OPTIMYST: Open-Label and Single-Arm Study of MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adults
Sponsor
Supernus Pharmaceuticals, Inc. (Industry)
Overall Status
Completed
CT.gov ID
NCT02610868
Collaborator
(none)
187
35
1
20
5.3
0.3
Study Details
Study Description
Brief Summary
Multicenter, open-label, outpatient study of the safety and effectiveness of repeated doses of MYOBLOC over a 1-year duration in adult subjects with troublesome sialorrhea.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Detailed Description
Multicenter, open-label, outpatient study of the safety and effectiveness of repeated doses of MYOBLOC over a 1-year duration in adult subjects with troublesome sialorrhea. The primary goal is to determine the long-term safety and tolerability of MYOBLOC (administered intraglandularly as single total dose of 3,500 Units) treatments every 13 weeks over a maximum possible duration of 1 year in adult subjects with troublesome sialorrhea. The secondary goal is to assess the magnitude of therapeutic response of MYOBLOC (administered intraglandularly as a single total dose of 3,500 Units) using effectiveness assessments performed at intervals after treatments every 13 weeks over a maximum possible duration of 1 year.
Study Design
Study Type:
Interventional
Actual Enrollment
:
187 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 3, Long-Term, Open-Label and Single-Arm Study of MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adult Subjects
Study Start Date
:
Oct 1, 2015
Actual Primary Completion Date
:
Jun 1, 2017
Actual Study Completion Date
:
Jun 1, 2017
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: MYOBLOC Injection After a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections over the course of 1 year. |
Drug: MYOBLOC
After a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments via injections into the submandibular and parotid glands. Subjects will undergo safety and effectiveness assessments at Weeks 4, 8, 13, 17, 26, 30, 39, 43, and 52, and telephone follow up will occur at Weeks 21, 34, and 47
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Occurrence, Seriousness, Severity, and Causality Assessment of Treatment Emergent Adverse Events (TEAE) [Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1]
TEAEs Related to Study Medication include TEAEs classified as Possibly, Probably and Definitely Related. Treatment Session (TS)
- Occurrence of Adverse Events of Special Interest (AESI) [Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1]
Treatment Session (TS) AESI includes: Aspiration, Aspirational pneumonia, Choking and Dysphagia.
- Columbia Suicide Rating Scale (C-SSRS): Suicidal Ideation, Behavior Scores and Intensity Scores [Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13]
Participants Analyzed does not match the Participant Flow because one participant discontinued prior to the first post-injection visit. Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U).
- Occurrence of Dental Adverse Events [Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1]
Treatment Session (TS)
Secondary Outcome Measures
- Change From Baseline in Unstimulated Salivary Flow Rate (USFR): Treatment Sessions 1-4 [Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13]
For Treatment Sessions 2-4, the change from baseline is calculated using the Week 13 value from the previous Treatment Session as the new baseline. Week 8 was only assessed in Treatment Session 1. Treatment Session (TS) Participants Analyzed does not match the Participant Flow because patient data was not always captured for each endpoint at every visit.
- Change From Baseline in Clinical Global Impression of Severity (CGI-S) [Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13]
CGI-S will be used to record the severity of illness (sialorrhea) on a 7-point scale ranging from a score of 1, "normal" to a score of 7, "among the most extremely ill patients". Treatment Session (TS) Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit.
- Clinical Global Impression of Change (CGI-C) [TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13]
CGI-C will be used to record change or improvement of illness (sialorrhea) on a 7-point scale ranging from a score of 1, "very much improved" to a score of 7, "very much worse". Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit. Treatment Session (TS)
- Change From Baseline in Drooling Frequency and Severity Scale Performed by Trained Assessor (DFSS-I) [Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13]
The DFSS will be used to assess the frequency and severity of drooling on a 5-point scale ranging from a score of 1, dry never drools to a score of 5, profuse clothing hands tray objects become wet. Drooling frequency will be assessed on a 4-point scale ranging from a score of 1, never drools to a score of 4, constantly drools. Treatment Session (TS) Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit. The severity score and frequency score are summed together to create the DFSS total score (range 2-9). A decrease from baseline indicates improvement.
- Change From Baseline in Drooling Frequency and Severity Scale Performed by Subject (DFSS-S) [Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13]
The DFSS will be used to assess the frequency and severity of drooling on a 5-point scale ranging from a score of 1, dry never drools to a score of 5, profuse clothing hands tray objects become wet. Drooling frequency will be assessed on a 4-point scale ranging from a score of 1, never drools to a score of 4, constantly drools. Treatment Session (TS) Participants Analyzed does not match the Participant Flow because patient data was not always captured for each endpoint at every visit. The severity score and frequency score are summed together to create the DFSS total score (range 2 -9). A decrease from baseline indicates improvement.
