What side effects are associated with this type of intervention?

Common treatments for Becker Muscular Dystrophy, particularly those involving antisense oligonucleotides and gene therapies, often have side effects such as upper respiratory tract infections, injection site reactions, cough, fever, headache, and gastrointestinal issues like vomiting and nausea. Additionally, renal toxicity is a concern with some antisense oligonucleotides, necessitating regular monitoring of kidney function. Hypersensitivity reactions, including bronchospasm and chest pain, have also been reported.

What prior FDA approvals exist?

FDA-approved treatments for Becker Muscular Dystrophy (BMD) include genetic therapies such as exon skipping drugs. These drugs, including eteplirsen, golodirsen, viltolarsen, and casimersen, are designed to increase dystrophin production by skipping specific exons in the DMD gene. While these treatments are primarily approved for Duchenne Muscular Dystrophy (DMD), they may also be relevant for BMD due to similar genetic mechanisms. Specific information on treatments with mechanisms similar to Sevasemten (EDG-5506) was not provided.

What other types of research exist?

Viltolarsen and Casimersen are promising alternatives for Becker Muscular Dystrophy patients. Viltolarsen has shown an increase in dystrophin levels and improvement in timed function tests in clinical trials. Casimersen, approved by the FDA, targets exon 45 skipping in the DMD gene, which is relevant for some BMD cases.

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Small Molecule

EDG-5506 for Becker Muscular Dystrophy

Q: What is the mechanism of action of this treatment?

The most common treatments for Becker Muscular Dystrophy (BMD) include genetic therapies and pharmacological interventions. Genetic therapies, such as exon skipping drugs (e.g., eteplirsen, golodirsen, viltolarsen), work by skipping over specific exons in the dystrophin gene to produce a shorter but functional dystrophin protein, which is crucial for muscle function. Pharmacological treatments often target downstream effects of the disease, such as chronic inflammation and muscle degeneration. For example, glucocorticoids are used to reduce inflammation and slow disease progression. These treatments are important for BMD patients as they aim to improve muscle function, reduce symptoms, and enhance quality of life by addressing both the genetic cause and the secondary effects of the disease.

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

Able to complete the 100-meter timed test in < 150 seconds.

Adults (aged 18 to 50 years, inclusive) with a documented dystrophin mutation and phenotype consistent with Becker muscular dystrophy

Must not have

Forced vital capacity predicted <60% or using daytime ventilatory support

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months (canyon cohorts 1, 2, 4, 5), 18 months (grand canyon cohort 6)

Awards & highlights

Summary

This trial is testing a new drug called sevasemten to see if it can help people with Becker muscular dystrophy, a condition that weakens muscles. The study aims to find out if the drug is safe and effective in improving muscle function and reducing symptoms.

Who is the study for?

This trial is for adolescents (12-17) and adults (18-50) with Becker muscular dystrophy confirmed by genetic testing. Participants must be able to complete a 100-meter walk within certain times, perform specific physical assessments, and have been ambulatory beyond certain ages with or without steroids.

What is being tested?

The GRAND CANYON study tests the safety and effectiveness of EDG-5506 at different doses (5 mg, 10 mg, 12.5 mg) compared to a placebo in treating Becker muscular dystrophy. It's randomized and double-blind, meaning neither participants nor researchers know who gets the drug or placebo.

What are the potential side effects?

While specific side effects of EDG-5506 are not listed here, common side effects in trials like this may include gastrointestinal symptoms, fatigue, muscle pain or cramps, headaches, and potential allergic reactions.

Eligibility Criteria

Inclusion Criteria

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

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I can walk 100 meters in less than 150 seconds.

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I am between 18 and 50 years old with a confirmed diagnosis of Becker muscular dystrophy.

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I was able to walk after 16 without steroids and after 18 with steroids.

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I am a teenager with a confirmed genetic mutation for Becker muscular dystrophy.

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I can do a specific physical test and score between 5 and 32.

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I am 18-50 years old with Becker muscular dystrophy, and I could walk after 16 without steroids or after 18 with them.

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I can complete a physical ability test with a specific score range.

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I can walk 100 meters in less than 150 seconds.

Exclusion Criteria

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

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My lung function is severely reduced or I need help breathing during the day.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months (canyon cohorts 1, 2, 4, 5), 18 months (grand canyon cohort 6)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months (canyon cohorts 1, 2, 4, 5), 18 months (grand canyon cohort 6)

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months (canyon cohorts 1, 2, 4, 5), 18 months (grand canyon cohort 6) for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Change from Baseline in serum Creatine Kinase

Number of adverse events in those treated with sevasemten or placebo

Severity of adverse events in those treated with sevasemten or placebo

Secondary study objectives

Change from Baseline in 100-meter timed test

Change from Baseline in growth as assessed by height centile on World Health Organization growth charts

Change from Baseline in stride velocity (95th percentile)

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