Open-label Study of Arimoclomol in Inclusion Body Myositis (IBM)
Research type
Research Study
Full title
An open-label, non-randomized trial to investigate the efficacy and safety of early versus delayed start of arimoclomol in patients with sporadic inclusion body myositis who have completed the IBM4809 trial
IRAS ID
266012
Contact name
Michael Hanna
Contact email
Sponsor organisation
Orphazyme A/S
Eudract number
2019-000749-11
Clinicaltrials.gov Identifier
076773, IND NUMBER
Duration of Study in the UK
2 years, 2 months, 30 days
Research summary
Inclusion Body Myositis (IBM) is a chronic disorder in which muscles become inflamed and can cause muscle weakening. Weakness comes on slowly (over months or years) and progresses steadily and usually leads to severe weakness and wasting of arm and leg muscles. Patients may become unable to perform activities of daily living and
most require assistive devices within 5 to 10 years of symptom onset.The cause of IBM is unknown but there is a new evidence which suggests that the pathology of IBM results from cellular changes caused by a variety of stressful events and diseases. In response to these stressful events the body’s normal response is to increase the levels of Heat Shock Proteins (HSP) to help counteract and stop these cellular changes. In people with IBM this increase does not appear sufficient to reverse these toxic cellular changes.
We know that arimoclomol causes people’s bodies to make more of the HSP protein. By increasing HSP levels in IBM patients we hope to reverse the toxic cellular changes that might be responsible for the pathology of IBM.
The IBM4809 trial was designed to determine the efficacy arimoclomol compared to placebo, in patients with IBM. The purpose of this study is to further evaluate the safety and efficacy of arimoclomol, in paitents that have completed the IBM4809 trail. In this trial, on completion of the IBM4809 trial, subjects will be invited to receive open-label arimoclomol at the same daily dosage for a further 20 months. This design allows the comparison of efficacy and safety of arimoclomol while affording continued access to arimoclomol in the absence of approved effective treatments for IBM.
REC name
London - Fulham Research Ethics Committee
REC reference
19/LO/1182
Date of REC Opinion
9 Oct 2019
REC opinion
Further Information Favourable Opinion