MYSTICOL: A multisite, double-blind, randomized, placebo-controlled trial of rimabotulinumtoxinB for the treatment of sialorrhea in Parkinson’s disease (PD) and other neurological conditions (P3.026)

Objective: To determine the efficacy, safety and tolerability of rimabotulinumtoxinB (Myobloc) in the treatment of sialorrhea Background: Sialorrhea, or excessive drooling, can be a major morbidity in Parkinson’s disease (PD) and other neurological disorders. Current oral and topical treatments are suboptimal. Botulinum toxins reduce saliva production by inhibiting acetylcholine release, when injected into the submandibular (SM) and parotid (PA) glands. Design/Methods: Subjects who met inclusion criteria of troublesome sialorrhea (including patients with PD, ALS, stroke, cancer) were assigned to three injection treatment arms: rimabotulinumtoxinB (Myobloc) 2500U total (250u to each SM, 1000u to each PA), 3500U total (250u to each SM, 1500u to each PA), or placebo vehicle injection using anatomical landmarks with or without ultrasound confirmation. Subjects were evaluated at 4, 8, and 13 weeks post-injection. Co-primary endpoints were: Unstimulated Salivary Flow Rate (USFR) and Clinical Global Impression of Change (CGI-C) at Week 4 post-injection compared to baseline. Results: A total of 187 subjects were enrolled. USFR for both rimabotulinumtoxinB (Myobloc) treatment groups significantly improved at week 4 versus placebo. Significant improvement was observed in USFR week 1 -8 post-injection in the 2500u and 3500u treatment groups, and through week 13 in the higher dose group. There was also a significant difference observed in CGI-C at week 4 for both treatment groups versus placebo, and at week 1- 8 post-injection, and in the low dose group at week 13. The most common adverse effect was dry mouth. Conclusions: Injection of rimabotulinumtoxinB (Myobloc) into the parotid and submandibular glands wase effective for the treatment of sialorrhea in patients with PD and other neurologic disorders, atf both 2500U and 3500U treatment arms. Injections were generally well tolerated Study Supported by: US WorldMeds Disclosure: Dr. Isaacson has nothing to disclose. Dr. Jackson has received personal compensation for activities with GLG, Guidepoint Global, Marathon Pharmaceuticals, OneWorld Meds and Cytokinetics as a consultant. Dr. Jackson has received research support from OneWorld Meds and Cytokinetics. Dr. Molho has received personal compensation for activities with Lundbeck Research USA, Inc. and US WorldMeds as a consult and/or speaker. Dr. Trosch has received personal compensation for activities with Ipsen as a speaker and consultant. Dr. Ondo has received personal compensation for activities with TEVA, UCB Pharma, Lundbeck Research USA, Inc, ACADIA, and IMPAX as speaker and consultant. Dr. Clinch has received personal compensation for activities with US WorldMeds as an employee.