The SIDEROS study: addressing DMD-associated respiratory impairment.

The SIDEROS study 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 trial overview

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...

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 males born worldwide.

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 boys have lost their ability to walk…

View Respiratory function in DMD
  • Inclusion criteria include:

    1

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

  • Inclusion criteria include:

    2

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

  • Inclusion criteria include:

    3

    Forced Vital Capacity between 30% and 80% of predicted value.

  • Inclusion criteria include:

    4

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

  • Inclusion criteria include:

    5

    Pneumonia immunization and annually immunized with inactivated influenza vaccine.

SIDEROS trial overview

The SIDEROS study 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 hospitals located within the United States, Europe and Israel...

View Trial Locations
USA_en@300x
Europe_en@300x
World_en@300x

Trial endpoints

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

View trial endpoints