted on
2019-08-20
Participant Flow
Protocol enrollment identifies participants who agreed to participate and sign the consent form (total N=54). However, in the total started participant flow, two participants did not have a high enough tremor severity or consistent tremor severity during the scripted tasks held during the kinematic technology assessment, leaving N=52 in the study
Participant milestones
| Measure |
Essential Tremor Treatment
IncobotulinumtoxinA: A serotype of botulinum toxins that has specificity for cleavage of SYNAPTOSOMAL-ASSOCIATED PROTEIN 25 (SNAP-25). BoNT-A's pharmacological action is to inhibit the release of acetylcholine from the neuromuscular junction. BoNT-A peripherally applied using optimal parameters by intramuscular injections to treat tremor in the most bothersome upper extremity every 16 weeks over 96 weeks. The study will be extended for those participants who benefited and will receive treatment every 12 weeks over 96 weeks. BoNT-A dose will range from 50-300 U per arm.
|
Parkinson Disease Tremor Treatment
IncobotulinumtoxinA: A serotype of botulinum toxins that has specificity for cleavage of SYNAPTOSOMAL-ASSOCIATED PROTEIN 25 (SNAP-25). BoNT-A's pharmacological action is to inhibit the release of acetylcholine from the neuromuscular junction. BoNT-A peripherally applied using optimal parameters by intramuscular injections to treat tremor in the most bothersome upper extremity every 16 weeks over 96 weeks. The study will be extended for those participants who benefited and will receive treatment every 12 weeks over 96 weeks. BoNT-A dose will range from 50-300 U per arm.
|
|---|---|---|
|
Overall Study
STARTED
|
24
|
28
|
|
Overall Study
COMPLETED
|
16
|
14
|
|
Overall Study
NOT COMPLETED
|
8
|
14
|
Reasons for withdrawal
| Measure |
Essential Tremor Treatment
IncobotulinumtoxinA: A serotype of botulinum toxins that has specificity for cleavage of SYNAPTOSOMAL-ASSOCIATED PROTEIN 25 (SNAP-25). BoNT-A's pharmacological action is to inhibit the release of acetylcholine from the neuromuscular junction. BoNT-A peripherally applied using optimal parameters by intramuscular injections to treat tremor in the most bothersome upper extremity every 16 weeks over 96 weeks. The study will be extended for those participants who benefited and will receive treatment every 12 weeks over 96 weeks. BoNT-A dose will range from 50-300 U per arm.
|
Parkinson Disease Tremor Treatment
IncobotulinumtoxinA: A serotype of botulinum toxins that has specificity for cleavage of SYNAPTOSOMAL-ASSOCIATED PROTEIN 25 (SNAP-25). BoNT-A's pharmacological action is to inhibit the release of acetylcholine from the neuromuscular junction. BoNT-A peripherally applied using optimal parameters by intramuscular injections to treat tremor in the most bothersome upper extremity every 16 weeks over 96 weeks. The study will be extended for those participants who benefited and will receive treatment every 12 weeks over 96 weeks. BoNT-A dose will range from 50-300 U per arm.
|
|---|---|---|
|
Overall Study
Lack of Efficacy
|
2
|
3
|
|
Overall Study
Physician Decision
|
1
|
4
|
|
Overall Study
Protocol Violation
|
3
|
3
|
|
Overall Study
Bothersome hand weakness
|
2
|
4
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Essential Tremor Treatment
n=24 Participants
IncobotulinumtoxinA: A serotype of botulinum toxins that has specificity for cleavage of SYNAPTOSOMAL-ASSOCIATED PROTEIN 25 (SNAP-25). BoNT-A's pharmacological action is to inhibit the release of acetylcholine from the neuromuscular junction. BoNT-A peripherally applied using optimal parameters by intramuscular injections to treat tremor in the most bothersome upper extremity every 16 weeks over 96 weeks. The study will be extended for those participants who benefited and will receive treatment every 12 weeks over 96 weeks. BoNT-A dose will range from 50-300 U per arm.
|
Parkinson Disease Tremor Treatment
n=28 Participants
IncobotulinumtoxinA: A serotype of botulinum toxins that has specificity for cleavage of SYNAPTOSOMAL-ASSOCIATED PROTEIN 25 (SNAP-25). BoNT-A's pharmacological action is to inhibit the release of acetylcholine from the neuromuscular junction. BoNT-A peripherally applied using optimal parameters by intramuscular injections to treat tremor in the most bothersome upper extremity every 16 weeks over 96 weeks. The study will be extended for those participants who benefited and will receive treatment every 12 weeks over 96 weeks. BoNT-A dose will range from 50-300 U per arm.
|
Total
n=52 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
72 years
STANDARD_DEVIATION 8.9 • n=24 Participants
|
65 years
STANDARD_DEVIATION 11.5 • n=28 Participants
|
68 years
STANDARD_DEVIATION 11 • n=52 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=24 Participants
|
7 Participants
n=28 Participants
|
18 Participants
n=52 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=24 Participants
|
21 Participants
n=28 Participants
|
34 Participants
n=52 Participants
|
|
Race and Ethnicity Not Collected
|
—
|
—
|
0 Participants
Race and Ethnicity were not collected from any participant.
|
|
Region of Enrollment
Canada
|
24 participants
n=24 Participants
|
28 participants
n=28 Participants
|
52 participants
n=52 Participants
|
|
Number of participants receiving injection in their motor-dominant limb
|
16 Participants
n=24 Participants
|
27 Participants
n=28 Participants
|
43 Participants
n=52 Participants
|
PRIMARY outcome
Timeframe: 0 to 96 weeksPopulation: Mean total FTM score was compared from baseline to each time-point. Here we state the baseline and final visit scores per group
Improvement in hand tremor as determined by a reduction of \>8 points on a standardized clinical assessment tool (Fahn-Tolosa-Marin Tremor Assessment Scale) pre and post Xeomin® injection using kinematic guided injection parameters for both IPD and ET. Lower scores indicate a better outcome. Means and standard deviations are provided in the data tables. FTM minimum and maximum scores range from 0 to 92 FTM points.
