We are pleased to provide you with detailed information from Santhera Pharmaceuticals about an ongoing clinical trial that your son may be able to participate in.
Beyond investing in early scientific research to accelerate a pathway to a cure for Duchenne, CureDuchenne is committed to providing the Duchenne community with education, help and hope. We take a holistic approach for all members of the Duchenne community and are committed to providing current, comprehensive and credible information and resources. Santhera Pharmaceuticals has allowed us to deliver this important trial information. Below are details:
Santhera Pharmaceuticals (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 treating boys and men with Duchenne-associated respiratory impairment. Idebenone is thought to act by reducing oxidative stress and improving mitochondrial respiratory chain function and energy production. This is the second Phase III trial in which idebenone is being investigated as a potential treatment for respiratory 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 (there is no restriction on upper age limit)
- Forced Vital Capacity between 35% and 80% of predicted value and being in the respiratory function decline phase according to the investigator’s opinion
- Corticosteroid use for at least 12 months prior to randomization without 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 flu vaccine
Full eligibility requirements for this study will be assessed at participating study sites.
If a boy or man is eligible to participant in 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. Their participation will last approximately 21 months, and require approximately 10 visits to the study site.
All participants completing SIDEROS and found eligible to continue on drug will be able to participate in an extension study (SIDEROS-E). Those who participate in the extension study will receive Idebenone until idebenone is commercially available for patients included in this study or SIDEROS-E is terminated by Santhera.
In addition, eligible participants will receive reimbursement for 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.
A total of 266 males with DMD taking glucocorticoid steroids will participate in the trial.
The SIDEROS trial will take place at various sites located within the United States, Europe (Austria, Belgium, Bulgaria, France, Germany, Hungary, Ireland, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom and Israel.
If you would like to participate or if you have questions, please visit: www.SiderosDMD.com or ClinicalTrials.gov (NCT#02814019), contact your neuromuscular physician, or reach out to email@example.com.
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