Efficacy and safety of onabotulinumtoxinA with standardized occupational therapy for treatment of pediatric upper limb spasticity: Phase III placebo-controlled randomized trial.

Abstract

BACKGROUND\nThis is the first large study of onabotulinumtoxinA as treatment for pediatric upper limb spasticity.\n\n\nOBJECTIVE\nEvaluate efficacy and safety of a single treatment with onabotulinumtoxinA plus occupational therapy (OT).\n\n\nMETHODS\nIn this registrational phase 3, multinational study (NCT01603602), participants were randomized 1:1:1 to onabotulinumtoxinA 3\u200aU/kg/OT, 6\u200aU/kg/OT, or placebo/OT. Primary endpoint was average change from baseline at weeks 4 and 6 in Modified Ashworth Scale-Bohannon (MAS) score. Secondary endpoints included Modified Tardieu Scale (MTS), Clinical Global Impression of Change (CGI) and functional Goal Attainment Scale (GAS).\n\n\nRESULTS\n235 participants were randomized. At weeks 4 and 6, onabotulinumtoxinA groups had greater mean reductions in MAS (both -1.9; p\u200a< \u200a0.001) versus placebo (-1.2). OnabotulinumtoxinA doses improved dynamic tone per MTS. Mean CGI at weeks 4 and 6 was unchanged in the overall population, but improved in a post hoc analysis of patients with a single affected UL muscle group (elbow or wrist). GAS score for passive goals was significantly higher for 6\u200aU/kg versus placebo at week 12. Most AEs were mild/moderate in severity; overall incidence was similar between groups.\n\n\nCONCLUSIONS\nOnabotulinumtoxinA (3 and 6\u200aU/kg) was safe and effective in reducing upper limb spasticity in pediatric participants.