Outcome measures
| Measure |
Essential Tremor Treatment
n=24 Participants
IncobotulinumtoxinA: A serotype of botulinum toxins that has specificity for cleavage of SYNAPTOSOMAL-ASSOCIATED PROTEIN 25 (SNAP-25). BoNT-A's pharmacological action is to inhibit the release of acetylcholine from the neuromuscular junction. BoNT-A peripherally applied using optimal parameters by intramuscular injections to treat tremor in the most bothersome upper extremity every 16 weeks over 96 weeks. The study will be extended for those participants who benefited and will receive treatment every 12 weeks over 96 weeks. BoNT-A dose will range from 50-300 U per arm. There is reduction in the total number of participants at week 96 compared to week 0 due to lost to follow-up (change in medication and other symptoms arose (n=5), and discontinued from intervention (n=3)).
|
Parkinson Disease Tremor Treatment
n=28 Participants
IncobotulinumtoxinA: A serotype of botulinum toxins that has specificity for cleavage of SYNAPTOSOMAL-ASSOCIATED PROTEIN 25 (SNAP-25). BoNT-A's pharmacological action is to inhibit the release of acetylcholine from the neuromuscular junction. BoNT-A peripherally applied using optimal parameters by intramuscular injections to treat tremor in the most bothersome upper extremity every 16 weeks over 96 weeks. The study will be extended for those participants who benefited and will receive treatment every 12 weeks over 96 weeks. BoNT-A dose will range from 50-300 U per arm. There is reduction in the total number of participants at week 96 compared to week 0 due to lost to follow-up (change in medication and other symptoms arose (n=8), and discontinued from intervention (n=6)).
|
|---|---|---|
|
Clinical Tremor Rating Scale (Fahn-Tolosa-Marin Tremor Rating Scale)
Week 96
|
14.9 scores on total FTM scale
Standard Deviation 3.5
|
14.1 scores on total FTM scale
Standard Deviation 3.4
|
|
Clinical Tremor Rating Scale (Fahn-Tolosa-Marin Tremor Rating Scale)
Week 0
|
30.6 scores on total FTM scale
Standard Deviation 5.5
|
19.4 scores on total FTM scale
Standard Deviation 2.7
|
SECONDARY outcome
Timeframe: 96 weeksPopulation: Mean kinematic tremor amplitude was compared from baseline to each time-point. Here we state the baseline and final visit tremor amplitudes per group
For two subgroups of participants: (A) those with a \> 8 point improvement on the tremor rating scale and (B) those with a change ≤ 8 points on the tremor rating scale, Group A will have 50% reduction in overall tremor amplitude as measured by kinematics. Kinematic measures were graphically represented as mean angular RMS amplitude and standard deviations of the population at the wrist over three trials during scripted task. Kinematic tremor analysis is shown in angular root mean square (RMS) amplitudes
Outcome measures
| Measure |
Essential Tremor Treatment
n=24 Participants
IncobotulinumtoxinA: A serotype of botulinum toxins that has specificity for cleavage of SYNAPTOSOMAL-ASSOCIATED PROTEIN 25 (SNAP-25). BoNT-A's pharmacological action is to inhibit the release of acetylcholine from the neuromuscular junction. BoNT-A peripherally applied using optimal parameters by intramuscular injections to treat tremor in the most bothersome upper extremity every 16 weeks over 96 weeks. The study will be extended for those participants who benefited and will receive treatment every 12 weeks over 96 weeks. BoNT-A dose will range from 50-300 U per arm. There is reduction in the total number of participants at week 96 compared to week 0 due to lost to follow-up (change in medication and other symptoms arose (n=5), and discontinued from intervention (n=3)).
|
Parkinson Disease Tremor Treatment
n=28 Participants
IncobotulinumtoxinA: A serotype of botulinum toxins that has specificity for cleavage of SYNAPTOSOMAL-ASSOCIATED PROTEIN 25 (SNAP-25). BoNT-A's pharmacological action is to inhibit the release of acetylcholine from the neuromuscular junction. BoNT-A peripherally applied using optimal parameters by intramuscular injections to treat tremor in the most bothersome upper extremity every 16 weeks over 96 weeks. The study will be extended for those participants who benefited and will receive treatment every 12 weeks over 96 weeks. BoNT-A dose will range from 50-300 U per arm. There is reduction in the total number of participants at week 96 compared to week 0 due to lost to follow-up (change in medication and other symptoms arose (n=8), and discontinued from intervention (n=6)).
|
|---|---|---|
|
Kinematic Tremor Severity
Week 0
|
0.73 Angular root mean square amplitude
Standard Deviation 0.69
|
1.04 Angular root mean square amplitude
Standard Deviation 0.27
|
|
Kinematic Tremor Severity
Week 96
|
0.15 Angular root mean square amplitude
Standard Deviation 0.26
|
0.33 Angular root mean square amplitude
Standard Deviation 0.11
|
Adverse Events
Essential Tremor Treatment
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Parkinson Disease Tremor Treatment
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place