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Study on the Effects of EDG-5506 in Adults with Becker Muscular Dystrophy, McArdle Disease, or Limb-Girdle 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 new treatment called EDG-5506 on certain muscle-related diseases. The diseases being studied are Becker Muscular Dystrophy, McArdle Disease, and Limb-Girdle Muscular Dystrophy. These are conditions that affect the muscles, leading to weakness and other symptoms. The treatment being tested, EDG-5506, is taken in the form of a tablet.

The purpose of the study is to understand how EDG-5506 affects markers in the body that indicate muscle damage. Participants in the study will be randomly assigned to receive either the EDG-5506 tablet or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This helps ensure the results are unbiased.

Throughout the study, participants will undergo various assessments to monitor their health and the effects of the treatment. These assessments will include checking for any side effects, measuring changes in muscle-related markers, and evaluating overall health through tests like blood work and physical exams. The study will last for a period of time, during which participants will be closely monitored to gather important information about the safety and effectiveness of EDG-5506.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment and consent

    Upon joining the study, participants will be required to sign an informed consent form. This document confirms understanding of the study's purpose, procedures, and potential risks.

    Participants must meet specific criteria, such as age and medical condition, to be eligible for the study.

  2. Step 2

    Initial assessment

    Participants will undergo an initial assessment to evaluate their health status. This includes a review of medical history and a physical examination.

    Laboratory tests will be conducted to establish baseline health parameters.

  3. Step 3

    Medication administration

    Participants will be randomly assigned to receive either the study medication, EDG-5506, or a placebo. The medication is administered orally in tablet form.

    The dosage and frequency of administration will be determined by the study protocol and communicated to participants.

  4. Step 4

    Exercise protocol

    Participants will follow a cycle exercise protocol as part of the study. This is designed to assess the effect of the medication on muscle biomarkers.

    The ability to comply with this exercise protocol is a requirement for participation.

  5. Step 5

    Ongoing monitoring

    Participants will attend regular visits for monitoring of health status and response to the medication. This includes physical examinations and laboratory tests.

    Monitoring will continue throughout the study duration, which is estimated to end on August 15, 2025.

  6. Step 6

    Final assessment

    At the end of the study, participants will undergo a final assessment to evaluate the overall impact of the medication.

    This includes a review of any adverse events and changes in health parameters since the start of the study.

Who can join the trial?

9 criteria

  • Participants with Becker Muscular Dystrophy or Limb-Girdle Muscular Dystrophy Type 2I must be between 18 and 65 years old. Participants with McArdle Disease can be up to 75 years old.
  • Participants must have a confirmed diagnosis of Becker Muscular Dystrophy, McArdle Disease, or Limb-Girdle Muscular Dystrophy Type 2I.
  • Participants must be able to follow an exercise routine on a stationary bike, as judged by the study doctor.
  • Participants should not have any other medical condition that would make it difficult to understand the study or attend study visits, according to the study doctor.
  • Participants should not have any active muscle injury at the start of the study.
  • Participants must be willing and able to follow all study requirements.
  • Participants must be able to speak and read fluently in Danish or English.
  • Participants must agree to use birth control methods as described in the study.
  • Participants must be able to give written consent to join the study, which includes agreeing to follow the study rules and restrictions.

Who cannot join the trial?

10 criteria

  • Patients with any other serious health condition that could interfere with the study.
  • Patients who are pregnant or breastfeeding.
  • Patients who have participated in another clinical trial within the last 30 days.
  • Patients with a history of allergic reactions to the study medication or similar drugs.
  • Patients who are unable to follow the study procedures or instructions.
  • Patients with a history of drug or alcohol abuse within the past year.
  • Patients with severe heart problems.
  • Patients with uncontrolled high blood pressure.
  • Patients with severe liver or kidney disease.
  • Patients with active infections that require treatment.
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Investigated drugs

  • EDG-5506

    is a medication being studied to see how it affects certain markers in the body that show muscle damage. This study is focused on people with specific muscle diseases, like Becker Muscular Dystrophy, McArdle Disease, or Limb-Girdle Muscular Dystrophy. The goal is to understand if EDG-5506 can help improve the body's response to exercise in these conditions.

  • Sevasemten

    is another medication being tested in this trial. It is being studied to see if it is safe for people with neuromuscular diseases. Researchers are also looking at how sevasemten affects markers in the body that indicate muscle damage, specifically in people with Becker Muscular Dystrophy, McArdle Disease, or Limb-Girdle Muscular Dystrophy Type 2I. The aim is to see if sevasemten can help reduce muscle damage in these conditions.

What is already known about the treatment

Sevasemten – Sevasemten is administered orally in the form of a tablet and is currently being studied in clinical trials for its potential benefits in treating certain neuromuscular diseases. It is primarily being investigated for its effects on muscle damage biomarkers in conditions such as Becker Muscular Dystrophy, McArdle Disease, and Limb-Girdle Muscular Dystrophy. The medication works by targeting specific pathways in muscle cells to reduce damage and improve muscle function. Sevasemten is classified as a therapeutic agent for neuromuscular disorders, and its safety and effectiveness are being evaluated in ongoing research.

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. The disease progresses slowly, with symptoms often appearing in adolescence or early adulthood. Muscle weakness typically begins in the hips and pelvic area, then spreads to the thighs and shoulders. Over time, individuals may experience difficulty walking, climbing stairs, and lifting objects. The progression of muscle weakness can vary widely among individuals.

  • McArdle Disease

    McArdle Disease, also known as Glycogen Storage Disease Type V, is a metabolic disorder affecting muscle metabolism. It is caused by a deficiency of the enzyme myophosphorylase, which is necessary for breaking down glycogen in muscle cells. This leads to an inability to produce energy during exercise, resulting in muscle pain, cramps, and fatigue. Symptoms typically appear during childhood or adolescence and are often triggered by physical activity. The disease progresses with repeated episodes of muscle pain and potential muscle damage. Some individuals may develop a second wind phenomenon, where symptoms improve after a brief rest during exercise.

  • Limb-Girdle Muscular Dystrophy

    Limb-Girdle Muscular Dystrophy is a group of genetic disorders characterized by progressive muscle weakness and wasting, primarily affecting the shoulder and hip areas. The disease can begin in childhood or adulthood, with varying rates of progression. Muscle weakness typically starts in the proximal muscles, such as those around the hips and shoulders, and may later affect the distal muscles. As the disease progresses, individuals may experience difficulty with activities such as walking, running, and lifting objects. The severity and progression of symptoms can vary significantly among individuals. Different genetic mutations are responsible for the various subtypes of this condition.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2022-500215-39-00Protocol codeEDG-5506-202Estimated enrolment21 patientsSponsorEdgewise Therapeutics Inc.

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