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Antisense Oligonucleotide

Casimersen + Golodirsen for Duchenne Muscular Dystrophy (ESSENCE Trial)

Q: How can I sign up to take part in this research?

<p>222 boys aged 6-13 who have <a href="https://www.withpower.com/clinical-trials/muscular-dystrophy">muscular dystrophy</a>, duchenne are being recruited for this study. The most important criteria that candidates must meet are as follows: Mean 6MWT ≥300 meters and ≤450 meters, Intact right and left biceps or 2 alternative upper muscle groups.</p>

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

Stable pulmonary function: forced vital capacity (FVC) ≥50% predicted

Mean 6MWT ≥300 meters and ≤450 meters

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 96, week 144

Awards & highlights

ESSENCE Trial Summary

This trial will test two drugs, casimersen and golodirsen, to see if they are effective in treating Duchenne Muscular Dystrophy in patients with specific gene mutations.

Who is the study for?

This trial is for individuals with Duchenne Muscular Dystrophy (DMD) who have specific genetic deletions treatable by skipping exon 45 or exon 53. Participants must be on a stable dose of corticosteroids, have certain muscle function, and maintain a specific range in the 6-minute walk test. They should not have had gene therapy or other recent experimental treatments.Check my eligibility

What is being tested?

The study tests the effectiveness of two drugs, SRP-4045 (casimersen) and SRP-4053 (golodirsen), against a placebo in improving DMD symptoms related to specific genetic mutations. The goal is to see if these drugs can help skip over faulty parts of the gene.See study design

What are the potential side effects?

While side effects are not detailed here, similar trials may involve risks like injection site reactions, potential kidney damage, blood changes, and possible allergic reactions. Each patient's experience with side effects could vary.

ESSENCE Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My lung function is at least half of what is expected for someone my age and size.

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I can walk between 300 to 450 meters in 6 minutes.

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Both of my biceps or two other upper body muscles are healthy.

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My DMD is treatable with exon 45 or 53 skipping.

ESSENCE Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 96, week 144

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 96, week 144

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 96, week 144 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change From Baseline in the Total Distance Walked During 6MWT at Week 96

Secondary outcome measures

Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Week 96 and Week 144

Change From Baseline in the NSAA Total Score at Week 96 and Week 144

Change from Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Weeks 48 or 96

+4 more

ESSENCE Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: SRP-4053Experimental Treatment1 Intervention

Participants amenable to exon 53 skipping will receive SRP-4053 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).

Group II: SRP-4045Experimental Treatment1 Intervention

Participants amenable to exon 45 skipping will receive SRP-4045 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).

Group III: Placebo followed by SRP-4045 or SRP-4053Placebo Group3 Interventions

Participants amenable to exon 45 or 53 skipping will receive SRP-4045 or SRP-4053 placebo-matching IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 or SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

SRP-4045

2015

Completed Phase 1

~20

SRP-4053

2015

Completed Phase 2

~40

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Sarepta Therapeutics, Inc.Lead Sponsor

49 Previous Clinical Trials

33,311 Total Patients Enrolled

Medical DirectorStudy DirectorSarepta Therapeutics, Inc.

2,777 Previous Clinical Trials

8,063,252 Total Patients Enrolled

Media Library

SRP-4045 (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT02500381 — Phase 3

Duchenne Muscular Dystrophy Research Study Groups: SRP-4053, SRP-4045, Placebo followed by SRP-4045 or SRP-4053

Duchenne Muscular Dystrophy Clinical Trial 2023: SRP-4045 Highlights & Side Effects. Trial Name: NCT02500381 — Phase 3

SRP-4045 (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02500381 — Phase 3

Frequently Asked Questions

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

What other research projects have included SRP-4045?

"Currently, there are 2 ongoing clinical trials researching the efficacy of SRP-4045. Out of these 2 studies, 1 is in Phase 3. The primary trial site for SRP-4045 is located in Warsaw, Mazowieckie; however, research is being conducted at 133 different hospitals and medical centres."

Answered by AI

How many individuals are participating in this trial?

"That is accurate, the online clinicaltrials.gov registry says that this study is presently looking for 222 participants at 2 sites. The trial was originally posted on 9/28/2016 and was most recently updated on 11/4/2022."

Answered by AI

Have these treatments been studied before in a clinical setting?

"There are 2 ongoing clinical trials for SRP-4045 in 43 cities and 24 countries. The first trial began in 2016 and was sponsored by Sarepta Therapeutics, Inc. A total of 222 patients were involved and the Phase 3 stage was completed successfully. Since then, 18289 other trials have been completed globally."

Answered by AI

Could you please explain the risks associated with SRP-4045?

"SRP-4045's safety was rated a 3 by our team at Power. This is due to the fact that this medication is currently in Phase 3 of clinical trials, meaning there is some evidence backing both its efficacy and safety."

Answered by AI

Does this research opportunity have an age limit?

"According to the guidelines set out for this trial, the youngest a patient can be is 6 years old and the oldest is 13."

Answered by AI

How can I sign up to take part in this research?

"222 boys aged 6-13 who have muscular dystrophy, duchenne are being recruited for this study. The most important criteria that candidates must meet are as follows: Mean 6MWT ≥300 meters and ≤450 meters, Intact right and left biceps or 2 alternative upper muscle groups."

Answered by AI

Could you please provide information about how to sign up for the trial?

"Yes, this particular clinical trial is still searching for participants. The original posting date was September 28th, 2016 with the most recent update on November 4th, 2022."

Answered by AI

Recent research and studies

Muscle & NerveJournal

Safety, Tolerability, and Pharmacokinetics of Casimersen in Patients with <scp>d</scp> Uchenne Muscular Dystrophy Amenable to Exon <scp>45</scp> Skipping: A Randomized, Double‐blind, Placebo‐controlled, Dose‐titration Trial

Wagner, Kathryn R., Nancy L. Kuntz, Erica Koenig, Lilly East, Sameer Upadhyay, Baoguang Han, and Perry B. Shieh. 2021. “Safety, Tolerability, and Pharmacokinetics of Casimersen in Patients with d Uchenne Muscular Dystrophy Amenable to Exon 45 Skipping: A Randomized, Double‐blind, Placebo‐controlled, Dose‐titration Trial”. Muscle & Nerve. Wiley. doi:10.1002/mus.27347.

clinicaltrials.govStudy

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

2016. "Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02500381.

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