The SIDEROS study: evaluating DMD-associated respiratory impairment.

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.

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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...

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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 patients have lost their ability to walk…

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  • 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 35% and 80% of predicted value and being in the respiratory function decline phase according to the investigator’s opinion.

  • Inclusion criteria include:

    4

    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:

    5

    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…

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Trial locations

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

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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 participating patients will be instructed on the use of the site-based spirometer and a handheld nmd-1 device...

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