In short
There is currently 1 ongoing clinical trial for Glycogen Storage Disease Type V (also known as McArdle Disease). This trial is investigating a new medication aimed at reducing muscle damage in people with this condition and other related muscle diseases. The study is being conducted in Denmark and involves testing an oral medication called EDG-5506, which may help improve the body's response to exercise.
Clinical trial locations
Study on the Effects of EDG-5506 in Adults with Becker Muscular Dystrophy, McArdle Disease, or Limb-Girdle Muscular Dystrophy
This clinical trial is investigating how a new medication called EDG-5506 affects people with muscle-related diseases, including McArdle Disease. The study aims to understand whether this treatment can help reduce markers in the body that indicate muscle damage. EDG-5506 is taken as a tablet, and the trial is designed to compare its effects against a placebo in a double-blind study, meaning neither participants nor researchers know who receives the actual medication during the trial period.
Main focus and goal: The primary purpose of this study is to evaluate how EDG-5506 affects biomarkers related to muscle damage in people with specific muscle conditions. Researchers want to understand if this medication can improve the body's response to exercise and potentially reduce muscle damage. The study also includes a cycle exercise protocol to assess how the medication performs during physical activity. Another medication called sevasemten is also being tested in this trial for similar purposes.
Who can participate: The trial is open to adults with a confirmed diagnosis of McArdle Disease who are between 18 and 75 years old. Participants must be able to follow an exercise routine on a stationary bike as determined by the study doctor. They should be fluent in Danish or English, able to attend all study visits, and willing to follow the study requirements. Participants must also agree to use appropriate birth control methods during the study and be able to provide written consent.
Who cannot participate: People cannot join this trial if they have other serious health conditions that could interfere with the study, such as severe heart problems, uncontrolled high blood pressure, or severe liver or kidney disease. Pregnant or breastfeeding women are not eligible. The trial also excludes those who have participated in another clinical trial within the last 30 days, have a history of allergic reactions to the study medication or similar drugs, have active infections requiring treatment, or have a history of drug or alcohol abuse within the past year. People with active muscle injuries at the start of the study are also excluded.
Investigational drugs: The main medication being tested is EDG-5506, an oral tablet designed to affect markers showing muscle damage. The study also involves testing sevasemten, which is being evaluated for safety and its effects on reducing muscle damage biomarkers in people with neuromuscular diseases. Both medications target specific pathways in muscle cells to potentially reduce damage and improve muscle function during exercise.
Summary
Currently, there is one active clinical trial available for people with Glycogen Storage Disease Type V, being conducted in Denmark. This trial focuses on investigating new oral medications that may help reduce muscle damage and improve exercise tolerance in people with this condition. The study is part of a broader research effort that also includes people with Becker Muscular Dystrophy and Limb-Girdle Muscular Dystrophy, reflecting the shared challenges these conditions present regarding muscle damage during physical activity. The trial offers an opportunity for eligible patients in Denmark to access experimental treatments that may help manage their symptoms.