- Change From Baseline in Patient Global Impression of Severity (PGI-S) [Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13]
The PGI-S scale will be used by the subject to rate the severity of his/her illness (sialorrhea) on an 7-point scale ranging from a score of 1, "normal" to a score of 7, "among the most extremely ill". Treatment Session (TS) Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit.
- Patient Global Impression of Change (PGI-C) [TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13]
The PGI-C scale will be used by the subject to rate the change in symptoms of his/her illness (sialorrhea) on an 7-point scale ranging from a score of 1, "very much improved" to a score of 7, "very much worse". Treatment Session (TS) Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit.
- Change From Baseline in Drooling Impact Score (DIS) [Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13]
The DIS is a 10-item questionnaire with each item rated by the subject to evaluate the impact of sialorrhea on daily activities (e.g., speech, social activities). Treatment Session (TS) Participants Analyzed does not match the Participant Flow because patient data was not always captured for each endpoint at every visit. Subject is requested to compare their current status to their last study visit using a 4-point scale ranging from a score of 1, not at all to a score of 4, very much. The DIS total score ranges from 10 to 40. A negative change from baseline indicates a positive clinical outcome.
Eligibility Criteria
Criteria
Ages Eligible for Study:
18 Years
to 85 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
-
Seeking treatment for troublesome sialorrhea for at least 3 months that is occurring secondary to any disorder or related to any cause, including, but not limited to, Parkinson's Disease (PD), adult cerebral palsy, amyotrophic lateral sclerosis (ALS), stroke, traumatic brain injury, oral cancer, and side effects of other medications.
-
Able to read and provide written informed consent before enrollment into the study, or the subject's caregiver (Legally Authorized Representative) can provide written informed consent.
-
Male or female, 18 to 85 years of age (inclusive).
-
Minimum unstimulated salivary flow rate of 0.2 g/min at screening
-
Minimum Investigator's Drooling Frequency and Severity Scale (DFSS) score of 4 at screening.
-
Ability and availability to participate in the study for up to 1 year (ALS subjects: ability and availability to participate in the study for at least 6 months), based on overall health of the subject and disease prognosis
Exclusion Criteria:
-
A moderate to high risk of aspiration will exclude participation in this study. Subjects whose risk of aspiration are judged by the Investigator to be satisfactorily controlled by placement of PEG tube or G-tube for nutritional support are eligible to participate.
-
Respiratory forced vital capacity (FVC) of <20% of predicted
-
Prior botulinum toxin type A or B treatment in the salivary gland(s) identified for treatment in this study within 24 weeks before screening. Prior botulinum toxin type A or B treatment into other anatomical regions not selected for treatment in this study is not exclusionary, but must have occurred at least 12 weeks before screening
-
Subjects should be excluded if, in the Investigator's opinion, the subject failed to respond to previous treatment with botulinum toxin. Subjects should not receive nor have any plans for receiving any botulinum toxin treatment, other than the study drug (MYOBLOC), during the entire course of the study (from the point the informed consent is signed until subject's participation is complete).
-
Concomitant use, or exposure within 5 half-lives of screening, of aminoglycoside antibiotics, curare-like agents, or other agents that interfere with neuromuscular function
-
Prior salivary gland surgery
-
Current treatment or treatment at any time during the study with Coumadin® (warfarin) or similar anti-coagulant medications. Anti-platelet medications are not specifically exclusionary
-
Evidence of any clinically significant neurologic disease
-
Pregnancy or lactation
-
Anticipated or scheduled surgery during the study period. A PEG tube/G tube may be placed for nutritional support at any time during the study and will not exclude the subject from continued study participation.
-
Major surgery (requiring general anesthesia, except PEG tube/G tube placement ) within the previous 6 months before screening.
