SIDEROS: Phase 3 study of Idebenone in people with Duchenne

Phase 3 Study Assessing the Efficacy, Safety and Tolerability of the Investigational drug Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids (SIDEROS)

Questions and Answers

What stage is this research?

The SIDEROS clinical trial is a Phase 3 clinical trial.

What is the goal or purpose of this study?

The primary objective of this study is to assess the efficacy and safety of the investigational drug idebenone in slowing the loss of pulmonary function in boys with Duchenne receiving glucocorticoid steroids.

What is idebenone and how does it work?

Idebenone is thought to have a dual effect in Duchenne – mitochondrial activation and protection. Idebenone is thought to increase the energy output of the cells’ mitochondria – the parts (“factories”) of the cell that generate all of a cells’ energy. Mitochondria produce the energy necessary for the cell functioning through a process called “cellular respiration” which requires oxygen and produces energy. During cellular respiration, some toxic forms of oxygen (called oxygen free radicals) can be produced. These free radicals must be neutralized by other substances to avoid cellular damage. Idebenone is expected to act as a neutralizer of these toxic forms of oxygen. Thus, idebenone is expected to have an antioxidant effect, and consequently prevent cellular damage. Idebenone is optimized to dissolve in water and lipids and able to cross the mitochondrial membrane.

Who is funding this study?

This study is funded by Santhera Pharmaceuticals.

Who may be eligible to participate in this study?

To participate in this study, you must be male with Duchenne, at least 10 years old and have stable use of glucocorticoid steroids continuously for at least 12 months with no dose adjustments for the past six months, with the exception of weight changes. This includes prednisone or deflazacort, and any dosing regimens. Changes between prednisone and deflazacort are allowed if dose is comparable.

Participants must have a baseline Forced Vital Capacity between 35%-80% and be able to provide reliable and reproducible pulmonary function testing.

What do I have to do if I decide to participate in this study?

This study involves a screening assessment, and participants will be randomized to receive either 900 mg/daily of idebenone (2 tablets, 3 times daily with food) or placebo for 18 months. Parents will be asked to keep a daily diary of medication intake.

Participants will complete safety assessments, pulse-oximetry, and pulmonary function tests at study site visits every three months, as well as be given a hand-held spirometry device to complete weekly pulmonary function assessments at home.

Funding will be provided to assist families with costs to participate in the study and travel to and from the study site.

How long will this study last, and will I have access to the drug/treatment once the study has ended?

Patients’ active participation is up to 21-22 months (88-92 weeks) including screening and follow-up visits, and 18 months receiving study medication or placebo.

Patients completing the study through week 78 will be eligible to participate in an open-label extension study and will continue to receive medication until the trial is terminated or idebenone is approved.

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