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

Fast replyInvestigationalNo placebo
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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 a condition called Becker Muscular Dystrophy (BMD), which is a genetic disorder that affects muscle strength and function. The study will evaluate a treatment known as EDG-5506, which is taken in the form of a tablet. The purpose of the study is to assess the safety and effects of EDG-5506 on individuals with BMD, including both adults and adolescents.

Participants in the study will be randomly assigned to receive either the EDG-5506 treatment or a placebo. The study will monitor various health indicators, such as muscle function and certain blood markers, over a period of time. The treatment period can last up to 52 weeks, during which participants will have regular check-ups to ensure their safety and to track any changes in their condition.

The study aims to gather information on how well EDG-5506 works in improving muscle function and its overall safety for people with Becker Muscular Dystrophy. This information could help in developing better treatments for this condition in the future. Participants will be closely monitored for any side effects or changes in their health throughout the study.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, participants will be assigned to one of several groups, known as cohorts. Each cohort has specific objectives and procedures tailored to assess the effects of the medication being tested.

  2. Step 2

    Medication administration

    Participants will receive the study medication, EDG-5506, in the form of a tablet. The medication is taken orally. The dosage, frequency, and duration of administration will be determined based on the specific cohort to which the participant is assigned.

  3. Step 3

    Safety and tolerability assessment

    Throughout the study, the safety and tolerability of the medication will be closely monitored. This involves regular check-ups to observe any side effects or adverse reactions to the medication.

  4. Step 4

    Biomarker evaluation

    Participants will undergo tests to measure specific biomarkers, such as serum creatine kinase (CK), which can provide information about muscle health and the effects of the medication.

  5. Step 5

    Functional measures assessment

    Participants will be evaluated using various functional tests, such as the North Star Ambulatory Assessment (NSAA), to assess their physical abilities and any changes over time.

  6. Step 6

    Pharmacokinetics study

    The study will include an analysis of how the medication is processed in the body, known as pharmacokinetics. This involves measuring the concentration of the medication in the blood at different times.

  7. Step 7

    End of study procedures

    At the conclusion of the study, participants will undergo final assessments to evaluate the overall effects of the medication. This includes a review of all collected data and a final health check.

Who can join the trial?

9 criteria

  • Must be an adult aged 18 to 50 years or an adolescent aged 12 to 17 years.
  • Must have a documented dystrophin mutation and a condition consistent with Becker Muscular Dystrophy (BMD).
  • For adults, must have a history of being able to walk beyond 16 years of age without steroids, or beyond 18 years of age with steroids.
  • For adolescents, must have genetic confirmation of a dystrophin mutation and either an in-frame mutation or, if an out-of-frame mutation, the ability to stand up from lying down in less than 10 seconds.
  • Must be male at birth.
  • Must be able to complete a 100-meter timed test in less than 200 seconds, with or without mobility aids.
  • Must be able to perform the North Star Ambulatory Assessment (NSAA) and achieve a score of 5 to 32 for adults or a score of 5 or more for adolescents.
  • Must be willing to follow contraception requirements as described in the study protocol.
  • Must be capable of giving signed informed consent, agreeing to follow the study requirements and restrictions.

Who cannot join the trial?

4 criteria

  • Participants cannot join if they do not have Becker Muscular Dystrophy (BMD).
  • Only male participants are allowed in the study.
  • Participants must be within the specified age range for the study.
  • Participants cannot join if they are part of a vulnerable population, which means groups that might need special protection or care.
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Investigated drugs

  • EDG-5506

    is a medication being studied to see how it affects people with Becker Muscular Dystrophy, a condition that causes muscle weakness. The trial is looking at how safe the medication is, how it moves through the body, and how it might help improve muscle function. Researchers are also checking if it can change certain markers in the blood that are linked to muscle damage.

  • Sevasemten

    is another medication being tested in this trial. It is being studied to understand its safety and how well people with Becker Muscular Dystrophy can tolerate it. The trial is also examining if sevasemten can lower levels of a specific enzyme in the blood called creatine kinase, which is often higher in people with muscle damage. The study includes both adults and adolescents to see how the medication works in different age groups.

What is already known about the treatment

Sevasemten – Sevasemten is administered orally and is currently being studied in clinical trials for its potential use in treating Becker Muscular Dystrophy (BMD). It is in the experimental phase, with ongoing research to evaluate its safety, tolerability, and effects on biomarkers like serum creatine kinase in both adults and adolescents with BMD. The main therapeutic indication for sevasemten is to improve muscle function and reduce muscle damage in individuals with BMD. At the molecular level, sevasemten works by modulating specific pathways that are involved in muscle contraction and repair, aiming to enhance muscle strength and function. It is classified pharmacologically as a muscle modulator, focusing on improving muscle health in those affected by muscular dystrophies.

Investigated diseases

Becker Muscular Dystrophy (BMD) – 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 the heart muscle. The disease is caused by mutations in the dystrophin gene, leading to insufficient production of the dystrophin protein, which is crucial for muscle function. Symptoms often begin in adolescence or early adulthood and include difficulty walking, muscle cramps, and fatigue. As the disease progresses, individuals may experience increased difficulty with mobility and may require assistance with walking. The progression of muscle weakness can vary significantly among individuals with BMD.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2022-500090-13-00Protocol codeEDG-5506-201Estimated enrolment170 patientsSponsorEdgewise Therapeutics Inc.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).