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Study on the Long-term Effects of EDG-5506 for Adults and Adolescents with Becker Muscular Dystrophy

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a medication called EDG-5506 in individuals with Becker Muscular Dystrophy. Becker Muscular Dystrophy is a genetic disorder that causes muscle weakness and wasting over time. The medication being tested, EDG-5506, is taken in the form of a tablet and is designed to help manage symptoms associated with this condition.

The purpose of the study is to assess the long-term safety and effects of EDG-5506 on various health markers and physical abilities in both adults and adolescents who have Becker Muscular Dystrophy. Participants in the study will receive the medication over an extended period, and researchers will monitor their health and any changes in their condition. This includes looking at safety aspects, such as any side effects, and measuring changes in physical and neurological health.

Throughout the study, participants will undergo regular health check-ups, which may include laboratory tests, heart and lung function assessments, and other evaluations to ensure their well-being. The study aims to provide valuable information on how EDG-5506 can be used to support individuals living with Becker Muscular Dystrophy in the long term.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after completing a previous study related to Becker Muscular Dystrophy. Eligibility requires completion of specific visits in prior studies.

    Participants must agree to follow certain requirements, including those related to contraception, and provide informed consent.

  2. Step 2

    Medication administration

    The medication used in this study is EDG-5506, administered in the form of a tablet.

    The route of administration is oral. Specific dosage and frequency details are not provided in the available information.

  3. Step 3

    Monitoring and assessments

    The study aims to assess the safety and tolerability of long-term treatment with EDG-5506.

    Primary assessments include monitoring the incidence, frequency, and severity of any adverse events.

    Secondary assessments involve checking laboratory test results, vital signs, physical and neurological examinations, heart and lung function, and other health indicators.

  4. Step 4

    Study duration

    The estimated end date for the study is June 30, 2026.

    Recruitment for the study is expected to start on December 26, 2023.

Who can join the trial?

7 criteria

  • Must be a male with a diagnosis of Becker Muscular Dystrophy (BMD).
  • Must have participated in one of the following studies: EDG-5506-002, EDG-5506-201, or EDG-5506-202.
  • For EDG-5506-002, must have completed the final study visit at Month 24 or the early termination visit before Month 24.
  • For EDG-5506-201, must have completed the final study visit for Cohorts 1, 2, 4, and 5 at Month 12, or for Cohort 6 at Month 18.
  • For EDG-5506-202, must have completed at least 36 weeks of open-label treatment, which is Visit 14 at Week 52.
  • Must be willing to follow the contraception requirements as described in the study protocol.
  • Must be able to provide signed informed consent, which means agreeing to follow the study's rules and restrictions.

Who cannot join the trial?

4 criteria

  • Only males can participate, so females are excluded.
  • Participants must be within a specific age range, so those outside this range cannot join.
  • Individuals with conditions other than Becker Muscular Dystrophy are not eligible.
  • People considered part of a vulnerable population are not allowed to participate.
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Investigated drugs

  • EDG-5506

    is a medication being studied for its long-term effects on safety, biomarkers, and functional measures in adults and adolescents with Becker Muscular Dystrophy. This study aims to understand how the medication can help manage symptoms and improve the quality of life for those affected by this condition.

  • Sevasemten

    is another medication involved in the trial, with the main goal of assessing its safety and tolerability when used over a long period. This medication is being evaluated to determine how well patients can handle it and to identify any potential side effects that may occur with extended use.

What is already known about the treatment

Sevasemten – Sevasemten is administered orally and is currently being studied in clinical trials for its long-term effects on safety, biomarkers, and functional measures in individuals with Becker Muscular Dystrophy. It is not yet widely available in medical practice, as it is still under investigation. The main therapeutic indication for sevasemten is Becker Muscular Dystrophy, a genetic disorder that affects muscle function. At the molecular level, sevasemten works by modulating specific pathways involved in muscle contraction and strength. It is classified pharmacologically as a myosin modulator, aiming to improve muscle performance and reduce muscle damage.

Investigated diseases

Becker Muscular Dystrophy – Becker Muscular Dystrophy is a genetic disorder characterized by progressive muscle weakness and wasting. It primarily affects the skeletal muscles, which are responsible for movement, and can also impact the heart muscle. Symptoms often begin in childhood or adolescence and may include difficulty walking, muscle cramps, and fatigue. As the disease progresses, individuals may experience increased difficulty with physical activities and may require mobility aids. The progression of muscle weakness is generally slower compared to other forms of muscular dystrophy, such as Duchenne Muscular Dystrophy. The condition is caused by mutations in the dystrophin gene, which leads to insufficient production of the dystrophin protein necessary for muscle function.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-506231-15-00Protocol codeEDG-5506-203Estimated enrolment260 patientsSponsorEdgewise Therapeutics Inc.

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