The results of the first clinical trial of vamorolone (which ended earlier this year) in people living with Duchenne were published last week in Pharmalogical Research – a peer reviewed journal. The results have already been announced in a press release, but publication in a peer-reviewed journal means that the results have been inspected in detail by independent researchers.  

Vamorolone was developed as a potential alternative to steroids – with the hope that it would have reduced side effects and it is currently in phase 3 trial in people living with Duchenne.

The phase 2a trial, published here, was carried out at the CINRG sites in the US, Canada, Sweden, Israel, UK and Australia. Twelve boys with Duchenne aged 4-7 took part, and received 4 different doses of vamorolone for 2 weeks, followed by 2 further weeks of monitoring. After completing the trial the participants could take part in a 6 month extension.

The trial demonstrated that there were no serious safety concerns with the potential drug, although some minor side effects were noted. Although the trial did not aim to determine how effective vamorolone was, a reduction in immune system activity and a reduction in the amount of creatine kinase in the blood (a measure of muscle damage) was seen, which is encouraging.

Publication of the results in a peer-reviewed journal is a key part of the scientific and clinical trials process. As well as giving independent researchers the chance to scrutinise the data, publication also means that researchers around the world can study the trial data, which could help advance their work.

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