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This study will evaluate the efficacy and safety of MYOBLOC in the treatment of Sialorrhea (drooling), which can be a symptom of many disease conditions. MYOBLOC will be injected directly into the salivary glands. MYOBLOC has been shown in previous trials to safely decrease saliva production, thereby demonstrating its potential as a safe and effective treatment for troublesome sialorrhea.
Primary Objective: To determine the efficacy of MYOBLOC (administered via intraglandular as a single total dose of 2,500 Units or 3,500 Units) versus placebo in the treatment of troublesome sialorrhea in adult subjects via the assessment of unstimulated salivary flow rate and clinical global impression of sialorrhea severity and improvement at Week 4 post-injection (Part A). It is hypothesized that both MYOBLOC doses will achieve greater efficacy than placebo in relieving sialorrhea at 4 weeks post-injection.
To compare the safety and tolerability of MYOBLOC versus placebo over a 13 week post-injection period (Part A).
Secondary Objective: To assess the onset and duration of therapeutic response of MYOBLOC using efficacy assessments performed at Weeks 1, 2, 4, 8, and 13 after the first treatment (Part A).
To assess the duration of therapeutic response of MYOBLOC (administered via intraglandular as a single total dose of 3,500 Units) using efficacy assessments performed at intervals after treatments every 13 weeks over a maximum possible duration of 65 weeks (Part B).
To determine the long-term safety and tolerability of MYOBLOC treatments every 13 weeks over a maximum possible duration of 65 weeks (Part B).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MYOBLOC 2500 U | Active Comparator | Subjects will receive specified dose of MYOBLOC |
|
| MYOBLOC 3500 U | Active Comparator | Subjects will receive specified dose of MYOBLOC |
|
| Placebo | Placebo Comparator | Subjects will receive volume matched Placebo |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MYOBLOC | Drug | MYOBLOC (rimabotulinumtoxinB) Injection, or botulinum toxin type B, is the "B" serotype of botulinum toxin. It is the only commercially available "B" serotype, and also the only available botulinum toxin that does not require reconstitution for use. |
| Measure | Description | Time Frame |
|---|---|---|
| Unstimulated Salivary Flow Rate (USFR) at Week 4 Post-injection Visit (Part A) | Change weight of expectorated saliva at a Week 4 post-injection visit. | 4 Weeks |
| Clinical Global Impression Change (CGI-C) at Week 4 Post-injection (Part A) | CGI-C was assessed on a 7-point scale ranging from "very much improved" to "very much worse" with 1 assigned to "very much improved" and 7 assigned to "very much worse"; ranging from a minimum score of 1 and a maximum score of 7. | 4 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jonathan Rubin, MD | Supernus Pharmaceuticals, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Loma Linda | California | 92354 | United States | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 21887710 | Result | Chinnapongse R, Gullo K, Nemeth P, Zhang Y, Griggs L. Safety and efficacy of botulinum toxin type B for treatment of sialorrhea in Parkinson's disease: a prospective double-blind trial. Mov Disord. 2012 Feb;27(2):219-26. doi: 10.1002/mds.23929. Epub 2011 Sep 1. | |
| 14718694 | Result | Ondo WG, Hunter C, Moore W. A double-blind placebo-controlled trial of botulinum toxin B for sialorrhea in Parkinson's disease. Neurology. 2004 Jan 13;62(1):37-40. doi: 10.1212/01.wnl.0000101713.81253.4c. |
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DB: Double Blind (Part A) OL: Open Label (Part B)
The study was conducted at 33 sites including United States, Russia and Ukraine.
