A Phase III Double-blind Randomized Placebo-Controlled Study assessing the Efficacy Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids (SIDEROS)
Investigating the Effects of an Investigational Medication on Breathing in Duchenne Muscular Dystrophy (DMD) Patients
Brief description of study.
In this study, idebenone will be tested in order to determine the effect it has on breathing on DMD patients receiving Glucocorticoid Steroids compared to placebo (a pill with no drug in it) and to see how well idebenone is tolerated over an 18 months treatment period.
Detailed description of study
In this study, idebenone will be tested in order to determine the effect it has on breathing on DMD patients receiving Glucocorticoid Steroids compared to placebo (a pill with no drug in it) and to see how well idebenone is tolerated over an 18 months treatment period.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Duchenne muscular dystrophy
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Age: 100 years or below
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Gender: All
This study investigates the effects of an investigational medication on breathing in patients with Duchenne Muscular Dystrophy (DMD) who are also receiving glucocorticoid steroids. Duchenne Muscular Dystrophy is a genetic disorder characterized by progressive muscle degeneration and weakness. The purpose of this study is to compare the investigational medication with a placebo, which is a pill that looks like the investigational medication but contains no active ingredients, to see its effects on breathing and its tolerance over time.
Participants in this study will be randomly assigned to receive either the investigational medication or a placebo. The study will involve regular assessments of breathing function to determine the effects of the medication. Participants will continue their usual treatment with glucocorticoid steroids while participating in the study.
- Who can participate: The study is open to patients diagnosed with Duchenne Muscular Dystrophy who are currently receiving glucocorticoid steroids. Participants must be between X and Y years old and meet specific health criteria to ensure safety.
- Study details: Participants will take either the investigational medication or a placebo, which is an inactive substance that looks like the investigational medication but does not contain any medicine. Breathing function will be regularly assessed to monitor the effects of the treatment.
- Study Timelines: The study will last 18 months.
Interested in the study?
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