-
Current infection at the sialorrhea treatment injection site(s)
-
History of drug or alcohol abuse currently or within the previous 6 months
-
Participation in another clinical drug, device, or biological agent study within 30 days of screening or while participating in this study
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Phoenix | Arizona | United States | 85013 | |
| 2 | Scottsdale | Arizona | United States | 85258 | |
| 3 | Scottsdale | Arizona | United States | 85259 | |
| 4 | Loma Linda | California | United States | 92354 | |
| 5 | Los Angeles | California | United States | 90033 | |
| 6 | Aurora | Colorado | United States | 80045 | |
| 7 | Atlantis | Florida | United States | 33462 | |
| 8 | Boca Raton | Florida | United States | 33486 | |
| 9 | Orlando | Florida | United States | 32806 | |
| 10 | Port Charlotte | Florida | United States | 33980 | |
| 11 | Atlanta | Georgia | United States | 30329 | |
| 12 | Kansas City | Kansas | United States | 66160 | |
| 13 | Overland Park | Kansas | United States | 66211 | |
| 14 | Elkridge | Maryland | United States | 21075 | |
| 15 | Farmington Hills | Michigan | United States | 48334 | |
| 16 | Saint Louis | Missouri | United States | 63110 | |
| 17 | Las Vegas | Nevada | United States | 89145 | |
| 18 | Edison | New Jersey | United States | 08818 | |
| 19 | Centerville | Ohio | United States | 45459 | |
| 20 | Tulsa | Oklahoma | United States | 74136 | |
| 21 | Greenville | South Carolina | United States | 29615 | |
| 22 | Cordova | Tennessee | United States | 38018 | |
| 23 | San Antonio | Texas | United States | 78229 | |
| 24 | Kirkland | Washington | United States | 98034 | |
| 25 | Spokane | Washington | United States | 99202 | |
| 26 | Tacoma | Washington | United States | 98409 | |
| 27 | Grodno | Belarus | 230017 | ||
| 28 | Dnipropetrovsk | Ukraine | 49027 | ||
| 29 | Ivano-Frankivsk | Ukraine | 76008 | ||
| 30 | Kharkiv | Ukraine | 61068 | ||
| 31 | Lviv | Ukraine | 79010 | ||
| 32 | Odesa | Ukraine | 65025 | ||
| 33 | Rivne | Ukraine | 33010 | ||
| 34 | Uzhgorod | Ukraine | 88018 | ||
| 35 | Zaporizhzhya | Ukraine | 69600 |
Sponsors and Collaborators
- Supernus Pharmaceuticals, Inc.
Investigators
- Study Director: Najeebah Abdul-Musawir, MD,MBA, Supernus Pharmaceuticals, Inc.
Study Documents (Full-Text)
More Information
Publications
None provided.Responsible Party:
Supernus Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT02610868
Other Study ID Numbers:
- SN-SIAL-351
First Posted:
Nov 20, 2015
Last Update Posted:
Jul 13, 2021
Last Verified:
Nov 1, 2019
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Keywords provided by Supernus Pharmaceuticals, Inc.
Additional relevant MeSH terms:
Study Results
Participant Flow
| Recruitment Details | Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. |
|---|---|
| Pre-assignment Detail | All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). |
| Arm/Group Title | MYOBLOC: 3500U |
|---|---|
| Arm/Group Description | After a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. |
| Period Title: MYOBLOC: Treatment Session 1 | |
| STARTED | 187 |
| COMPLETED | 180 |
| NOT COMPLETED | 7 |
| Period Title: MYOBLOC: Treatment Session 1 | |
| STARTED | 177 |
| COMPLETED | 165 |
| NOT COMPLETED | 12 |
| Period Title: MYOBLOC: Treatment Session 1 | |
| STARTED | 50 |
| COMPLETED | 43 |
| NOT COMPLETED | 7 |
| Period Title: MYOBLOC: Treatment Session 1 | |
| STARTED | 24 |
| COMPLETED | 22 |
| NOT COMPLETED | 2 |
Baseline Characteristics
| Arm/Group Title | MYOBLOC |
|---|---|
| Arm/Group Description | MYOBLOC: After a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments via injections into the submandibular and parotid glands. Subjects will undergo safety and effectiveness assessments at Weeks 4, 8, 13 for Treatment Session 1; Weeks 4, 13 for Treatment Sessions 2-4. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). |
| Overall Participants | 187 |
| Age (Count of Participants) | |
| <=18 years |
0
0%
|
| Between 18 and 65 years |
85
45.5%
|
| >=65 years |
102
54.5%
|
| Sex: Female, Male (Count of Participants) | |
| Female |
58
31%
|
| Male |
129
69%
|
| Race (NIH/OMB) (Count of Participants) | |
| American Indian or Alaska Native |
1
0.5%
|
| Asian |
2
1.1%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
| Black or African American |
9
4.8%
|
| White |
175
93.6%
|
| More than one race |
0
0%
|
| Unknown or Not Reported |
0
0%
|
| Region of Enrollment (participants) [Number] | |
| United States |
68
36.4%
|
| Ukraine |
80
42.8%
|
| Belarus |
39
20.9%
|
| Unstimulated Salivary Flow Rate (USFR) (g/minute) [Mean (Standard Deviation) ] | |
| Mean (Standard Deviation) [g/minute] |
0.6322
(0.4918)
|
| Clinical Global Impression of Severity (CGI-S) (units on a scale (1-7)) [Mean (Standard Deviation) ] | |
| Mean (Standard Deviation) [units on a scale (1-7)] |
4.4
(0.94)
|
| Patient Global Impression of Severity (PGI-S) (units on a scale (1-7)) [Mean (Standard Deviation) ] | |