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| ID | Title | Description |
|---|---|---|
| FG000 | DB: MYOBLOC 2500 U | Participants will receive specified dose of MYOBLOC |
| FG001 | DB: MYOBLOC 3500 U | Participants will receive specified dose of MYOBLOC |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Double-blind Phase (Part A) |
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| PLACEBO | Other |
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| Los Angeles |
| California |
| 90033 |
| United States |
| National City | California | 91950 | United States |
| Aurora | Colorado | 80045 | United States |
| Washington D.C. | District of Columbia | 20007 | United States |
| Boca Raton | Florida | 33486 | United States |
| Port Charlotte | Florida | 33980 | United States |
| Carmel | Indiana | 46032 | United States |
| Baltimore | Maryland | 21287 | United States |
| Elkridge | Maryland | 21075 | United States |
| Detroit | Michigan | 48334 | United States |
| St Louis | Missouri | 63110 | United States |
| Edison | New Jersey | 08818 | United States |
| Albany | New York | 12208 | United States |
| New York | New York | 10003 | United States |
| Cincinnati | Ohio | 45219 | United States |
| Tulsa | Oklahoma | 74136 | United States |
| Port Royal | South Carolina | 29935 | United States |
| Cordova | Tennessee | 38018 | United States |
| Houston | Texas | 77030 | United States |
| San Antonio | Texas | 78229 | United States |
| Salt Lake City | Utah | 84132 | United States |
| Kirkland | Washington | 98034 | United States |
| Tacoma | Washington | 98409 | United States |
| Irkutsk | Irkutsk Oblast | 664079 | Russia |
| Vsevolozhsk | Leningradskaya Oblast' | 188643 | Russia |
| Saint Petersburg | Petrodvorets | 198510 | Russia |
| Krasnoyarsk | 660037 | Russia |
| Dnipropetrovsk | 49027 | Ukraine |
| Ivano-Frankivsk | 76008 | Ukraine |
| Kharkiv | 61068 | Ukraine |
| Lviv | 79010 | Ukraine |
| Rivne | 33010 | Ukraine |
| Uzhhorod | 88018 | Ukraine |
| 31930364 | Derived | Isaacson SH, Ondo W, Jackson CE, Trosch RM, Molho E, Pagan F, Lew M, Dashtipour K, Clinch T, Espay AJ; MYSTICOL Study Group. Safety and Efficacy of RimabotulinumtoxinB for Treatment of Sialorrhea in Adults: A Randomized Clinical Trial. JAMA Neurol. 2020 Apr 1;77(4):461-469. doi: 10.1001/jamaneurol.2019.4565. |
| FG002 | DB: Placebo | Participants will receive volume matched Placebo |
| FG003 | OL: ALL MYOBLOC | All Participants were to receive 3500 U MYOBLOC but could receive lower doses at investigator discretion. |
| COMPLETED |
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| NOT COMPLETED |
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| Open-label Phase (Part B) |
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Demographic Characteristics of intent-to-treat (ITT) Population.
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| ID | Title | Description |
|---|---|---|
| BG000 | MYOBLOC 2500 U | Subjects were dosed per treatment assignment of 2500 U MYOBLOC. |
| BG001 | MYOBLOC 3500 U | Subjects were dosed per treatment assignment of 3500 U MYOBLOC. |
| BG002 | PLACEBO | Placebo was dosed as an exact match of MYOBLOC. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Unstimulated Salivary Flow Rate (USFR) at Week 4 Post-injection Visit (Part A) | Change weight of expectorated saliva at a Week 4 post-injection visit. | The per protocol included subjects with a minimum USFR of 0.2 g/min. | Posted | Least Squares Mean | Standard Error | g/minute | 4 Weeks |
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| Primary | Clinical Global Impression Change (CGI-C) at Week 4 Post-injection (Part A) | CGI-C was assessed on a 7-point scale ranging from "very much improved" to "very much worse" with 1 assigned to "very much improved" and 7 assigned to "very much worse"; ranging from a minimum score of 1 and a maximum score of 7. | Per protocol the intent-to-treat population who were injected with study medication and had at least 1 post injection CGI-C measurement up to week 4 (inclusive). | Posted | Least Squares Mean | Standard Error | score on a scale | 4 weeks |
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Part A: 13-Week Post-Injection Period; Part B: 4 Sessions of 13-Week (+/- 2 Weeks) Post-Injection Periods
Part A (Session 1): Serious TEAEs Reported During Double-Blind Phase (Safety Population) Part B (Sessions 2-5): Serious TEAEs Reported During Open-Label Phase (OL Population)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | MYOBLOC 2500 U (Part A) | Participants received specified dose of MYOBLOC. Participants were evaluated for 13 weeks following the injection. | 0 | 63 | 1 | 63 | 44 | 63 |
| EG001 | MYOBLOC 3500 U (Part A) | Participants received specified dose of MYOBLOC. Participants were evaluated for 13 weeks following the injection. | 2 | 64 | 4 | 64 | 38 | 64 |
| EG002 | Placebo (Part A) | Participants received volume matched Placebo. Participants were evaluated for 13 weeks following the injection. | 1 | 60 | 4 | 60 | 16 | 60 |
| EG003 | OL: ALL MYOBLOC (Part B) | Participants received Open Label treatment with 3500 U (decreased dose at investigator discretion) every 13 weeks for a maximum of 4 treatment sessions. | 9 | 170 | 32 | 170 | 143 | 170 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardio-respiratory arrest | Cardiac disorders | Systematic Assessment |
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| Small intestinal obstruction | Gastrointestinal disorders | Systematic Assessment |
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| Malaise | General disorders | Systematic Assessment |
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| Pneumonia | Infections and infestations | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | Systematic Assessment |
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| Urosepsis | Infections and infestations | Systematic Assessment |
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| Brain cancer metastatic | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
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| Nodular melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
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| Syncope | Nervous system disorders | Systematic Assessment |
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| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Circulatory collapse | Vascular disorders | Systematic Assessment |
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| Atrial fibrillaiton | Cardiac disorders | Systematic Assessment |
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| Cardiac arrest | Cardiac disorders | Systematic Assessment |
