P.02Phase 2/3 study of Arimoclomol in sporadic inclusion body myositis: study design

Abstract

Sporadic inclusion body myositis (IBM) is the most common idiopathic inflammatory myopathy occurring in patients over the age of 45 years. IBM muscle displays both inflammatory and degenerative features, yet immune suppression has proven to be ineffective. Modulating the cytoprotective "heat shock response" (HSR) represents a therapeutic strategy targeting both inflammation and degeneration. Arimoclomol is an orally administered pharmacological agent that can up-regulate the HSR by amplifying heat shock protein expression. In a pilot Phase 2a study Arimoclomol was safe and well tolerated and demonstrated a preliminary signal for potential therapeutic benefit in patients with IBM. These data supported further research of Arimoclomol in IBM. To evaluate the efficacy in a phase 2/3 randomized controlled trial of Arimoclomol in IBM and to gather data on safety and tolerability. We present herein the study protocol of NCT02753530. In this double-blind, placebo-controlled, multicenter, international study, 150 subjects are being randomizing 1:1 to receive orally three times a day either Arimoclomol 400 mg or matching placebo capsules for 20 months. The primary outcome measure is the IBM functional rating scale (IBMFRS) at Month 20. Secondary outcome measures include other efficacy measures, namely manual muscle testing (MMT), dynamometry of the quadriceps and grip, Health Assessment Questionnaire (HAQ-DI) and Short-Form 36 (SF-36). Safety and tolerability will also be evaluated. MRI of the lower extremities is an exploratory outcome measure. This is an ongoing study. We anticipate the last subject will be enrolled by April of 2019. We present herein this pivotal study design and anticipate being able to share results in 2021.