Drooling
Conditions
Keywords
Sialorrhea
Brief summary
To determine safety, tolerability and preliminary efficacy of intraglandular injections of MYOBLOC for the treatment of sialorrhea in Parkinsons' Disease patients
Detailed description
This is a multi-center, outpatient, double-blind, placebo-controlled, single treatment, sequential dose escalation study designed to evaluate the safety, tolerability and preliminary efficacy of single doses of MYOBLOC versus placebo for the treatment of sialorrhea in Parkinson's disease patients, lasting approximately 20 weeks.
Interventions
BIOLOGICALBotulinum Toxin Type B (Myobloc)
BIOLOGICALMatched placebo to Myobloc
Sponsors
Solstice Neurosciences, LLC, a subsidiary of US WorldMeds, LLC
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
* Parkinsons' Disease patients with Sialorrhea for at least 3 months
Exclusion criteria
* Patients with non-idiopathic PD parkinsonism * Patients previously exposed to botulinum toxins * Patients with a history of aspiration pneumonia, moderate/severe choking and/or moderate/severe dysphagia * Patients with prior salivary gland surgery
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Drooling Frequency & Severity Scale (DFSS)at Wk 4 Post-injection | baseline versus 4 weeks post-injection | 9 point scale, 0 = no drooling, 9 = severe drooling |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Drooling Frequency and Severity Scale (DFSS) at Wk 12 Post-injection | baseline vs 12 weeks post injection | 9 point scale (0=no drooling, 9=severe drooling) |
| Change in Unstimulated Salivary Flow Rate at Wk 4 Post-injection | baseline vs 4 weeks post-injection | saliva is collected over 5 minutes and weighed to produce a grams/minute "rate" |
| Change in Unstimulated Salivary Flow Rate at Wk 12 Post-injection | baseline vs 12 weeks post-injection | saliva collected over 5 minutes and weighed to produce a grams/minute "rate" |
Countries
United States
Contacts
STUDY_DIRECTORJonathan Rubin, MD,MBA
Supernus Pharmaceuticals, Inc.
Participant flow
Recruitment details
24 centers were initiated to screen and enroll subjects. Enrollment began in July 2007 and completed in March 2008. 54 subjects were enrolled at 17 centers
Participants by arm
| Arm | Count |
|---|---|
| 1500U Myobloc 1500U Myobloc | 14 |
| 2500U Myobloc 2500U Myobloc | 12 |
| 3500U Myobloc 3500U Myobloc | 13 |
| Placebo Placebo | 15 |
| Total | 54 |
Baseline characteristics
| Characteristic | 2500U Myobloc | 3500U Myobloc | 1500U Myobloc | Placebo | Total |
|---|---|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 10 Participants | 13 Participants | 9 Participants | 12 Participants | 44 Participants |
| Age, Categorical Between 18 and 65 years | 2 Participants | 0 Participants | 5 Participants | 3 Participants | 10 Participants |
| Age, Continuous | 71 years STANDARD_DEVIATION 8 | 74 years STANDARD_DEVIATION 5 | 67 years STANDARD_DEVIATION 7 | 71 years STANDARD_DEVIATION 11 | 71 years STANDARD_DEVIATION 8 |
| Region of Enrollment United States | 12 participants | 13 participants | 14 participants | 15 participants | 54 participants |
| Sex: Female, Male Female | 0 Participants | 2 Participants | 2 Participants | 2 Participants | 6 Participants |
| Sex: Female, Male Male | 12 Participants | 11 Participants | 12 Participants | 13 Participants | 48 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| other Total, other adverse events | 2 / — | 3 / — | 2 / — | 1 / — |
| serious Total, serious adverse events | 0 / — | 1 / — | 1 / — | 3 / — |
Outcome results
Primary
Change in Drooling Frequency & Severity Scale (DFSS)at Wk 4 Post-injection
9 point scale, 0 = no drooling, 9 = severe drooling
Time frame: baseline versus 4 weeks post-injection
Population: Intent to Treat (ITT)
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| 1500U Myobloc | Change in Drooling Frequency & Severity Scale (DFSS)at Wk 4 Post-injection | -2.1 points on a scale | Standard Deviation 1.2 |
| 2500U Myobloc | Change in Drooling Frequency & Severity Scale (DFSS)at Wk 4 Post-injection | -3.3 points on a scale | Standard Deviation 1.4 |
| 3500U Myobloc | Change in Drooling Frequency & Severity Scale (DFSS)at Wk 4 Post-injection | -3.5 points on a scale | Standard Deviation 1 |
| Placebo | Change in Drooling Frequency & Severity Scale (DFSS)at Wk 4 Post-injection | -1.3 points on a scale | Standard Deviation 1.3 |
Secondary
Change in Drooling Frequency and Severity Scale (DFSS) at Wk 12 Post-injection
9 point scale (0=no drooling, 9=severe drooling)
Time frame: baseline vs 12 weeks post injection
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| 1500U Myobloc | Change in Drooling Frequency and Severity Scale (DFSS) at Wk 12 Post-injection | -1.9 points on a scale | Standard Deviation 1.5 |
| 2500U Myobloc | Change in Drooling Frequency and Severity Scale (DFSS) at Wk 12 Post-injection | -2.2 points on a scale | Standard Deviation 1.6 |
| 3500U Myobloc | Change in Drooling Frequency and Severity Scale (DFSS) at Wk 12 Post-injection | -2.2 points on a scale | Standard Deviation 0.8 |
| Placebo | Change in Drooling Frequency and Severity Scale (DFSS) at Wk 12 Post-injection | -1.2 points on a scale | Standard Deviation 1.3 |
Secondary
Change in Unstimulated Salivary Flow Rate at Wk 12 Post-injection
saliva collected over 5 minutes and weighed to produce a grams/minute rate
Time frame: baseline vs 12 weeks post-injection
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| 1500U Myobloc | Change in Unstimulated Salivary Flow Rate at Wk 12 Post-injection | -0.414 grams/minute | Standard Deviation 0.37 |
| 2500U Myobloc | Change in Unstimulated Salivary Flow Rate at Wk 12 Post-injection | -0.166 grams/minute | Standard Deviation 0.31 |
| 3500U Myobloc | Change in Unstimulated Salivary Flow Rate at Wk 12 Post-injection | -0.028 grams/minute | Standard Deviation 0.21 |
| Placebo | Change in Unstimulated Salivary Flow Rate at Wk 12 Post-injection | 0.108 grams/minute | Standard Deviation 0.35 |
Secondary
Change in Unstimulated Salivary Flow Rate at Wk 4 Post-injection
saliva is collected over 5 minutes and weighed to produce a grams/minute rate
Time frame: baseline vs 4 weeks post-injection
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| 1500U Myobloc | Change in Unstimulated Salivary Flow Rate at Wk 4 Post-injection | -0.497 grams/minute | Standard Deviation 0.38 |
| 2500U Myobloc | Change in Unstimulated Salivary Flow Rate at Wk 4 Post-injection | -.381 grams/minute | Standard Deviation 0.34 |
| 3500U Myobloc | Change in Unstimulated Salivary Flow Rate at Wk 4 Post-injection | -0.146 grams/minute | Standard Deviation 0.14 |
| Placebo | Change in Unstimulated Salivary Flow Rate at Wk 4 Post-injection | -0.052 grams/minute | Standard Deviation 0.33 |