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| Visual acuity reduced | Eye disorders | Systematic Assessment |
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| Dysphagia | Gastrointestinal disorders | Systematic Assessment |
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| Asthenia | General disorders | Systematic Assessment |
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| Fatigue | General disorders | Systematic Assessment |
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| Hypothermia | General disorders | Systematic Assessment |
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| Cellulitis | Infections and infestations | Systematic Assessment |
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| Fungal skin infection | Infections and infestations | Systematic Assessment |
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| Herpes zoster | Infections and infestations | Systematic Assessment |
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| Sepsis | Infections and infestations | Systematic Assessment |
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| Staphylococcal infection | Infections and infestations | Systematic Assessment |
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| Burns first degree | Injury, poisoning and procedural complications | Systematic Assessment |
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| Burns second degree | Injury, poisoning and procedural complications | Systematic Assessment |
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| Cervical vertebral fracture | Injury, poisoning and procedural complications | Systematic Assessment |
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| Facial bones fracture | Injury, poisoning and procedural complications | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | Systematic Assessment |
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| Hip fracture | Injury, poisoning and procedural complications | Systematic Assessment |
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| Lumbar vertebral fracture | Injury, poisoning and procedural complications | Systematic Assessment |
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| Subdural haematoma | Injury, poisoning and procedural complications | Systematic Assessment |
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| Malnutrition | Metabolism and nutrition disorders | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Lung neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
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| Pharyngeal cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
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| Amyotrophic lateral sclerosis | Nervous system disorders | Systematic Assessment |
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| Dementia with Lewy bodies | Nervous system disorders | Systematic Assessment |
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| Encephalopathy | Nervous system disorders | Systematic Assessment |
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| Ischaemic stroke | Nervous system disorders | Systematic Assessment |
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| Parkinson's disease | Nervous system disorders | Systematic Assessment |
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| Parkinsonism | Nervous system disorders | Systematic Assessment |
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| Radiculopathy | Nervous system disorders | Systematic Assessment |
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| Transient ischaemic attack | Nervous system disorders | Systematic Assessment |
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| Vocal cord paralysis | Nervous system disorders | Systematic Assessment |
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| Aggression | Psychiatric disorders | Systematic Assessment |
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| Aspiration | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Respiratory failure | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Arteriosclerosis | Vascular disorders | Systematic Assessment |
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| Deep vein thrombosis | Vascular disorders | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dental caries | Gastrointestinal disorders | Systematic Assessment |
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| Dry mouth | Gastrointestinal disorders | Systematic Assessment |
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| Dysphagia | Gastrointestinal disorders | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | Systematic Assessment |
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| Dizziness | Nervous system disorders | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | Systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | Systematic Assessment |
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| Weight decreased | Investigations | Systematic Assessment |
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Sponsor has the first right, within 9 months following the later to occur 1) close out meeting or 2) Sponsor possessing all data, to publish the lead paper. Following the foregoing, Institution has the right to publish the results provided the Institution provides a Publications Committee, which includes the Sponsor, with a draft at least 30 days before submission. At Sponsor's request, Institution shall withhold publication for 45 days to allow filing of a patent application.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Regulatory Affairs Associate | Solstice Neurosciences | regulatoryaffairs@usworldmeds.com |
| ID | Term |
|---|---|
| D012798 | Sialorrhea |
| D010300 | Parkinson Disease |
| D000690 | Amyotrophic Lateral Sclerosis |
| D020521 | Stroke |
| ID | Term |
|---|---|
| D012466 | Salivary Gland Diseases |
| D009059 | Mouth Diseases |
| D009057 | Stomatognathic Diseases |
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |
| D013118 | Spinal Cord Diseases |
| D016472 | Motor Neuron Disease |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D002561 | Cerebrovascular Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
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| ID | Term |
|---|---|
| C096323 | rimabotulinumtoxinB |
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| Between 18 and 65 years |
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| >=65 years |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Superiority |
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