Trial Outcomes & Findings for Investigation of the Efficacy and Safety of IncobotulinumtoxinA (Xeomin) in Parkinson's Tremor: A Customized Approach (NCT NCT02419313)

NCT ID: NCT02419313

Last Updated: 2016-03-14

Results Overview

The primary outcome measure in this protocol is significant improvement of tremor (equal or over 2 grade improvement) of the Unified Parkinson's Disease Rating Scale 4 weeks after Xeomin injection. The score is 0 to 4 , ) being no tremor and 4 severe tremor. The higher the score, the more severe the tremor.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 33 participants
• Primary outcome timeframe: 4 weeks
• Results posted on: 2016-03-14

Participant Flow

Participant milestones

Measure	Placebo First, Then Incobotulinumtoxin A Subjects will all receive injections with saline first. At 12 weeks, these subjects will then cross over and receive Xeomin in the same distribution as their second treatment.	Incobotulinumtoxin A First, Then Placebo Subjects will all receive injections with incobotulinumtoxinA injections first. At 12 weeks, these subjects will then cross over and receive placebo in the same distribution as their second treatment.
First Intervention STARTED	18	15
First Intervention COMPLETED	16	15
First Intervention NOT COMPLETED	2	0
Second Intervention STARTED	16	15
Second Intervention COMPLETED	15	15
Second Intervention NOT COMPLETED	1	0

Reasons for withdrawal

Measure	Placebo First, Then Incobotulinumtoxin A Subjects will all receive injections with saline first. At 12 weeks, these subjects will then cross over and receive Xeomin in the same distribution as their second treatment.	Incobotulinumtoxin A First, Then Placebo Subjects will all receive injections with incobotulinumtoxinA injections first. At 12 weeks, these subjects will then cross over and receive placebo in the same distribution as their second treatment.
First Intervention Lost to Follow-up	2	0
Second Intervention Lost to Follow-up	1	0

Baseline Characteristics

Investigation of the Efficacy and Safety of IncobotulinumtoxinA (Xeomin) in Parkinson's Tremor: A Customized Approach

Baseline characteristics by cohort

Measure	All Study Participants n=30 Participants All participants included in this crossover design.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	18 Participants n=5 Participants
Age, Categorical >=65 years	12 Participants n=5 Participants
Age, Continuous	61.7 years n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	24 Participants n=5 Participants
Region of Enrollment United States	30 participants n=5 Participants

PRIMARY outcome

Timeframe: 4 weeks

The primary outcome measure in this protocol is significant improvement of tremor (equal or over 2 grade improvement) of the Unified Parkinson's Disease Rating Scale 4 weeks after Xeomin injection. The score is 0 to 4 , ) being no tremor and 4 severe tremor. The higher the score, the more severe the tremor.

Outcome measures

Measure	Placebo, Saline n=30 Participants Subjects randomized to receive the placebo ( saline) will receive an equivalent volume as the active study drug (1cc). The injections will be into -10 hand and forearm muscles. A series of rating scale and an examination will take place prior to treatment and at 4 and 8 weeks post treatment. The subject will then cross over to an Active Intervention arm to receive incobotulinumtoxinA which will be injected in the same pattern as the saline. Saline: same volume as injected for incobotulinum toxin A into forearm muscles under EMG guidance.	incobotulinumtoxinA, Xeomin n=30 Participants Subjects will all receive injections with incobotulinumtoxinA injections, 100unit/cc into 6-10 muscles of the forearm. A series of rating scale and an examination will take place prior to treatment and at 4 and 8 weeks post treatment. These subjects will then cross over and receive placebo ( saline) in the same distribution as their second treatment. incobotulinumtoxinA: Subjects randomized to the active drug intervention arm will receive incobotulinumtoxinA (Xeomin). A series of rating scales and examinations will take place prior to treatment and for 24 weeks after treatment. At 12 weeks the subjects will cross over to the placebo (saline) arm.
Unified Parkinsons Disease Rating Scale (UPDRS) Tremor Scale	0 participants	8 participants

SECONDARY outcome

Timeframe: 4 weeks

The PGIC is a 7 point scale that requires the clinician to assess how much the patient's pain has improved or worsened relative to a baseline state at the beginning of the intervention. and rated as:

No change (or condition has gotten worse) (1) Almost the same, hardly any change at all (2) A little better, but no noticeable change (3) Somewhat better, but the change has not made any real difference (4) Moderately better, and a slight but noticeable change (5) Better and a definite improvement that has made a real and worthwhile difference (6) A great deal better and a considerable improvement that has made all the difference (7)improved

This outcome is number of patients who chose a 6 or above on the PGIC 6 weeks after treatment.

