Eteplirsen + Golodirsen + Casimersen for Duchenne Muscular Dystrophy
(EVOLVE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to observe the long-term outcomes of three treatments—eteplirsen, golodirsen, and casimersen, all exon-skipping therapies—in people with Duchenne Muscular Dystrophy (DMD). The goal is to gather data on how these treatments work in everyday settings, outside of tightly controlled clinical studies. It suits those with a confirmed diagnosis of DMD who are already using or planning to start one of these treatments. Importantly, participants should not be part of other DMD treatment studies.
As a Phase 4 trial, this study focuses on treatments already FDA-approved and proven effective, helping to understand how they benefit more patients.
Do I have to stop taking my current medications for this trial?
The trial information does not specify if you need to stop taking your current medications. It seems focused on observing outcomes for those already prescribed eteplirsen, golodirsen, or casimersen, so you may not need to stop other medications, but it's best to confirm with the study team.
What is the safety track record for eteplirsen, golodirsen, and casimersen?
Previous studies have shown golodirsen to be safe. It was generally well-tolerated, with only mild, non-serious side effects reported over up to six years, and no significant risk of interaction with other medications. Research on casimersen found it to be well-tolerated as well, with most side effects being mild and not directly caused by the treatment. However, animal studies suggested possible kidney issues, so monitoring kidney function is important. Eteplirsen has also been well-tolerated. Studies showed no one stopped treatment due to side effects, and there were no serious side effects like kidney problems. Most reported side effects were mild. All these treatments have FDA approval for treating Duchenne Muscular Dystrophy, indicating a good safety record in humans.12345
Why are researchers enthusiastic about this study treatment?
Researchers are excited about Eteplirsen, Golodirsen, and Casimersen because they offer a novel approach to treating Duchenne muscular dystrophy (DMD). Unlike traditional treatments that primarily focus on managing symptoms, these drugs employ exon skipping, a cutting-edge technique that targets specific genetic mutations to produce functional dystrophin protein. This approach has the potential to slow disease progression more effectively than current options. By directly addressing the underlying genetic cause of DMD, these treatments represent a significant shift in how we approach this condition.
What is the effectiveness track record for eteplirsen, golodirsen, and casimersen in treating Duchenne Muscular Dystrophy?
This trial will compare the effectiveness of three treatments for Duchenne Muscular Dystrophy (DMD): eteplirsen, golodirsen, and casimersen. Research has shown that eteplirsen can slow the progression of DMD, helping patients live longer and improving heart function over time. Golodirsen promotes exon skipping, which helps correct the DMD gene and may slow the disease's progression. Casimersen has been shown to increase levels of dystrophin, a protein that protects muscle cells, which is important for managing DMD symptoms. All three treatments have received approval for use, demonstrating their effectiveness in treating certain types of DMD. Participants in this trial will be assigned to one of these treatment arms to evaluate their specific effects.36789
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Observational
Participants' medical history data and prospective data on DMD treatment outcomes are collected
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Casimersen
- Eteplirsen
- Golodirsen
How Is the Trial Designed?
3
Treatment groups
Experimental Treatment
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sarepta Therapeutics, Inc.
Lead Sponsor
Citations
Casimersen (AMONDYS 45™): An Antisense Oligonucleotide ...
It was approved by the Food and Drug Administration (FDA) in February 2021 to treat Duchenne muscular dystrophy (DMD) in patients whose DMD gene ...
2.
mdaconference.org
mdaconference.org/abstract-library/casimersen-in-patients-with-duchenne-muscular-dystrophy-amenable-to-exon-45-skipping-interim-results-from-the-phase-3-essence-trial/Interim Results From the Phase 3 ESSENCE Trial
Western blot analysis showed significantly increased mean dystrophin levels from baseline after 48 weeks of casimersen (0.93% vs 1.74% of normal; P<0.001) and ...
NCT02500381 | Study of SRP-4045 (Casimersen) and ...
The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD ...
4.
medicalpolicy.bcbstx.com
medicalpolicy.bcbstx.com/content/dam/bcbs/medicalpolicy/pdf/rx/RX501.135_2024-02-01.pdfCasimersen
In the absence of clinical data convincingly demonstrating a clinical effect, it cannot be concluded that the amount of dystrophin expressed ...
P.132 Casimersen in patients with Duchenne muscular ...
Western blot analysis showed significantly increased mean dystrophin levels from baseline after 48 weeks of casimersen (0.93% vs 1.74% of normal; P<0.001) and ...
AMONDYS 45 (casimersen) Label - accessdata.fda.gov
Kidney Toxicity: Based on animal data, may cause kidney toxicity. Kidney function should be monitored; creatinine may not be a reliable measure of renal ...
Safety, tolerability, and pharmacokinetics of casimersen in ...
Casimersen was well tolerated in participants with DMD amenable to exon 45 skipping. Most TEAEs were mild, nonserious, and unrelated to casimersen.
8.
medicalpolicy.bcbstx.com
medicalpolicy.bcbstx.com/content/dam/bcbs/medicalpolicy/pdf/rx/RX501.135_2025-01-01.pdfCasimersen
approved by the U. S. Food and Drug Administration (FDA) when it is prescribed for a use recognized as safe and effective for the treatment of a ...
9.
parentprojectmd.org
parentprojectmd.org/sarepta-therapeutics-announces-fda-acceptance-of-casimersen-srp-4045-nda-for-patients-with-dy-amenable-to-skipping-exon-45/Sarepta Announces FDA Acceptance of Casimersen NDA ...
Safety is being assessed through the collection of adverse events, laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital ...
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