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Antisense Oligonucleotide

Casimersen for Duchenne Muscular Dystrophy

Phase 3
Waitlist Available
Research Sponsored by Sarepta Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Other inclusion/exclusion criteria apply.
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 30 days after the last infusion of study drug (assessed up to 148 weeks)
Awards & highlights

Study Summary

This trial is testing if two drugs are safe and tolerated by patients with Duchenne muscular dystrophy when taken long-term.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
There are additional requirements to participate in this study that have not been listed here.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 30 days after the last infusion of study drug (assessed up to 148 weeks)
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 30 days after the last infusion of study drug (assessed up to 148 weeks) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Patients With Serious Adverse Events (SAEs)

Trial Design

2Treatment groups
Experimental Treatment
Group I: GolodirsenExperimental Treatment1 Intervention
Patients amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen will receive open-label golodirsen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks.
Group II: CasimersenExperimental Treatment1 Intervention
Patients amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen will receive open-label casimersen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks.

Find a Location

Who is running the clinical trial?

Sarepta Therapeutics, Inc.Lead Sponsor
49 Previous Clinical Trials
23,234 Total Patients Enrolled
Medical DirectorStudy DirectorSarepta Therapeutics, Inc.
2,769 Previous Clinical Trials
8,062,029 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI
~26 spots leftby Mar 2025