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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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

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.

Detailed 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. 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.
Sponsor: US WorldMeds LLC

Current Primary Outcome: Adverse Events [ Time Frame: through study completion, up to 1 year ]

Adverse events are recorded at each study visits of both Parts A and B, starting from the first injection of MYOBLOC or placebo on Day 1 of Treatment Session 1 and ending at study completion. The occurrence, seriousness, severity, and causality assessment of adverse events will be assessed through evaluation of vital signs, physical exam, pulmonary function by spirometry, clinical laboratory tests, and neurological exams.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Adverse Events of Special Interest [ Time Frame: through study completion, up to 1 year ]
    Adverse events indicative of compromised airway protection as manifested by aspiration, aspiration pneumonia, choking, and dysphagia will be closely evaluated as adverse events of special interest (AESI) at each study visit and at discontinuation of the study.
  • Columbia Suicide Rating Scale (C-SSRS) [ Time Frame: through study completion, up to 1 year ]
    The C-SSRS measures both suicidal ideation and suicidal behavior and will be completed on Day 1 (before the first injection of study drug) and each subsequent clinic visit or, if applicable at discontinuation from the study.
  • Occurrence of Dental Adverse Events [ Time Frame: through study completion, up to 1 year ]
    Dental and oral health will be followed closely throughout the course of the study, with exams performed by either a licensed dentist (DDS or DMD) at each site, or oral examination by the Investigator. Dental adverse events include anything related to oral and dental health.
  • Unstimulated Salivary Flow Rate (USFR) [ Time Frame: through study completion, up to 1 year ]
    Salivary flow rate, will be determined in an unstimulated state (i.e., at least 1 hour postprandial) at screening, just before the first injection of study drug (Day 1) to establish a baseline salivary flow rate, and at each subsequent study visit.
  • Clinical Global Impression of Severity (CGI-S) and Change (CGI-C) [ Time Frame: through study completion, up to 1 year ]
    CGI-S will be used to record the severity of illness (sialorrhea) on a 7-point scale ranging from "normal" to "among the most extremely ill patients".
  • Patient Global Impression of Severity (PGI-S) and Change (PGI-C) [ Time Frame: through study completion, up to 1 year ]
    The PGI-S scale will be used by the subject to rate the severity of his/her illness (sialorrhea) on an 8-point scale ranging from "normal" to "among the most extremely ill".
  • Drooling Frequency and Severity Scale (DFSS) [ Time Frame: through study completion, up to 1 year ]
    The DFSS will be used to assess the frequency and severity of drooling on a 5-point scale ranging from 1 (dry, never drools) to 5 (profuse, clothing, hands, tray, objects become wet). Drooling frequency will be assessed on a 4-point scale ranging from 1 (never drools) to 4 (constantly drools).
  • Drooling Impact Score (DIS) [ Time Frame: through study completion, up to 1 year ]
    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).


Original Secondary Outcome: Same as current

Information By: US WorldMeds LLC

Dates:
Date Received: November 2, 2015
Date Started: October 2015
Date Completion: July 2017
Last Updated: May 1, 2017
Last Verified: May 2017