DMD research


VISION-DMD is an EC funded project undertaking Phase 2 clinical studies on vamorolone, a new treatment for Duchenne Muscular Dystrophy (DMD). The project was launched in January 2016 and aims to advance the clinical development of vamorolone (also known as VBP15) a dissociative steroid designed to have the efficacy of a steroid, without the severe associated side effects.

The Phase 2a study completed recruitment in November 2017. The purpose of this study was to test how safe and tolerated vamorolone was in boys with Duchenne, and how the acts drug in the body.

This study is an open label, multiple ascending dose study of vamorolone on 48 steroid naïve boys aged from 4 years old to less than 7 years old. Boys were enrolled in four dose cohorts sequentially – 0.25 mg/kg, 0.75 mg/kg, 2.0 mg/kg and 6.0 mg/kg vamorolone, dosing once a day for 2 weeks followed by 2 weeks of follow up. The primary outcome of this trial is acute safety, tolerability and pharmacokinetics of vamorolone administration. The Phase 2a study database lock was achieved in February 2018. Results of this study will be published imminently.

The Phase 2a extension study is ongoing with the participants from the 2a study, at the same dose level for 24 weeks. The primary outcome of the 2a extension trial is long term safety, tolerability, efficacy as measured by the time to stand test and safety as measured by body mass index. Database lock for the 2a extension study is expected in June 2018. Following this study patients have the option of entering a long term extension study.
Twelve trial sites in seven countries are participating in the 2a studies including the USA, Canada, the UK, Israel, Sweden, and Australia.

The first Phase 2b study site is expected to open for recruitment in April 2018 in the USA. Phase 2b trial sites are expected in the USA, Canada, Belgium, Czech Republic, Germany, Italy, the Netherlands, Sweden, Israel, the UK and Australia

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Together with Joining Jack, Duchenne UK has invested $800,000 into the phase 1 trial.

Published on 4 April 2018

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