What side effects are associated with this type of intervention?

Common treatments for Duchenne Muscular Dystrophy (DMD) that target dystrophin production or muscle function include eteplirsen, golodirsen, and glucocorticoids. Eteplirsen and golodirsen, both antisense oligonucleotides, can cause side effects such as balance disorder, vomiting, contact dermatitis, rash, and upper respiratory tract infections. Glucocorticoids, such as prednisone and deflazacort, are associated with weight gain, osteoporosis, increased risk of fractures, hypertension, and potential behavioral changes. These treatments aim to improve muscle function and slow disease progression but come with a range of potential adverse effects.

What prior FDA approvals exist?

Several drugs targeting dystrophin production have received FDA approval for the treatment of Duchenne Muscular Dystrophy (DMD). Eteplirsen, approved in September 2016, is designed for patients with a confirmed deletion of the DMD gene amenable to exon 51 skipping. Golodirsen, approved in December 2019, targets exon 53 skipping. Viltolarsen, approved for exon 53 skipping, showed increased dystrophin levels and functional improvements in clinical trials. Casimersen, approved in February 2021, targets exon 45 skipping. These drugs work by modifying the splicing of dystrophin pre-messenger RNA to increase dystrophin production in muscle cells.

What other types of research exist?

Promising novel alternatives for DMD treatment in 2024 include gene therapies targeting dystrophin production, such as exon-skipping therapies (e.g., eteplirsen, golodirsen), and CRISPR/Cas9-based gene editing. Additionally, therapies aimed at enhancing muscle function, such as utrophin upregulation and myostatin inhibitors, are also being explored. These approaches are in various stages of clinical development and hold potential for improving outcomes in DMD patients.

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EDG-5506 for Duchenne Muscular Dystrophy (LYNX Trial)

Phase 2

Recruiting

Research Sponsored by Edgewise Therapeutics, Inc.

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 24 months

Summary

This trial tests EDG-5506, a pill taken regularly, in children aged 4 to 9 with Duchenne muscular dystrophy. It aims to see if the medication is safe and can reduce muscle damage. The study includes both children who are and are not currently on corticosteroids.

Who is the study for?

This trial is for children with Duchenne muscular dystrophy who have a specific DMD gene mutation. They must be able to stand and climb stairs quickly, weigh between 15-35 kg, and either be aged 4-9 years on stable corticosteroids or aged 4-7 not on steroids recently.

What is being tested?

The LYNX study tests different doses of EDG-5506 against a placebo in two parts: one where neither the researchers nor participants know who's getting what (double-blind), followed by an open-label part where everyone knows which treatment is given.

What are the potential side effects?

While specific side effects are not listed here, they will monitor safety closely. This could include checking how the body processes the drug (pharmacokinetics) and changes in certain health markers.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Number of adverse events during treatment with sevasemten or placebo

Severity of adverse events during treatment with sevasemten or placebo

Secondary study objectives

Change from Baseline in fast skeletal muscle troponin I

Change from Baseline in serum creatinine kinase

Incidence of abnormal clinical chemistry test results

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