Open-Label and Single-Arm Study of MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adults (OPTIMYST)

A Phase 3, Long-Term, Open-Label and Single-Arm Study of MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adult Subjects

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.

Study Overview

• Status: Completed
• Conditions: Sialorrhea
• Intervention / Treatment: Drug: MYOBLOC

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 Type: Interventional
• Enrollment (Actual): 187
• Phase: Phase 3

Contacts and Locations

Study Locations

• Belarus
  • Grodno, Belarus, 230017
• Ukraine
  • Dnipropetrovsk, Ukraine, 49027
  • Ivano-Frankivsk, Ukraine, 76008
  • Kharkiv, Ukraine, 61068
  • Lviv, Ukraine, 79010
  • Odesa, Ukraine, 65025
  • Rivne, Ukraine, 33010
  • Uzhhorod, Ukraine, 88018
  • Zaporizhzhya, Ukraine, 69600
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85013
    • Scottsdale, Arizona, United States, 85258
    • Scottsdale, Arizona, United States, 85259
  • California
    • Loma Linda, California, United States, 92354
    • Los Angeles, California, United States, 90033
  • Colorado
    • Aurora, Colorado, United States, 80045
  • Florida
    • Atlantis, Florida, United States, 33462
    • Boca Raton, Florida, United States, 33486
    • Orlando, Florida, United States, 32806
    • Port Charlotte, Florida, United States, 33980
  • Georgia
    • Atlanta, Georgia, United States, 30329
  • Kansas
    • Kansas City, Kansas, United States, 66160
    • Overland Park, Kansas, United States, 66211
  • Maryland
    • Elkridge, Maryland, United States, 21075
  • Michigan
    • Farmington Hills, Michigan, United States, 48334
  • Missouri
    • St Louis, Missouri, United States, 63110
  • Nevada
    • Las Vegas, Nevada, United States, 89145
  • New Jersey
    • Edison, New Jersey, United States, 08818
  • Ohio
    • Centerville, Ohio, United States, 45459
  • Oklahoma
    • Tulsa, Oklahoma, United States, 74136
  • South Carolina
    • Greenville, South Carolina, United States, 29615
  • Tennessee
    • Cordova, Tennessee, United States, 38018
  • Texas
    • San Antonio, Texas, United States, 78229
  • Washington
    • Kirkland, Washington, United States, 98034
    • Spokane, Washington, United States, 99202
    • Tacoma, Washington, United States, 98409

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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: rimabotulinumtoxinB; botulinum toxin type B

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence, Seriousness, Severity, and Causality Assessment of Treatment Emergent Adverse Events (TEAE)	TEAEs Related to Study Medication include TEAEs classified as Possibly, Probably and Definitely Related. Treatment Session (TS)	Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1
Occurrence of Adverse Events of Special Interest (AESI)	Treatment Session (TS) AESI includes: Aspiration, Aspirational pneumonia, Choking and Dysphagia.	Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1
Columbia Suicide Rating Scale (C-SSRS): Suicidal Ideation, Behavior Scores and Intensity Scores	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).	Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13
Occurrence of Dental Adverse Events	Treatment Session (TS)	Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Unstimulated Salivary Flow Rate (USFR): Treatment Sessions 1-4	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.	Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13
Change From Baseline in Clinical Global Impression of Severity (CGI-S)	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.	Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13
Clinical Global Impression of Change (CGI-C)	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)	TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13
Change From Baseline in Drooling Frequency and Severity Scale Performed by Trained Assessor (DFSS-I)	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.	Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13
Change From Baseline in Drooling Frequency and Severity Scale Performed by Subject (DFSS-S)	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.	Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13
Change From Baseline in Patient Global Impression of Severity (PGI-S)	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.	Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13
Patient Global Impression of Change (PGI-C)	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.	TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13
Change From Baseline in Drooling Impact Score (DIS)	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.	Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13

Collaborators and Investigators

• Sponsor: Solstice Neurosciences, LLC, a subsidiary of US WorldMeds, LLC
• Investigators: Study Director: Jonathan Rubin, PhD, Supernus Pharmaceuticals, Inc.

Publications and helpful links

General Publications

• Pahwa R, Molho E, Lew M, Dashtipour K, Gil RA, Revilla FJ, Clinch T, Qin P, Isaacson SH; OPen Label Trial of Intraglandular MYobloc injections for Sialorrhea Treatment (OPTIMYST) study group. Long-Term Safety and Efficacy of Repeated Cycles of RimabotulinumtoxinB in the Treatment of Chronic Sialorrhea: Results of the OPTIMYST Trial. Neurol Ther. 2025 Aug;14(4):1553-1567. doi: 10.1007/s40120-025-00777-z. Epub 2025 Jun 11.

Study record dates

Study Major Dates

• Study Start: October 1, 2015
• Primary Completion (Actual): June 1, 2017
• Study Completion (Actual): June 1, 2017

Study Registration Dates

• First Submitted: November 2, 2015
• First Submitted That Met QC Criteria: November 19, 2015
• First Posted (Estimated): November 20, 2015

Study Record Updates

• Last Update Posted (Actual): April 21, 2026
• Last Update Submitted That Met QC Criteria: March 31, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Cerebral Palsy; Stroke; ALS; Neuroleptics
• Additional Relevant MeSH Terms: Mouth Diseases; Stomatognathic Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases; Salivary Gland Diseases; Brain Damage, Chronic; Stroke; Cerebral Palsy; Sialorrhea; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Cholinergic Agents; Membrane Transport Modulators; Acetylcholine Release Inhibitors; Anti-Dyskinesia Agents; rimabotulinumtoxinB
• Other Study ID Numbers: SN-SIAL-351

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO