Orphazyme sponsors IBM trial at University College London

Danish biotech company, Orphazyme, has assumed sponsorship of a phase 2/3 trial assessing the safety and efficacy of arimoclomol for the treatment of sporadic inclusion body myositis (sIBM).

The trial has sites at the University of Kansas Medical Center, USA, and the Institute of Neurology at University College London (UCL). The UCL site is expected to begin recruitment soon; we will let you know when this starts.

MDUK’s Chair, Professor Mike Hanna, is the principal investigator at the UCL trial site. He said in a press release:

Arimoclomol has strong potential benefit in patients with this disabling disease. We made the case quite strongly that arimoclomol should be properly tested in a large trial, and it is most exciting that we have now started the next step in the clinical development.

The exact cause of sIBM is not fully understood. Some research suggests that it is partly caused by certain proteins being folded incorrectly. These misfolded proteins accumulate in clumps inside the muscle, causing inflammation and muscle damage.

Arimoclomol is an experimental drug that enhances the cells’ ability to re-fold proteins. This could help to clear the toxic clumps in muscles affected by sIBM. You can read more about arimoclomol on Orphazyme’s website.


Further information

Find out more about the trial on clinicaltrials.gov

Read our news story about the previous arimoclomal study

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Our Clinical Trials Information Service gives you the latest news related to research and clinical trials. For more information, visit our online resources, or get in touch with the Research team or research@musculardystrophyuk.org.