arimoclomol-ibm

A Randomised, Double-blinded, Placebo-controlled Pilot Study Assessing the Safety and Tolerability of Arimoclomol in Adult Patients with Sporadic Inclusion Body Myositis

Background

sIBM is the commonest acquired disease of muscle affecting people aged 50 years and over. It is a debilitating disease with progressive muscle weakness and wasting. It affects muscles mostly in the thigh and fingers.

Over time the condition can lead to severe disability, falls and swallowing problems. Affected muscles show inflammation and degeneration.

Arimoclomol is a new drug. It enhances a normal, inbuilt protective cell reaction to stresses. The products of this response are Heat Shock Proteins (HSPs).

HSPs counteract processes that lead to abnormal protein deposition and to damage by inflammation.

Trial information

This was a multi-centre, double-blind, placebo-controlled parallel study lasting twelve weeks. 

Primary objective

To assess the safety and tolerability of Arimoclomol in IBM patients.

Recruitment took place at the National Hospital for Neurology and Neurosurgery.

12 patients participated in the study.