Efficacy and Tolerability of Rizatriptan for Treatment of a Single Migraine Attack in Pediatric Migraineurs: Results from a Randomized, Double-Blind, Placebo-Controlled Trial Using a Novel Enrichment Design

Objective: To evaluate the efficacy and tolerability of rizatriptan versus placebo in pediatric migraineurs. Background Acute migraine treatment options for children are limited because it has been difficult to demonstrate efficacy over placebo in trials. Design/Methods: Children, ages 6-17yrs, who had not, historically, achieved satisfactory response to treatment with NSAIDs/acetaminophen, were enrolled in a novel design utilizing a single-blind run-in phase, followed by randomization into a double-blind, placebo-controlled phase with weight-based rizatriptan dosing (5-mg for Results: 702 patients 12-17yrs of age treated, with 570 evaluable for efficacy. Rizatriptan demonstrated a significantly higher response rate compared to placebo for pain-freedom at 2hrs in 12-17yr-olds: 87/284 (30.6%) versus 63/286 (22.0%), odds ratio=1.55 [95% CI: 1.06, 2.26], p-value=0.025. The incidences of adverse events within 14-days postdose in 12-17yr-olds were similar between rizatriptan and placebo: 81/337 (24.0%) versus 83/365 (22.7%). 977 patients 6-17yrs of age treated during the study, with 770 evaluable for efficacy; the pattern of findings was similar to 12-17yr-olds. Conclusions: Rizatriptan was statistically significantly more effective than placebo in eliminating pain and generally well-tolerated in pediatric migraineurs. Supported by: Merck & Co., Inc. Disclosure: Dr. Ho has received personal compensation for activities with Merck & Co., Inc. as an employee.Dr. Ho holds stock and/or stock options in Merck & Co., Inc. Dr. Pearlman has received personal compensation for activities with GSK, Merck, Allergan, and Nautilus Pharmaceuticals as a consultant.Dr. Pearlman has received research support from GSK, Merck, and AstraZenenca. Dr. Lewis has received personal compensation for activities with Merck, Astra Zeneca, and GlaxoSmithKline.Dr. Lewis has received research support from Abbott, Astra Zeneca, Almirall, GlaxoSmithKline, Merck, and Ortho-McNeil. Dr. Hamalainen has received personal compensation for activities with Merck. Ms. Connor has received personal compensation for activities with Merck & Co., Inc. Ms. Connor holds stock and/or stock options in Merck & Co., Inc. Dr. Michelson has nothing to disclose. Dr. Zhang has received personal compensation for activities with Merck & Co., Inc. Dr. Harper-Mozley has received personal compensation for activities with Merck as an employee.Dr. Harper-Mozley holds stock and/or stock options in Merck. Ms. Strickler has received personal compensation for activities with Merck & Co., Inc., as an employee. Ms. Strickler holds stock and/or stock options in Merck & Co., Inc. Mr. Bachman has received personal compensation for activities with Merck & Co., Inc. as an employee. Mr. Bachman holds stock and/or stock options in Merck & Co., Inc. Dr. Mahoney has received personal compensation for activities with Merck & Co, Inc. as an employee. Dr. Mahoney holds stock and/or stock options in Merck & Co, Inc. Dr. Lines has received personal compensation for activities with Merck as an employee.Dr. Lines holds stock and/or stock options in Merck. Dr. Hewitt has received personal compensation for activities with Merck & Co., Inc. as an employee.