EDG-5506 for Becker Muscular Dystrophy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial is testing a new drug called sevasemten to see if it can help people with Becker muscular dystrophy, a condition that weakens muscles. The study aims to find out if the drug is safe and effective in improving muscle function and reducing symptoms.
Do I have to stop taking my current medications for this trial?
The trial protocol does not specify if you must stop taking your current medications. However, you cannot have taken oral corticosteroids for Becker muscular dystrophy in the past 6 months or any investigational drug within 30 days or 5 half-lives before the screening.
Will I have to stop taking my current medications?
The trial requires that you have not taken oral corticosteroids for Becker muscular dystrophy in the past 6 months. If you are on other medications, the protocol does not specify whether you need to stop them, so it's best to discuss this with the study team.
What safety data is available for EDG-5506 in Becker Muscular Dystrophy?
The provided research does not contain specific safety data for EDG-5506 in Becker Muscular Dystrophy. The studies mentioned focus on other treatments and conditions, such as Drisapersen, DT-DEC01, Edasalonexent, and Vamorolone, primarily in Duchenne Muscular Dystrophy or animal models. Therefore, no relevant safety data for EDG-5506 is available in the given research.12345
Is the drug EDG-5506, Placebo a promising treatment for Becker Muscular Dystrophy?
The information provided does not mention EDG-5506 or Placebo as promising treatments for Becker Muscular Dystrophy. The articles focus on other aspects of the disease and different treatments, like vamorolone, which shows potential in increasing dystrophin protein and improving muscle function in a mouse model. Therefore, there is no evidence here to suggest that EDG-5506, Placebo is promising for Becker Muscular Dystrophy.46789
How is the drug EDG-5506 different from other treatments for Becker muscular dystrophy?
What data supports the idea that EDG-5506 for Becker Muscular Dystrophy is an effective treatment?
The available research does not provide specific data on the effectiveness of EDG-5506 for Becker Muscular Dystrophy. However, it mentions that there are no drugs and few clinical trials for this condition, indicating a lack of direct evidence for EDG-5506. In contrast, another treatment, prednisone, showed dramatic and sustained improvement in strength for some patients with Becker Muscular Dystrophy, suggesting it might be a more effective option based on current data.78101112
Who Is on the Research Team?
Joanne Donovan, MD, PhD
Principal Investigator
Edgewise Therapeutics, Inc.
Roxana D. Dreghici
Principal Investigator
Edgewise Therapeutics, Inc.
Are You a Good Fit for This Trial?
This trial is for adolescents (12-17) and adults (18-50) with Becker muscular dystrophy confirmed by genetic testing. Participants must be able to complete a 100-meter walk within certain times, perform specific physical assessments, and have been ambulatory beyond certain ages with or without steroids.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive sevasemten or placebo to evaluate safety, pharmacokinetics, biomarkers, and functional measures
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Extension
Participants in the GRAND CANYON cohort receive extended treatment to evaluate long-term safety and efficacy
What Are the Treatments Tested in This Trial?
Interventions
- EDG-5506
- Placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
Edgewise Therapeutics, Inc.
Lead Sponsor
Sysnav
Industry Sponsor
Medpace, Inc.
Industry Sponsor
Dr. August J. Troendle
Medpace, Inc.
Chief Executive Officer since 1992
MD from the University of Maryland, School of Medicine; MBA from Boston University
Dr. Reinilde Heyrman
Medpace, Inc.
Chief Medical Officer since 2017
MD
ImagingNMD
Collaborator
SYSNAV
Industry Sponsor