Outcome measures

Measure	Placebo, Saline n=30 Participants Subjects randomized to receive the placebo ( saline) will receive an equivalent volume as the active study drug (1cc). The injections will be into -10 hand and forearm muscles. A series of rating scale and an examination will take place prior to treatment and at 4 and 8 weeks post treatment. The subject will then cross over to an Active Intervention arm to receive incobotulinumtoxinA which will be injected in the same pattern as the saline. Saline: same volume as injected for incobotulinum toxin A into forearm muscles under EMG guidance.	incobotulinumtoxinA, Xeomin n=30 Participants Subjects will all receive injections with incobotulinumtoxinA injections, 100unit/cc into 6-10 muscles of the forearm. A series of rating scale and an examination will take place prior to treatment and at 4 and 8 weeks post treatment. These subjects will then cross over and receive placebo ( saline) in the same distribution as their second treatment. incobotulinumtoxinA: Subjects randomized to the active drug intervention arm will receive incobotulinumtoxinA (Xeomin). A series of rating scales and examinations will take place prior to treatment and for 24 weeks after treatment. At 12 weeks the subjects will cross over to the placebo (saline) arm.
Number of Patients Whose Patient Global Impression of Change (PGIC) Improved	0 participants	10 participants

SECONDARY outcome

Timeframe: 4 Weeks

This scale measures the amplitude of the tremor. For instance tremor of more than 4cm oscillation is grade 4. UPDRS tremor scale is 0-4 , 4 being severe tremor. Significant improvement for this protocol considered two grades of improvement .

Outcome measures

Measure	Placebo, Saline n=30 Participants Subjects randomized to receive the placebo ( saline) will receive an equivalent volume as the active study drug (1cc). The injections will be into -10 hand and forearm muscles. A series of rating scale and an examination will take place prior to treatment and at 4 and 8 weeks post treatment. The subject will then cross over to an Active Intervention arm to receive incobotulinumtoxinA which will be injected in the same pattern as the saline. Saline: same volume as injected for incobotulinum toxin A into forearm muscles under EMG guidance.	incobotulinumtoxinA, Xeomin n=30 Participants Subjects will all receive injections with incobotulinumtoxinA injections, 100unit/cc into 6-10 muscles of the forearm. A series of rating scale and an examination will take place prior to treatment and at 4 and 8 weeks post treatment. These subjects will then cross over and receive placebo ( saline) in the same distribution as their second treatment. incobotulinumtoxinA: Subjects randomized to the active drug intervention arm will receive incobotulinumtoxinA (Xeomin). A series of rating scales and examinations will take place prior to treatment and for 24 weeks after treatment. At 12 weeks the subjects will cross over to the placebo (saline) arm.
Patients With Significant Improvement in Unified Parkinsons Disease Rating Tremor Scale	0 participants	8 participants

Adverse Events

Placebo, Saline

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

incobotulinumtoxinA, Xeomin

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Placebo, Saline n=33 participants at risk；n=30 participants at risk Subjects randomized to receive the placebo ( saline) will receive an equivalent volume as the active study drug (1cc). The injections will be into -10 hand and forearm muscles. A series of rating scale and an examination will take place prior to treatment and at 4 and 8 weeks post treatment. The subject will then cross over to an Active Intervention arm to receive incobotulinumtoxinA which will be injected in the same pattern as the saline. Saline: same volume as injected for incobotulinum toxin A into forearm muscles under EMG guidance.	incobotulinumtoxinA, Xeomin n=33 participants at risk；n=30 participants at risk Subjects will all receive injections with incobotulinumtoxinA injections, 100unit/cc into 6-10 muscles of the forearm. A series of rating scale and an examination will take place prior to treatment and at 4 and 8 weeks post treatment. These subjects will then cross over and receive placebo ( saline) in the same distribution as their second treatment. incobotulinumtoxinA: Subjects randomized to the active drug intervention arm will receive incobotulinumtoxinA (Xeomin). A series of rating scales and examinations will take place prior to treatment and for 24 weeks after treatment. At 12 weeks the subjects will cross over to the placebo (saline) arm.
Musculoskeletal and connective tissue disorders muscle weakness	9.1% 3/33 • Number of events 3 • 6 months Every 4 weeks regular check up Patient to call immediately if adverse effect happened as above	9.1% 3/33 • Number of events 3 • 6 months Every 4 weeks regular check up Patient to call immediately if adverse effect happened as above

Additional Information

Bahman Jabbari M.D , Professor Emeritus- Neurology

Yale-Department of Neurology

Phone: 914-482-8542

Email: bahman.jabbari@yale.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place