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Antisense Oligonucleotide

Casimersen + Golodirsen for Duchenne Muscular Dystrophy (ESSENCE Trial)

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

Must not have

Previous treatment with SMT C1100 within 1 week prior to Week 1 and previous treatment with PRO045 (BMN 045), PRO053 (BMN 053), or PRO051 (BMN 051) within 24 weeks prior to Week 1

Major surgery within 3 months prior to Week 1

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 96, week 144

Awards & highlights

Pivotal 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.

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been treated with SMT C1100 recently and PRO045, PRO053, or PRO051 in the last 6 months.

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I have not had major surgery in the last 3 months.

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I have never undergone gene therapy.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

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

Participant's Ability to Rise Independently From the Floor, as indicated by a North Star Ambulatory Assessment (NSAA) Subscore

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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