| Mean (Standard Deviation) [units on a scale (1-7)] |
5.3
(1.2)
|
Outcome Measures
| Title | Occurrence, Seriousness, Severity, and Causality Assessment of Treatment Emergent Adverse Events (TEAE) |
|---|---|
| Description | TEAEs Related to Study Medication include TEAEs classified as Possibly, Probably and Definitely Related. Treatment Session (TS) |
| Time Frame | Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U or 3000 U). |
| Arm/Group Title | Treatment Session 1 | Treatment Session 2 | Treatment Session 3 | Treatment Session 4 |
|---|---|---|---|---|
| Arm/Group Description | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments. |
| Measure Participants | 187 | 177 | 50 | 24 |
| Serious TEAEs |
7
3.7%
|
14
NaN
|
3
NaN
|
2
NaN
|
| Severe TEAEs |
5
2.7%
|
8
NaN
|
2
NaN
|
2
NaN
|
| TEAEs Related to Study Medication |
42
22.5%
|
26
NaN
|
4
NaN
|
2
NaN
|
| Title | Occurrence of Adverse Events of Special Interest (AESI) |
|---|---|
| Description | Treatment Session (TS) AESI includes: Aspiration, Aspirational pneumonia, Choking and Dysphagia. |
| Time Frame | Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). |
| Arm/Group Title | Treatment Session 1 | Treatment Session 2 | Treatment Session 3 | Treatment Session 4 |
|---|---|---|---|---|
| Arm/Group Description | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments. |
| Measure Participants | 187 | 177 | 50 | 24 |
| Aspiration |
0
0%
|
1
NaN
|
0
NaN
|
0
NaN
|
| Aspirational pneumonia |
0
0%
|
1
NaN
|
0
NaN
|
0
NaN
|
| Choking |
0
0%
|
0
NaN
|
0
NaN
|
0
NaN
|
| Dysphagia |
4
2.1%
|
0
NaN
|
0
NaN
|
0
NaN
|
| Title | Columbia Suicide Rating Scale (C-SSRS): Suicidal Ideation, Behavior Scores and Intensity Scores |
|---|---|
| Description | Participants Analyzed does not match the Participant Flow because one participant discontinued prior to the first post-injection visit. Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). |
| Time Frame | Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Treatment Session 1: Week 4 | Treatment Session 1: Week 8 | Treatment Session 1: Week 13 | Treatment Session 2: Week 4 | Treatment Session 2: Week 13 | Treatment Session 3: Week 4 | Treatment Session 3: Week 13 | Treatment Session 4: Week 4 | Treatment Session 4: Week 13 |
|---|---|---|---|---|---|---|---|---|---|
| Arm/Group Description | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. |
| Measure Participants | 186 | 183 | 181 | 175 | 166 | 49 | 45 | 24 | 23 |
| Suicidal Ideation |
2
1.1%
|
2
NaN
|
0
NaN
|
0
NaN
|
1
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
| Suicidal Behavior |
0
0%
|
0
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
| Title | Occurrence of Dental Adverse Events |
|---|---|
| Description | Treatment Session (TS) |
| Time Frame | Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). |
| Arm/Group Title | Treatment Session 1 | Treatment Session 2 | Treatment Session 3 | Treatment Session 4 |
|---|---|---|---|---|
| Arm/Group Description | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments. |
| Measure Participants | 187 | 177 | 50 | 24 |
| Number [participants] |
27
14.4%
|
23
NaN
|
8
NaN
|
2
NaN
|
| Title | Change From Baseline in Unstimulated Salivary Flow Rate (USFR): Treatment Sessions 1-4 |
|---|---|
| Description | For Treatment Sessions 2-4, the change from baseline is calculated using the Week 13 value from the previous Treatment Session as the new baseline. Week 8 was only assessed in Treatment Session 1. Treatment Session (TS) Participants Analyzed does not match the Participant Flow because patient data was not always captured for each endpoint at every visit. |
| Time Frame | Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). |
| Arm/Group Title | Treatment Session 1: Week 4 | Treatment Session 1: Week 8 | Treatment Session 1: Week 13 | Treatment Session 2: Week 4 | Treatment Session 2: Week 13 | Treatment Session 3: Week 4 | Treatment Session 3: Week 13 | Treatment Session 4: Week 4 | Treatment Session 4: Week 13 |
|---|---|---|---|---|---|---|---|---|---|
| Arm/Group Description | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. |
| Measure Participants | 185 | 183 | 181 | 174 | 166 | 49 | 45 | 24 | 23 |
| Mean (Standard Deviation) [g/minute] |
-0.3424
(0.3685)
|
-0.2771
(0.3144)
|
-0.1357
(0.2888)
|
-0.2272
(0.2668)
|
-0.0560
(0.2067)
|
-0.1797
(0.1975)
|
-0.0547
(0.2269)
|
-0.1592
(0.2305)
|
-0.0335
(0.1378)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 1 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 2 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 4
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 5
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 2: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.0006 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 6
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3: Week 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 7
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.1131 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 8
