Vamorolone clinical trial recruiting in Australia

A clinical trial of a promising new drug called 'vamorolone' (also known as VBP15) has started in Australia. Vamorolone is a 'designer steroid' with the potential to cause fewer side effects and greater benefits than other steroids which are currently commonly used in the treatment of children and young adults with Duchenne.

While corticosteroids (such as prednisolone and deflazacort) have been proven to help maintain muscle strength in Duchenne, their side-effects are a major barrier to their long-term use. These side-effects include weight gain, mood changes (irritability and hyperactivity) and puffiness in the face; as well as sleeping difficulties, headaches, stomach irritation, growth suppression, increased blood pressure, cataracts, bone fractures and an increased susceptibility to infection.

The Melbourne site (Royal Children's Hospital) of the Phase 2a clinical trial of vamorolone will recruit its first patients in the coming months with a trial site in Sydney (Westmead Children’s Hospital) due to open soon. The trial, which is also running in the US, Canada, Israel, Sweden and in the UK, will recruit around 48 children worldwide aged four through six years old who have not previously taken corticosteroids.

The boys will take vamorolone at one of four different doses for two weeks followed by a two-week follow-up period. They will then be eligible to join an extension trial to continue to receive the drug for up to two years. The trial primarily aims to assess the safety and tolerability of vamorolone and work out the best dose for further trials which will look more closely at its effectiveness.

The Phase 2b trial of vamorolone is expected to start later this year, and will enrol about 100 Duchenne boys at about 30 international sites. This study will be placebo controlled and will also involve comparison to traditional corticosteroids.

Save Our Sons Duchenne Foundation funded important preclinical work on this drug and worked with its developer – US-based ReveraGen – to bring the trial to Australia. They also fund the Clinical Trials Nurse in Melbourne who will be coordinating the Vamolorone study.

Further information

More details about the trial are available on clinicaltrials.gov [https://clinicaltrials.gov/ct2/show/NCT02760264]

Contact the DuchenneConnect Australia Coordinators:
Phone: +61 (03) 8592 6431
Email:
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