Efficacy and Safety Study of MYOBLOC® Followed by Open-Label Multiple-Treatment With MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adult Subjects (MYSTICOL)

• ClinicalTrials.gov Identifier: NCT01994109
• Recruitment Status: Completed
• First Posted: November 25, 2013
• Results First Posted: August 15, 2019
• Last Update Posted: July 13, 2021
• Sponsor: Supernus Pharmaceuticals, Inc.
• Information provided by (Responsible Party): Supernus Pharmaceuticals, Inc.

Tracking Information

• First Submitted Date: November 14, 2013
• First Posted Date: November 25, 2013
• Results First Submitted Date: May 2, 2019
• Results First Posted Date: August 15, 2019
• Last Update Posted Date: July 13, 2021
• Study Start Date: November 2013
• Actual Primary Completion Date: January 2016 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 14, 2019)

• Unstimulated Salivary Flow Rate (USFR) at Week 4 Post-injection Visit (Part A) [ Time Frame: 4 Weeks ]
  Change weight of expectorated saliva at a Week 4 post-injection visit.
• Clinical Global Impression Change (CGI-C) at Week 4 Post-injection (Part A) [ Time Frame: 4 weeks ]
  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.

• Original Primary Outcome Measures (submitted: November 20, 2013): Unstimulated Salivary Flow Rate [ Time Frame: 4 Weeks ]
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures (submitted: November 20, 2013): Clinical Global Impression Change (CGI-C) [ Time Frame: 4 weeks ]
• Current Other Pre-specified Outcome Measures: Not Provided

Original Other Pre-specified Outcome Measures (submitted: November 20, 2013)

Number of Subjects with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 4 weeks ]

Adverse events and adverse events of special interest will be assessed at each study visit. Adverse events may be assessed by subject report, review of symptoms, vital signs, laboratory values, physical examination, neurological examination, dental examination and the review of the Columbia Suicide Severity Rating Scale (C-SSRS)

Descriptive Information

• Brief Title: Efficacy and Safety Study of MYOBLOC® Followed by Open-Label Multiple-Treatment With MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adult Subjects
• Official Title: A Phase 3, Multicenter, Double-Blind, Placebo-Controlled, Single-Treatment Efficacy and Safety Study of MYOBLOC® (Part A) Followed by Open-Label, Multiple-Treatment With MYOBLOC® (Part B) in the Treatment of Troublesome Sialorrhea in Adult Subjects
• Brief Summary: 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Sialorrhea

Intervention

• Drug: MYOBLOC
  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.
  Other Name: rimabotulinumtoxinB, botulinum toxin type B
• Other: PLACEBO
  Other Name: volume-matched placebo

Study Arms

• Active Comparator: MYOBLOC 2500 U
  Subjects will receive specified dose of MYOBLOC
  Intervention: Drug: MYOBLOC
• Active Comparator: MYOBLOC 3500 U
  Subjects will receive specified dose of MYOBLOC
  Intervention: Drug: MYOBLOC
• Placebo Comparator: Placebo
  Subjects will receive volume matched Placebo
  Intervention: Other: PLACEBO

Publications *

• 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.
• 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.
• 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 14, 2019): 187
• Original Estimated Enrollment (submitted: November 20, 2013): 180
• Actual Study Completion Date: January 2017
• Actual Primary Completion Date: January 2016 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Seeking treatment for troublesome sialorrhea for at least 3 months that is occurring secondary to any disorder or related to any cause
• Investigator sites will review entire list of inclusion criteria with potential subjects

Exclusion Criteria:

• Any known prior exposure to botulinum toxin type B, or known adverse reaction or sensitivity to botulinum toxin type A, or known sensitivity to any of the MYOBLOC solution components.
• Prior botulinum toxin treatment to the salivary glands at any time
• Investigator sites will review entire list of exclusion criteria with potential subjects

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 85 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Russian Federation, Ukraine, United States

Administrative Information

• NCT Number: NCT01994109
• Other Study ID Numbers: SN-SIAL-301
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Supernus Pharmaceuticals, Inc.
• Original Responsible Party: USWM, LLC (dba US WorldMeds)
• Current Study Sponsor: Supernus Pharmaceuticals, Inc.
• Original Study Sponsor: USWM, LLC (dba US WorldMeds)
• Collaborators: Not Provided

Investigators

• Study Director: Najeebah Abdul-Musawir, MD,MBA; Supernus Pharmaceuticals, Inc.

• PRS Account: Supernus Pharmaceuticals, Inc.
• Verification Date: August 2019