Update on stoppage of SIDEROS clinical trial for Raxone/idebenone

In October we published news that Santhera, the company developing Raxone/idebenone (a potential treatment for delaying the loss of respiratory function in patients with Duchenne muscular dystrophy) was ending its development programme for this drug.

This was due to the results from an interim analysis of a clinical trial called SIDEROS, which led to the stopping of the trial.

The SIDEROS study aimed to analyse the effectiveness of Raxone/idebenone compared to placebo in delaying the loss of respiratory function for Duchenne muscular dystrophy patients receiving glucocorticoid steroids. The interim analysis revealed the clinical trial was unlikely to meet its objectives.

This summary document has been produced by patient groups working in partnership with the manufacturer Santhera to explain more about the trial, what its goal outcomes were, and why it was stopped.


Our helpline is available on 0800 652 6352 or at info@musculardystrophyuk.org.