What side effects are associated with this type of intervention?

Common treatments for Duchenne Muscular Dystrophy that involve exon-skipping therapy, such as eteplirsen and golodirsen, have been associated with several side effects. The most frequently reported adverse reactions include balance disorder, vomiting, contact dermatitis, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection. Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria, have also been observed. These treatments aim to increase dystrophin production in muscle tissue, which is crucial for managing DMD symptoms.

What prior FDA approvals exist?

Several exon-skipping therapies have received FDA approval for the treatment of Duchenne Muscular Dystrophy (DMD). Eteplirsen, approved for patients with mutations amenable to exon 51 skipping, increases dystrophin production and has shown benefits in walking distance and muscle function. Golodirsen, approved for exon 53 skipping, also increases dystrophin levels. Viltolarsen, another exon 53 skipping therapy, has demonstrated increased dystrophin production and functional improvements. Casimersen, targeting exon 45 skipping, was approved based on its ability to increase dystrophin production. These therapies aim to produce a functional dystrophin protein, similar to the approach being studied with DYNE-251.

What other types of research exist?

Promising alternatives to DYNE-251 for DMD patients include: 1) Ataluren, which targets nonsense mutations in the dystrophin gene and has shown some efficacy in clinical trials, though it is not approved in the US. 2) Eteplirsen, an exon 51 skipping therapy, which has shown benefits in maintaining forced vital capacity in nonambulatory patients. 3) Golodirsen, another exon-skipping therapy targeting exon 53, approved by the FDA based on increased dystrophin production. 4) Gene therapy approaches using microdystrophin or minidystrophin genes delivered via adeno-associated viral vectors, though these are still in early clinical stages.

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Exon Skipping Agent

DYNE-251 for Duchenne Muscular Dystrophy (DELIVER Trial)

Phase 1 & 2

Recruiting

Research Sponsored by Dyne Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, up to week 241

Summary

This trial tests DYNE-251, an IV drug, in people with Duchenne muscular dystrophy who can benefit from exon 51 skipping. The drug aims to help their muscles by increasing the dystrophin protein. It works by skipping a faulty part of their gene to produce more of this important protein. Eteplirsen (Exondys 51) was the first drug approved for exon 51 skipping in Duchenne muscular dystrophy.

Who is the study for?

Boys aged 4-16 with Duchenne muscular dystrophy (DMD) that can be treated by skipping exon 51, able to undergo muscle biopsy. They must have been on stable glucocorticoids for at least 12 weeks and have a healthy heart function. Non-ambulatory participants should not have lost mobility for more than two years.

What is being tested?

The trial is testing DYNE-251's effects on safety, tolerability, and muscle protein levels in DMD patients compared to a placebo over multiple stages: an initial controlled phase of 24 weeks, followed by an open-label period of the same duration and then a long-term extension for up to 96 weeks.

What are the potential side effects?

While specific side effects are not listed here, common concerns may include reactions at the infusion site, potential kidney or liver issues due to medication processing, allergic reactions, or general symptoms like fatigue or fever.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, up to week 241

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, up to week 241

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, up to week 241 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 North Star Ambulatory Assessment (NSAA) Total Score in Ambulatory Participants up to Week 241

Change From Baseline in Performance Upper Limb (PUL) Scale Version 2.0 Score up to Week 241

Trial Design

3Treatment groups

Experimental Treatment

Group I: Placebo-Controlled MAD Period - PlaceboExperimental Treatment1 Intervention

Placebo will be administered Q4W or Q8W over 24 weeks.

Group II: Placebo-Controlled MAD Period - DYNE-251Experimental Treatment1 Intervention

DYNE-251 will be administered once every 4 weeks (Q4W) or once every 8 weeks (Q8W) over 24 weeks.

Group III: Open-Label and Long-Term Extension Period - DYNE-251Experimental Treatment1 Intervention

DYNE-251 will be administered Q4W or Q8W for up to 192 weeks after participants complete the Placebo-Controlled MAD Period of the study.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Placebo

1995

Completed Phase 3

~2670

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Exon-skipping therapies, such as eteplirsen, golodirsen, and the investigational DYNE-251, are designed to treat Duchenne Muscular Dystrophy (DMD) by increasing the production of dystrophin, a protein essential for muscle function. These treatments use antisense oligonucleotides to skip over specific exons in the DMD gene, enabling the production of a shorter but functional dystrophin protein. This mechanism is crucial for DMD patients as it targets the root cause of the disease, potentially slowing its progression and improving muscle strength and function.