Santhera Pharmaceutical (Switzerland) Ltd SIDEROS(Efficacy, Safety of Idebenone in DMD PatiEnts Receiving GlucocOrticoid Steroids) clinical trial

This study is evaluating whether idebenone is safe and effective in treatingboys and men with Duchenne-associated respiratory impairment. Idebenone acts by reducing oxidative stress and improving mitochondrial respiratory chain function and energy production.This is the secondPhase III trial in which idebenone is being investigated as a potential treatment forrespiratory impairment related to DMD.

The primary objective is to evaluate the efficacy of oral idebenone compared to placebo in delaying the loss of respiratory function as measured by changes in the percent predicted Forced Vital Capacity (FVC) using spirometry.

Enrollment Criteria include:

  • Males with DMD confirmed by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein
  • 10 years of age or older
  • Forced Vital Capacity between 30% and 80% of predicted value
  • Corticosteroid use for at least 12 months prior to randomizationwithout any dose adjustments in the last 6 months
  • Immunization with 23-valent pneumococcal polysaccharide vaccine or any other pneumococcal polysaccharide vaccine as per national recommendations, as well as annually immunized with inactivated influenza vaccine

Full eligibility requirements for this study will be assessed at participating study sites. For more information on the enrollment criteria, please visit ClinicalTrials.gov(NCT#02814019).

If the patients qualify for the trial, they will be randomly assigned to one of two treatment groups: 1) those who receive idebenone, or 2) those who receive a matching placebo. Idebenone or the placebo is to be taken orally 3 times a day for 18 months.

A total of 266 boys with DMD taking glucocorticoid steroids will participate in the trial.

The SIDEROS trial will take place at various hospitals located within the United States and Europe (Austria, Belgium, Italy, France, Germany, Netherlands, Spain, Sweden, Switzerland, UK).

If the patients qualify for the trial, their participation will last approximately 21 months, and require approximately 10visits to the study site.

All patients completing the last visit, will be able to participate in an extension study (SIDEROS-E) and will receive idebenone until the SIDEROS-E is terminated or Marketing authorization is obtained for idebenone for DMD.

In addition, eligible participants will receivereimbursement for necessary travel expenses(air and train transportation, car rental, car service), hotel accommodations, meals and parking expenses, for the participant and one parent/caregiver, depending on the individual circumstances.

If you would like to participate or if you have questions, please click here to view the list of participating sitesand call or email the study coordinator at your preferred study site.

Thank you

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SNT-III-012 Information for PAG’s - V1.0 - 30-Nov-2016 (English)