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

NCT02610868 | Completed Phase 3 Results

• 1 other identifier: SN-SIAL-351
• Study Type: interventional
• Target: 187
• Locations: 3 countries
• Sites: 35

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.

Conditions

Sialorrhea

Keywords

Cerebral Palsy; ALS; Stroke; Neuroleptics

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (8)

• 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

• 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

• Clinical Global Impression of Change (CGI-C)

  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)

  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)

  Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13

Study Arms (1)

MYOBLOC Injection

EXPERIMENTAL

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

Interventions

MYOBLOC DRUG

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

Also known as: rimabotulinumtoxinB, botulinum toxin type B

MYOBLOC Injection

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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
• 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

Sponsors & Collaborators

• Solstice Neurosciences, LLC, a subsidiary of US WorldMeds, LLC: lead

Study Sites (35)

Unknown Facility

Phoenix, Arizona, 85013, United States

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Scottsdale, Arizona, 85258, United States

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Scottsdale, Arizona, 85259, United States

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Loma Linda, California, 92354, United States

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Los Angeles, California, 90033, United States

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Aurora, Colorado, 80045, United States

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Atlantis, Florida, 33462, United States

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Boca Raton, Florida, 33486, United States

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Orlando, Florida, 32806, United States

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Port Charlotte, Florida, 33980, United States

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Atlanta, Georgia, 30329, United States

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Kansas City, Kansas, 66160, United States

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Overland Park, Kansas, 66211, United States

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Elkridge, Maryland, 21075, United States

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Farmington Hills, Michigan, 48334, United States

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St Louis, Missouri, 63110, United States

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Las Vegas, Nevada, 89145, United States

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Edison, New Jersey, 08818, United States

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Centerville, Ohio, 45459, United States

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Tulsa, Oklahoma, 74136, United States

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Greenville, South Carolina, 29615, United States

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Cordova, Tennessee, 38018, United States

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San Antonio, Texas, 78229, United States

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Kirkland, Washington, 98034, United States

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Spokane, Washington, 99202, United States

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Tacoma, Washington, 98409, United States

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Grodno, 230017, Belarus

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Dnipropetrovsk, 49027, Ukraine

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Ivano-Frankivsk, 76008, Ukraine

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Kharkiv, 61068, Ukraine

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Lviv, 79010, Ukraine

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Odesa, 65025, Ukraine

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Rivne, 33010, Ukraine

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Uzhhorod, 88018, Ukraine

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Zaporizhzhya, 69600, Ukraine

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Related Publications (1)

• 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.

  PMID: 40498248 DERIVED

MeSH Terms

Conditions

Sialorrhea; Cerebral Palsy; Stroke

Interventions

rimabotulinumtoxinB

Condition Hierarchy (Ancestors)

Salivary Gland Diseases; Mouth Diseases; Stomatognathic Diseases; Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases

Results Point of Contact

• Title: Najeebah Abdul-Musawir, MD, Director of Clinical Research
• Organization: Supernus Pharmaceuticals, Inc.

nabdulmusawir@supernus.com

2404035571

Study Officials

• Jonathan Rubin, PhD

  Supernus Pharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 2, 2015
• First Posted: November 20, 2015
• Study Start: October 1, 2015
• Primary Completion: June 1, 2017
• Study Completion: June 1, 2017
• Last Updated: April 21, 2026
• Results First Posted: November 4, 2019

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (26); BE (1); UA (8)