Various studies looking at the impact of steroids, are now being published. They show that, overall, children with Duchenne who are treated with steroids, stay walking for longer than those who are not treated with steroids. The effect is variable from child to child, and the children who respond well to steroids, also seem to develop other complications less frequently. They develop breathing difficulties later, and have fewer problems with their spines. There may also be a positive effect on the heart muscle.

Prednisolone and Deflazacort are the steroids most often prescribed for Duchenne.

However, there can be important side effects, the worst of which are significant growth delay, cushingoid (puffy face), delayed puberty and a weakening of the bones. There is ongoing research to establish the most effective dose and regimens for steroids, to enable clinicians to minimise the side effects and maximise the benefits.

Potential alternatives to steroids

ReveraGen Biopharma, a US pharmaceutical company, is developing a new steroidal drug called Vamorolone (previously VBP15) which has the potential to better balance the benefit and side effects than the more traditional steroids. A successful Phase 1 study in adult volunteers was funded by Duchenne Children’s Trust, Joining Jack and the MDA (USA).

As of September 2016 ReveraGen Biopharma has begun recruiting patients for its Phase 2a open label study of Vamorolone. The trial will be recruiting patients who haven’t yet started steroids, between the ages of 4-7, and is recruiting in the UK, United States, Canada, Israel, Sweden and Australia.

For more information please visit: http://vision-dmd.info/2a-trial-information/

CAT-1004 is being developed by the US company Catabasis. In animal models, the drug has shown to reduce muscle degeneration and improve muscle regeneration and function. The effects were seen across skeletal, cardiac and diaphragm muscle. CAT-1004 inhibits NF-ĸB, a protein that is activated in Duchenne as well as in other skeletal muscle disorders. CAT-1004 is not a steroid.

CAT-1004 is currently being tested on children with Duchenne who are not on steroids and are aged between 4-7 years in the phase 2 DMD-Move trial.

Taking part in a steroid alternative trial

All trials looking at steroid alternative treatments, will require participants to be “steroid naive”. This means they will not yet have started taking traditional steroids. These trials will most likely be recruiting younger patients.

Clinical stage

ReveraGen Biopharma

Vamorolone: extension study

An initial 2-week study to assess the safety of Vamorolone in boys with DMD led to an extension study, which is currently active.

Vamorolone: long-term extension study

  • The steroid alternative is designed to treat patients with any DMD mutation.
  • The study is enrolling by invitation (participants who have taken part in the previous Vamorolone extension study). The study is enrolling 48 males between ages 4 and 7.
  • The 24-month phase 2 trial will take place across multiple sites, including some in the UK.

Catabasis

CAT-1004 (Edasalonexent): 3-part phase 1/2 study

  • Parts A and B are complete. CAT-1004 was generally well tolerated with no safety signals.
  • Part C: patients will receive CAT-1004 for 112 weeks in an open label study.
  • This study recruited 31 participants between ages 4 and 7.
  • This study is taking place across 5 sites in the USA.

POLARIS DMD Trial. Phase 3 global trial for edasalonexent is currently being planned. Recruitment for this trial is not yet open. This trial is intended to support an application for commercial licensing of edasolonexent as a treatment for DMD.