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Casimersen for Duchenne Muscular Dystrophy
Study Summary
This trial is testing if two drugs are safe and tolerated by patients with Duchenne muscular dystrophy when taken long-term.
- Duchenne Muscular Dystrophy
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
Frequently Asked Questions
Does this experiment only use elderly individuals as participants?
"According to the study's inclusion criteria, eligible patients must be between 7 and 23 years old. Out of the total number of trials, 63 are for patients below 18 while 50 are for those over 65."
Is this clinical trial limited to North America or are there other participating continents?
"28 clinical trial sites are participating in this research, with 3 of them being Rady Children's Hospital- San Diego, Neuromuscular Research Center, and Ann and Robert H Lurie Childrens Hospital of Chicago."
Is this a full-fledged clinical trial or are there still opportunities for patients to enroll?
"This study is not currently looking for new recruits, as the last update was on August 3rd, 2022. If you are interested in other similar studies, there are 88 clinical trials for muscular dystrophies and 2 studies for Casimersen that are still looking for patients."
Has this particular clinical trial been done before?
"There have been 2 global clinical trials for Casimersen completed, with the first study starting in 2016. In total, these 222 patient trials have occurred across 41 cities and 22 countries. The 2016 study completed Phase 3 drug approval and was sponsored by Sarepta Therapeutics, Inc."
What is the government's opinion of Casimersen?
"There is some evidence that Casimersen is effective, as this is a Phase 3 trial. Additionally, there is data from multiple rounds of testing that supports Casimersen's safety, so it received a score of 3."
Who qualifies to participate in this research?
"The aim of this study is to enroll 260 participants that have been diagnosed with muscular dystrophy and are between the ages of 7 and 23. The most vital inclusion criterion is that patients must be 7-23 years old."
Could you please share what other researchers have looked into in regards to Casimersen?
"The first clinical trials testing the efficacy of casimersen were conducted in 2016 at John Radcliffe Hospital. Since then, there have been a total of 4 trials, 2 of which are ongoing. Currently, most of the active research is happening in San Diego, California."
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