Ongoing Clinical Trials for Becker’s Muscular Dystrophy
Currently, there are 3 clinical trials ongoing for Becker’s Muscular Dystrophy, testing new medications aimed at improving muscle function and reducing muscle damage. These studies are taking place across several European countries and include both adults and adolescents with the condition.
Clinical trial locations
- Belgium
- Denmark
- Study on the Effects of EDG-5506 for Adults and Adolescents with Becker Muscular Dystrophy
- Study on the Effects of EDG-5506 in Adults with Becker Muscular Dystrophy, McArdle Disease, or Limb-Girdle Muscular Dystrophy
- Study on the Long-term Effects of EDG-5506 for Adults and Adolescents with Becker Muscular Dystrophy
- France
- Germany
- Italy
- Netherlands
- Spain
Study on the Long-term Effects of EDG-5506 for Adults and Adolescents with Becker Muscular Dystrophy
This trial is designed to study the long-term safety and effects of a medication called EDG-5506, also known as sevasemten, in people with Becker’s Muscular Dystrophy. The medication comes in tablet form and is taken by mouth.
Who can join: This study is open only to males with a confirmed diagnosis who have already participated in one of three previous studies involving EDG-5506. Participants must have completed specific visits in those earlier studies. They also need to be willing to follow contraception requirements and provide informed consent to participate.
Who cannot join: Females are excluded from this study. People who are not within the specific age range required, those with conditions other than Becker’s Muscular Dystrophy, and individuals considered part of a vulnerable population are not eligible.
What the study focuses on: The main goal is to assess how safe and well-tolerated EDG-5506 is when used over a long period. Researchers will monitor for any side effects and check various health indicators including laboratory test results, vital signs, physical and neurological examinations, and heart and lung function. The study will also measure changes in blood markers related to muscle health and evaluate physical abilities using standardized tests.
Study medication: EDG-5506, also known as sevasemten, works by modulating specific pathways involved in muscle contraction and strength. It is classified as a myosin modulator and aims to improve muscle performance and reduce muscle damage in people with the condition.
Study on the Effects of EDG-5506 for Adults and Adolescents with Becker Muscular Dystrophy
This trial evaluates the safety and effectiveness of EDG-5506 (sevasemten) in both adults and adolescents. The study can last up to 52 weeks, and participants will be randomly assigned to receive either the active medication or a placebo.
Who can join: Males aged 18 to 50 years or adolescents aged 12 to 17 years with a documented dystrophin mutation consistent with the condition. Adults must have a history of walking ability beyond age 16 without steroids or beyond age 18 with steroids. Adolescents need genetic confirmation and specific functional abilities. All participants must be able to complete a 100-meter timed test in less than 200 seconds and achieve certain scores on the North Star Ambulatory Assessment. They must also agree to follow contraception requirements and provide informed consent.
Who cannot join: Only males are eligible for this study. People without the condition, those outside the specified age range, and members of vulnerable populations cannot participate.
What the study focuses on: The trial aims to understand how safe EDG-5506 is, how it moves through the body, and whether it can improve muscle function. Researchers will monitor blood markers of muscle damage, particularly serum creatine kinase, and assess functional abilities through physical tests. Throughout the study, participants will have regular check-ups including blood work and physical examinations.
Study medication: EDG-5506 (sevasemten) is a muscle modulator taken orally as a tablet. It targets pathways involved in muscle contraction and repair, with the goal of enhancing muscle strength and reducing damage.
Study on the Effects of EDG-5506 in Adults with Becker Muscular Dystrophy, McArdle Disease, or Limb-Girdle Muscular Dystrophy
This trial studies how EDG-5506 affects markers of muscle damage in adults with three different muscle-related conditions, including Becker’s Muscular Dystrophy. The study is double-blind, meaning neither participants nor researchers know who receives the actual medication or a placebo.
Who can join: Adults with Becker’s Muscular Dystrophy or Limb-Girdle Muscular Dystrophy Type 2I must be between 18 and 65 years old. Participants need a confirmed diagnosis and must be able to follow an exercise routine on a stationary bike. They should not have any other medical condition that would interfere with the study or any active muscle injury at the start. Participants must be fluent in Danish or English, willing to follow all study requirements including birth control methods, and able to provide written consent.
Who cannot join: People with serious health conditions that could interfere with the study, pregnant or breastfeeding individuals, those who participated in another trial within the last 30 days, people with allergies to the study medication, and those unable to follow study procedures. People with a history of drug or alcohol abuse, severe heart problems, uncontrolled high blood pressure, severe liver or kidney disease, or active infections requiring treatment are also excluded.
What the study focuses on: The main goal is to understand how EDG-5506 affects markers in the body that show muscle damage, particularly in response to exercise. Participants will follow a cycle exercise protocol as part of the study. Researchers will conduct regular health monitoring through physical examinations and laboratory tests.
Study medication: EDG-5506 (sevasemten) is given as an oral tablet. It works by targeting specific pathways in muscle cells to reduce damage and improve muscle function, with the aim of helping the body respond better to exercise.
Summary
All three clinical trials focus on testing the same investigational medication, EDG-5506 (sevasemten), which is designed to improve muscle function and reduce muscle damage in people with Becker’s Muscular Dystrophy. The medication works as a myosin modulator, targeting pathways involved in muscle contraction and repair.
Two of the trials are available across multiple European countries including Belgium, Denmark, France, Germany, Italy, Netherlands, and Spain, while one trial is currently only available in Denmark. The studies include both short-term assessments and long-term follow-up, with one trial specifically designed for participants who have already completed earlier studies with the same medication.
The trials are open to both adults and adolescents, though some studies focus only on adults. All studies require male participants with confirmed diagnoses and specific functional abilities. The research aims to gather comprehensive information about the medication’s safety, tolerability, and potential benefits for people living with this condition.