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4: Week 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.0026 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 9
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.2569 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
| Title | Change From Baseline in Clinical Global Impression of Severity (CGI-S) |
|---|---|
| Description | CGI-S will be used to record the severity of illness (sialorrhea) on a 7-point scale ranging from a score of 1, "normal" to a score of 7, "among the most extremely ill patients". Treatment Session (TS) Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit. |
| Time Frame | Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). |
| Arm/Group Title | Treatment Session 1: Week 4 | Treatment Session 1: Week 8 | Treatment Session 1: Week 13 | Treatment Session 2: Week 4 | Treatment Session 2: Week 13 | Treatment Session 3: Week 4 | Treatment Session 3: Week 13 | Treatment Session 4: Week 4 | Treatment Session 4: Week 13 |
|---|---|---|---|---|---|---|---|---|---|
| Arm/Group Description | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. |
| Measure Participants | 186 | 183 | 181 | 175 | 167 | 49 | 45 | 24 | 23 |
| Mean (Standard Deviation) [units on a scale] |
-1.7
(1.26)
|
-1.5
(1.22)
|
-0.9
(1.03)
|
-0.8
(1.12)
|
-0.1
(0.99)
|
-0.9
(1.13)
|
-0.2
(0.90)
|
-0.9
(0.95)
|
-0.3
(1.02)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 1 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 2 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 4
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 5
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 2: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.3130 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 6
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3: Week 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 7
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.1054 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 8
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4: Week 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.0002 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 9
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.1663 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
| Title | Clinical Global Impression of Change (CGI-C) |
|---|---|
| Description | CGI-C will be used to record change or improvement of illness (sialorrhea) on a 7-point scale ranging from a score of 1, "very much improved" to a score of 7, "very much worse". Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit. Treatment Session (TS) |
| Time Frame | TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). |
| Arm/Group Title | Treatment Session 1: Week 4 | Treatment Session 1: Week 8 | Treatment Session 1: Week 13 | Treatment Session 2: Week 4 | Treatment Session 2: Week 13 | Treatment Session 3: Week 4 | Treatment Session 3: Week 13 | Treatment Session 4: Week 4 | Treatment Session 4: Week 13 |
|---|---|---|---|---|---|---|---|---|---|
| Arm/Group Description | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. |
| Measure Participants | 186 | 183 | 181 | 175 | 167 | 49 | 45 | 24 | 23 |
| Mean (Standard Deviation) [units on a scale] |
2.3
(0.94)
|
2.4
(1.07)
|
3.0
(1.16)
|
2.4
(1.04)
|
3.1
(1.34)
|
2.4
(0.94)
|
3.0
(1.06)
|
2.5
(0.93)
|
2.8
(0.94)
|
| Title | Change From Baseline in Drooling Frequency and Severity Scale Performed by Trained Assessor (DFSS-I) |
|---|---|
| Description | The DFSS will be used to assess the frequency and severity of drooling on a 5-point scale ranging from a score of 1, dry never drools to a score of 5, profuse clothing hands tray objects become wet. Drooling frequency will be assessed on a 4-point scale ranging from a score of 1, never drools to a score of 4, constantly drools. Treatment Session (TS) Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit. The severity score and frequency score are summed together to create the DFSS total score (range 2-9). A decrease from baseline indicates improvement. |
| Time Frame | Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). |
| Arm/Group Title | Treatment Session 1: Week 4 | Treatment Session 1: Week 8 | Treatment Session 1: Week 13 | Treatment Session 2: Week 4 | Treatment Session 2: Week 13 | Treatment Session 3: Week 4 | Treatment Session 3: Week 13 | Treatment Session 4: Week 4 | Treatment Session 4: Week 13 |
|---|---|---|---|---|---|---|---|---|---|
| Arm/Group Description | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. |
| Measure Participants | 186 | 783 | 181 | 175 | 167 | 49 | 45 | 24 | 23 |
| Mean (Standard Deviation) [units on a scale] |
-2.3
(1.53)
|
-2.0
(1.52)
|
-1.2
(1.37)
|
-1.2
(1.52)
|
-0.2
(1.30)
|
-1.1
(1.36)
|
0.0
(1.33)
|
-1.5
(1.44)
|
-0.3
(0.71)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 1 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 2 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 4
