What side effects are associated with this type of intervention?

Common treatments for Becker Muscular Dystrophy (BMD) that aim to protect and improve muscle function, such as EDG-5506, often have side effects similar to those seen in treatments for Duchenne Muscular Dystrophy (DMD). These can include balance disorders, vomiting, contact dermatitis, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infections. Hypersensitivity reactions like bronchospasm, chest pain, cough, tachycardia, and urticaria have also been reported. It is important to discuss these potential side effects with a healthcare provider to manage and mitigate them effectively.

What prior FDA approvals exist?

The FDA has approved several treatments for Duchenne Muscular Dystrophy (DMD) that may have relevance for Becker Muscular Dystrophy (BMD) due to their similar pathophysiology. Eteplirsen, an exon-skipping drug, received accelerated approval for DMD patients with specific genetic mutations, as it increases dystrophin production in muscle fibers. Golodirsen, another exon-skipping therapy, was approved for DMD patients with deletions amenable to exon 53 skipping. These treatments aim to improve muscle function and slow disease progression, similar to the investigational drug EDG-5506 being studied for its potential to protect and enhance dystrophic muscle fibers in BMD.

What other types of research exist?

Promising novel alternatives to EDG-5506 for Becker Muscular Dystrophy patients include eteplirsen, which has shown efficacy in increasing dystrophin production and improving muscle function in Duchenne Muscular Dystrophy patients, and peptide-conjugated morpholinos (PPMOs) that restore dystrophin expression in animal models. These treatments could potentially be relevant for BMD.

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EDG-5506 for Becker Muscular Dystrophy (ARCH Trial)

Phase 1

Waitlist Available

Research Sponsored by Edgewise Therapeutics, Inc.

Eligibility Criteria Checklist

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

Must have

Body weight ≥ 50 kg at the Screening visit

Documented dystrophin mutation with phenotype consistent with BMD

Must not have

Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new drug called sevasemten to see if it can help people with Becker muscular dystrophy, a disease that weakens muscles. The drug aims to protect and improve muscle function. Participants will take the drug for an extended period with frequent check-ups to monitor safety and effectiveness.

Who is the study for?

This trial is for adult males aged 18-55 with Becker Muscular Dystrophy who can walk at least 100 meters. They must have completed a prior EDG-5506 study, weigh over 50 kg, and have a BMI of 20-34 kg/m2. Those on recent investigational drugs, with certain medical conditions or taking high doses of corticosteroids are excluded.

What is being tested?

The ARCH study is testing EDG-5506 in adults with Becker muscular dystrophy to see if it's safe and how the body processes it. It aims to protect and improve muscle function in patients by administering this experimental drug in an open-label, single-center Phase 1b setting.

What are the potential side effects?

As EDG-5506 is investigational, specific side effects aren't listed but generally may include reactions at the site of administration, potential muscle-related symptoms or changes in liver enzymes as commonly seen with new muscular treatments.

Eligibility Criteria

Inclusion Criteria

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

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I weigh at least 50 kg.

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I have a confirmed genetic mutation for Becker muscular dystrophy.

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I am a man between 18 and 55 years old.

Exclusion Criteria

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

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I am taking medication that strongly affects liver enzymes.

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.

Secondary study objectives

Number of participants with changes in FEV1

Number of participants with changes in FVC

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: TreatmentExperimental Treatment1 Intervention

Drug: Sevasemten

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Sevasemten

2021

Completed Phase 1

~20

0 / 4Eligibility criteria met

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

The most common treatments for Becker Muscular Dystrophy (BMD) aim to protect and improve the function of dystrophic muscle fibers. EDG-5506, an investigational drug, works by stabilizing muscle fibers to prevent damage during muscle contraction. Eteplirsen and casimersen are exon-skipping therapies that increase dystrophin production, a crucial protein missing or defective in BMD, thereby improving muscle function and slowing disease progression. Ataluren promotes ribosomal read-through of nonsense mutations, allowing for the production of functional dystrophin. These treatments are significant for BMD patients as they target the underlying genetic defects and muscle fiber instability, potentially improving quality of life and functional outcomes.

Pulmonary function and clinical correlation in DMD.ECM-Related Myopathies and Muscular Dystrophies: Pros and Cons of Protein Therapies.