The SIDEROS study: evaluating DMD - associated respiratory impairment.

SIDEROS is a phase III double-blind, randomized, placebo-controlled study assessing the efficacy, safety and tolerability of idebenone in patients with Duchenne muscular dystrophy receiving glucocorticoids. We regret to inform the Duchenne community, that based on the results of the interim analysis and recommendation from the trial’s Data and Safety Monitoring Board (DSMB), the SIDEROS trial has been deemed futile. Based on the results of the interim analysis, we have made the difficult decision to discontinue SIDEROS and the development of idebenone in Duchenne muscular dystrophy.

About idebenone

Mitochondria are specialized structures in the human body that serve as batteries, powering various functions of the cell and the organism as a whole... (1, 2)

View about Idebenone

Respiratory Function in DMD

Duchenne Muscular Dystrophy (DMD) is the most common and devastating type of muscular dystrophy and is characterized by a complete loss of functional dystrophin, leading to progressive muscle weakness and wasting. DMD is a rare disease that affects mostly males born worldwide (3,4).

In boys and men with DMD, weakness of respiratory muscles leads to a progressive decline in their ability to move air into/out of their lungs, leading to sleep disturbances and respiratory infections, especially when patients have lost their ability to walk…

View Respiratory function in DMD
  • Inclusion criteria include:


    Males with DMD confirmed by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein on a muscle biopsy (5).

  • Inclusion criteria include:


    10 years of age or older (there is no upper age limit).

  • Inclusion criteria include:


    Forced Vital Capacity (6, 7) between 35% and 80% of predicted value and being in the respiratory function decline phase according to the investigator’s opinion.

  • Inclusion criteria include:


    Corticosteroid use for at least 12 months prior to randomization without any dose adjustments in the last 6 months. Patients may be on any dosing regimen of deflazacort/calcort or prednisone/prednisolone.

  • Inclusion criteria include:


    Pneumonia immunization and annually immunized with inactivated influenza vaccine.

SIDEROS trial overview

SIDEROS is a phase III clinical trial, evaluating the efficacy of investigational drug idebenone compared to placebo, in delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids.

View Sideros Trial Overview

Trial locations

The SIDEROS trial will take place at various sites located within the United States, Europe and Israel

View Trial Locations
02 Eu-Map-English

Trial endpoints

Respiratory function will be assessed by a qualified and certified evaluator in each site. This is typically a respiratory therapist or physiotherapist. The participants will be instructed on the use of the site-based spirometer and a handheld nmd-1 device...

View trial endpoints