Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial
In RCT (n=150), this oral co-inducer of cellular heat shock response did not improve efficacy outcomes vs placebo (change from baseline to month 20 in Inclusion Body Myositis Functional Rating Scale total score = –3.26 vs –2.26 placebo group; mean difference –0.99, p=0.12).
Source:
The Lancet Neurology
SPS commentary:
Inclusion body myositis is the most common progressive muscle wasting disease in people older than 50 years, with no effective drug treatment. The researchers highlight that this is one of the largest trials done in people with inclusion body myositis, providing data on disease progression that might be used for subsequent clinical trial design.
A commentary commends the researchers for conducting a large trial during the COVID-19 pandemic, but the results are clearly disappointing considering the unmet need for an effective treatment for this condition. It notes the preliminary studies and the power calculation were performed according to the standard procedures, but the absence of benefit as assessed by the primary endpoint suggests that different approaches to trial design, such as the selection of appropriate outcome measures, or different drug targets, might be needed. In the meantime, it stresses that management of patients with inclusion body myositis should follow existing standards of care.