Arimoclomol for Inclusion Body Myositis

• Research type

  Research Study

• Full title

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

• IRAS ID

  13764

• Sponsor organisation

  University College London

• Eudract number

  2008-008173-62

• ISRCTN Number

  n/a

• Research summary

  Sporadic Inclusion Body Myositis (IBM) is the commonest acquired disease of muscle affecting people aged 50 years and over. It is a progressive and debilitating disease which causes both muscle weakness and wasting, characteristically of the quadriceps and finger flexors. Over time it can lead to severe disability, falls and swallowing impairment. Affected muscle tissue demonstrates inflammation and degeneration which are represented respectively by white cell invasion of the muscle and abnormal deposition of proteins within cells.Previous IBM trials have assessed numerous immunosuppressive agents and none has demonstrated significant clinical efficacy, even where the degree of muscle inflammation was reduced. We propose a novel therapeutic strategy which seeks to target more of the responsible disease processes than purely inflammation.Arimoclomol is a compound which acts by enhancing a normal, inbuilt protective cell reaction to stresses, termed 'The Heat Shock Response'. The products of this response are 'Heat Shock Proteins' which counteract processes leading to abnormal protein deposition and also damage by inflammation.This project proposes to assess the safety and tolerability of Arimoclomol in 12 patients with Sporadic IBM, taken as three 100mg oral capsules per day for a period of four months. We also seek to demonstrate evidence that Arimoclomol has its anticipated effect to augment levels of key HSPs in affected muscle. These effects will be compared with those of a superficially identical but inactive placebo capsule which 4 of the 12 participants will be randomly allocated. Study investigators will be Ó?blinded? of this participants?? allocation to Arimoclomol or placebo. Recruitment will take place at the National Hospital for Neurology and Neurosurgery.

• REC name

  London - Harrow Research Ethics Committee

• REC reference

  09/H0714/22

• Date of REC Opinion

  6 Apr 2009

• REC opinion

  Favourable Opinion