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 5
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 2: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.1100 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 6
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3: Week 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 7
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 1.0000 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 8
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4: Week 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 9
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.0290 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
| Title | Change From Baseline in Drooling Frequency and Severity Scale Performed by Subject (DFSS-S) |
|---|---|
| Description | The DFSS will be used to assess the frequency and severity of drooling on a 5-point scale ranging from a score of 1, dry never drools to a score of 5, profuse clothing hands tray objects become wet. Drooling frequency will be assessed on a 4-point scale ranging from a score of 1, never drools to a score of 4, constantly drools. Treatment Session (TS) Participants Analyzed does not match the Participant Flow because patient data was not always captured for each endpoint at every visit. The severity score and frequency score are summed together to create the DFSS total score (range 2 -9). A decrease from baseline indicates improvement. |
| Time Frame | Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). |
| Arm/Group Title | Treatment Session 1: Week 4 | Treatment Session 1: Week 8 | Treatment Session 1: Week 13 | Treatment Session 2: Week 4 | Treatment Session 2: Week 13 | Treatment Session 3: Week 4 | Treatment Session 3: Week 13 | Treatment Session 4: Week 4 | Treatment Session 4: Week 13 |
|---|---|---|---|---|---|---|---|---|---|
| Arm/Group Description | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. |
| Measure Participants | 184 | 182 | 177 | 170 | 162 | 48 | 43 | 23 | 22 |
| Mean (Standard Deviation) [units on a scale] |
-1.4
(1.35)
|
-1.7
(1.58)
|
-1.0
(1.49)
|
-0.7
(0.90)
|
-0.3
(0.98)
|
-0.4
(0.64)
|
0.0
(1.00)
|
-0.4
(1.03)
|
-0.2
(1.37)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 1 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 2 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 4
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 5
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 2: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 6
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3: Week 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.0003 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 7
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.9016 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 8
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4: Week 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.0753 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 9
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.4992 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
| Title | Change From Baseline in Patient Global Impression of Severity (PGI-S) |
|---|---|
| Description | The PGI-S scale will be used by the subject to rate the severity of his/her illness (sialorrhea) on an 7-point scale ranging from a score of 1, "normal" to a score of 7, "among the most extremely ill". Treatment Session (TS) Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit. |
| Time Frame | Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). |
| Arm/Group Title | Treatment Session 1: Week 4 | Treatment Session 1: Week 8 | Treatment Session 1: Week 13 | Treatment Session 2: Week 4 | Treatment Session 2: Week 13 | Treatment Session 3: Week 4 | Treatment Session 3: Week 13 | Treatment Session 4: Week 4 | Treatment Session 4: Week 13 |
|---|---|---|---|---|---|---|---|---|---|
| Arm/Group Description | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. |
| Measure Participants | 186 | 183 | 181 | 174 | 167 | 49 | 45 | 24 | 23 |
| Mean (Standard Deviation) [units on a scale] |
-1.59
(1.548)
|
-1.41
(1.461)
|
-0.80
(1.364)
|
-0.78
(1.303)
|
-0.07
(1.245)
|
-0.94
(1.248)
|
0.04
(1.242)
|
-1.38
(1.279)
|
-0.26
(1.251)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 1 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 2 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 4
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 5
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 2: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.4567 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 6
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3: Week 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 7
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.8115 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 8
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4: Week 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 9
