ReveraGen BioPharma have begun enrolment in the Phase 2a clinical trial of vamoroleone in the treatment of young people living with Duchenne muscular dystrophy, which Action Duchenne part-funded in the pre-clinical stage.

The Phase 2a trials are part of the VISION-DMD project, a steroid-like treatment. Vamorolone is developed as an alternative to traditional glucocorticoids for DMD.

In the trial, 48 steroid-naïve boys aged 4-7 will receive vamorolone once daily for 2 weeks followed by 2 weeks of follow-up off treatment. As part of the 2a trial extension, the boys will receive the same dose level for 24 weeks.

Enrolment will take place at sites of the Cooperative International Neuromuscular Research Group (CINRG) network within the United States, Canada, Israel, United Kingdom, Sweden and Australia.

The development of vamorolone as an alternative to traditional glucocorticoids for DMD is currently funded through a Venture Philanthropy model including an international community of patient groups and US and European public grants.

The Phase 2b trials are expected to begin in the second half of 2017 and will be part funded by a grant from the European Commission Horizon 2020 programme.

Preclinical funding was provided by DMD foundations and US government: Foundation to Eradicate Duchenne (USA); Parent Project Muscular Dystrophy (USA); Muscular Dystrophy Association (USA); Action Duchenne (UK), Save Our Sons (Aus); Michael’s Cause (USA), Pietro’s Fight (USA), Alex’s Wish (UK), Ryan’s Quest (USA); and US public grants from National Institutes of Health; and Department of Defense CDMRP.

For more information please contact iain@actionduchenne.org