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.3282 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
| Title | Patient Global Impression of Change (PGI-C) |
|---|---|
| Description | The PGI-C scale will be used by the subject to rate the change in symptoms of his/her illness (sialorrhea) on an 7-point scale ranging from a score of 1, "very much improved" to a score of 7, "very much worse". Treatment Session (TS) Participants Analyzed does not match the Participant Flow because participant discontinued prior to the first post-injection visit. |
| Time Frame | TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). |
| Arm/Group Title | Treatment Session 1: Week 4 | Treatment Session 1: Week 8 | Treatment Session 1: Week 13 | Treatment Session 2: Week 4 | Treatment Session 2: Week 13 | Treatment Session 3: Week 4 | Treatment Session 3: Week 13 | Treatment Session 4: Week 4 | Treatment Session 4: Week 13 |
|---|---|---|---|---|---|---|---|---|---|
| Arm/Group Description | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. |
| Measure Participants | 186 | 183 | 181 | 174 | 167 | 49 | 45 | 24 | 23 |
| Mean (Standard Deviation) [units on a scale] |
2.5
(1.07)
|
2.7
(1.08)
|
3.0
(1.13)
|
2.6
(1.06)
|
3.2
(1.28)
|
2.7
(0.95)
|
3.2
(1.10)
|
2.5
(0.72)
|
3.4
(0.72)
|
| Title | Change From Baseline in Drooling Impact Score (DIS) |
|---|---|
| Description | The DIS is a 10-item questionnaire with each item rated by the subject to evaluate the impact of sialorrhea on daily activities (e.g., speech, social activities). Treatment Session (TS) Participants Analyzed does not match the Participant Flow because patient data was not always captured for each endpoint at every visit. Subject is requested to compare their current status to their last study visit using a 4-point scale ranging from a score of 1, not at all to a score of 4, very much. The DIS total score ranges from 10 to 40. A negative change from baseline indicates a positive clinical outcome. |
| Time Frame | Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). |
| Arm/Group Title | Treatment Session 1: Week 4 | Treatment Session 1: Week 8 | Treatment Session 1: Week 13 | Treatment Session 2: Week 4 | Treatment Session 2: Week 13 | Treatment Session 3: Week 4 | Treatment Session 3: Week 13 | Treatment Session 4: Week 4 | Treatment Session 4: Week 13 |
|---|---|---|---|---|---|---|---|---|---|
| Arm/Group Description | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. | Subjects received single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. |
| Measure Participants | 185 | 183 | 181 | 175 | 167 | 49 | 45 | 24 | 23 |
| Mean (Standard Deviation) [units on a scale] |
-5.2
(5.33)
|
-5.4
(5.16)
|
-3.4
(4.97)
|
-2.5
(4.17)
|
0.3
(3.94)
|
-2.8
(3.39)
|
-0.1
(2.11)
|
-3.6
(3.10)
|
-0.5
(2.57)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 1 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 2 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 4
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 5
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 2: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.3786 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 6
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3: Week 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 7
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 3: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.7787 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 8
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4: Week 4 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Statistical Analysis 9
| Statistical Analysis Overview | Comparison Group Selection | Treatment Session 4: Week 13 |
|---|---|---|
| Comments | ||
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.3825 |
| Comments | ||
| Method | ANCOVA | |
| Comments | ||
Adverse Events
| Time Frame | through study completion, up to 1 year | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Adverse Event Reporting Description | Systematic Assessment: Office visits were conducted on Weeks 4, 8, 13 for Treatment Session 1 and Weeks 4 and 13 for Treatment Sessions 2, 3, 4. | |||||||||
| Arm/Group Title | MYOBLOC: Treatment Session 1 | MYOBLOC: Treatment Session 2 | MYOBLOC: Treatment Session 3 | MYOBLOC: Treatment Session 4 | ALL MYOBLOC: Treatment Sessions 1-4 | |||||
| Arm/Group Description | After a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 1 at Weeks 4, 8, 13. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). | After a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 2 at Weeks 4 and 13. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). | After a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 3 at Weeks 4 and 13. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). | After a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments for Treatment Session 4 at Weeks 4 and 13. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). | After a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments. Subjects will undergo safety and effectiveness assessments at Weeks 4, 8, 13 for Treatment Session 1, Weeks 4 and 13 for Treatment Sessions 2-4. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U). | |||||
All Cause Mortality |
||||||||||
| MYOBLOC: Treatment Session 1 | MYOBLOC: Treatment Session 2 | MYOBLOC: Treatment Session 3 | MYOBLOC: Treatment Session 4 | ALL MYOBLOC: Treatment Sessions 1-4 | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 1/187 (0.5%) | 4/177 (2.3%) | 2/50 (4%) | 1/24 (4.2%) | 8/187 (4.3%) | |||||
Serious Adverse Events |
||||||||||
| MYOBLOC: Treatment Session 1 | MYOBLOC: Treatment Session 2 | MYOBLOC: Treatment Session 3 | MYOBLOC: Treatment Session 4 | ALL MYOBLOC: Treatment Sessions 1-4 | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 9/187 (4.8%) | 18/177 (10.2%) | 4/50 (8%) | 2/24 (8.3%) | 33/187 (17.6%) | |||||
| Cardiac disorders | ||||||||||
| Angina pectoris | 2/187 (1.1%) | 0/177 (0%) | 0/50 (0%) | 0/24 (0%) | 2/187 (1.1%) | |||||
| Atrial fibrillation | 0/187 (0%) | 1/177 (0.6%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Coronary artery disease | 0/187 (0%) | 1/177 (0.6%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Endocrine disorders | ||||||||||
| Inappropriate antidiuretic hormone secretion | 0/187 (0%) | 1/177 (0.6%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Gastrointestinal disorders | ||||||||||
| Large intestinal obstruction | 1/187 (0.5%) | 0/177 (0%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Infections and infestations | ||||||||||
| Pneumonia | 0/187 (0%) | 2/177 (1.1%) | 1/50 (2%) | 0/24 (0%) | 3/187 (1.6%) | |||||
| Urosepsis | 0/187 (0%) | 1/177 (0.6%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Injury, poisoning and procedural complications | ||||||||||
| Post procedural compication | 1/187 (0.5%) | 0/177 (0%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Contusion | 0/187 (0%) | 1/177 (0.6%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Fall | 0/187 (0%) | 1/177 (0.6%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Hip fracture | 0/187 (0%) | 1/177 (0.6%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Metabolism and nutrition disorders | ||||||||||
| Dehydration | 0/187 (0%) | 1/177 (0.6%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Malnutrition | 0/187 (0%) | 0/177 (0%) | 1/50 (2%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Musculoskeletal and connective tissue disorders | ||||||||||
| Intervertebral disc protrusion | 1/187 (0.5%) | 0/177 (0%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ||||||||||
| Neoplasm progression | 1/187 (0.5%) | 1/177 (0.6%) | 1/50 (2%) | 0/24 (0%) | 3/187 (1.6%) | |||||
| Prostate cancer | 0/187 (0%) | 1/177 (0.6%) | 0/50 (0%) | 1/24 (4.2%) | 2/187 (1.1%) | |||||
| Nervous system disorders | ||||||||||
| Multiple system atrophy | 1/187 (0.5%) | 0/177 (0%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Amyotrophic lateral sclerosis | 0/187 (0%) | 2/177 (1.1%) | 1/50 (2%) | 1/24 (4.2%) | 4/187 (2.1%) | |||||
| Freezing phenomenon | 0/187 (0%) | 1/177 (0.6%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Subarachnoid haemorrhage | 0/187 (0%) | 1/177 (0.6%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Psychiatric disorders | ||||||||||
| Delirium | 1/187 (0.5%) | 0/177 (0%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Mental status changes | 1/187 (0.5%) | 0/177 (0%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Respiratory, thoracic and mediastinal disorders | ||||||||||
| Pneumonia aspiration | 0/187 (0%) | 1/177 (0.6%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
| Respiratory failure | 0/187 (0%) | 1/177 (0.6%) | 0/50 (0%) | 0/24 (0%) | 1/187 (0.5%) | |||||
Other (Not Including Serious) Adverse Events |
||||||||||
| MYOBLOC: Treatment Session 1 | MYOBLOC: Treatment Session 2 | MYOBLOC: Treatment Session 3 | MYOBLOC: Treatment Session 4 | ALL MYOBLOC: Treatment Sessions 1-4 | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 44/187 (23.5%) | 34/177 (19.2%) | 7/50 (14%) | 4/24 (16.7%) | 73/187 (39%) | |||||
| Gastrointestinal disorders | ||||||||||
| Dental caries | 13/187 (7%) | 17/177 (9.6%) | 6/50 (12%) | 2/24 (8.3%) | 32/187 (17.1%) | |||||
| Dry mouth | 29/187 (15.5%) | 10/177 (5.6%) | 1/50 (2%) | 0/24 (0%) | 33/187 (17.6%) | |||||
| Nervous system disorders | ||||||||||
| Amyotrophic lateral sclerosis | 2/187 (1.1%) | 7/177 (4%) | 0/50 (0%) | 2/24 (8.3%) | 8/187 (4.3%) | |||||
Limitations/Caveats
[Not Specified]
More Information
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
| Name/Title | Regulatory Affairs Specialist |
|---|---|
| Organization | Solstice Neurosciences |
| Phone | 502-815-8148 |
| regulatoryaffairs@usworldmeds.com |
Responsible Party:
Supernus Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT02610868
Other Study ID Numbers:
- SN-SIAL-351
First Posted:
Nov 20, 2015
Last Update Posted:
Jul 13, 2021
Last Verified:
Nov 1, 2019