Intended for U.S. residents 18 years of age and older.
Individuals should always discuss questions about their medical care with their health care provider.
Diagnosed With Becker Muscular Dystrophy (BMD)?
Find out about a new clinical study in Becker muscular dystrophy and if you are eligible to participate
CLINICALTRIALS.GOV ID: NCT05291091
With your participation, you may be able to help researchers investigate a potential treatment for Becker muscular dystrophy
Becker muscular dystrophy (also known as Becker) is a genetic disorder that affects mostly males. Individuals with Becker experience muscle damage that impacts everyday movement due to the progressive loss of muscle function. This weakness and wasting of the muscles of the body and heart may lead to the eventual loss of the ability to walk and/or maintain independent living.
About Becker
Muscular Dystrophy
The diagnosis of Becker muscular dystrophy is based on blood and genetic tests. Muscle weakness can start at any age, but once it starts, it progresses, leading to the loss of the ability to perform daily activities, such as preventing falls and being able to get up afterwards. Most individuals eventually progress to a wheelchair with additional arm weakness.
Not actual patients
About the Study
- The investigational study drug is called EDG-5506. Participants will be given either EDG-5506 or placebo,A placebo looks like EDG-5506 but contains no active ingredientsonce per day in tablet form, taken by mouth.
- The tablet will be taken at night prior to bedtime, either with or without food.
- Participants will be randomly assigned by a computer program to either EDG-5506 or placebo. Participants have a 2 out of 3 chance of receiving EDG-5506 and a 1 out of 3 chance of receiving placebo.
- There will be 7 visits to the study site and 4 phone calls with a study representative, over a period of about 20 months.
- Participants will wear an activity monitor (a simple watch-like device) during waking hours, at various times throughout the study.
The investigational study drug (EDG-5506) is currently not approved for use in Becker muscular dystrophy by any regulatory agency, as its safety and effectiveness have not been established for the treatment of Becker muscular dystrophy.
Who Can Participate
To participate in the study you must:
Be male, between 18 and 50 years old
Have a diagnosis of Becker muscular dystrophy confirmed by genetic testing
Be able to walk, with or without a cane or other assistive device, approximately 100 meters (the length of a football field) in less than 200 seconds
There are additional requirements individuals must meet in order to take part in this study. A study representative will discuss them with you.
Study Overview
*For participant's convenience, there are many sites across the U.S. and travel expenses will be covered through the study duration.
Is the
GRAND CANYON Study
Right for You?
Not actual patients
Frequently Asked Questions
What is known about EDG-5506?
EDG-5506 is an investigational drug being studied for the treatment of Becker. EDG-5506 has not been approved by any regulatory authority. The safety and effectiveness of EDG-5506 have not been proven. By participating, you are helping us learn more about how EDG-5506 could help you and help others living with Becker, too.
How is EDG-5506 thought to work?
EDG-5506 is designed to prevent contraction-induced muscle injury that occurs with daily activity in individuals with Becker. In this study, we are testing whether EDG-5506 decreases damage to muscle and maintains function by changing how contraction occurs. Limiting muscle damage in people with Becker could help change disease progression.
What will happen in the study?
The GRAND CANYON study will last up to 20 months, which includes a screening period of up to 4 weeks. If you join the study, you will take tablets of either EDG-5506 or placebo (looks like EDG-5506 but contains no active ingredients) once daily by mouth for 18 months. You will be randomly assigned by chance (like the flip of a coin) to EDG-5506 or placebo. Neither you nor the study doctor will know whether you receive EDG-5506 or placebo, but in an emergency, the study doctor can obtain this information.You have a 2 out of 3 chance of receiving EDG-5506 and a 1 out of 3 chance of receiving placebo. You will visit the site approximately 7 times for safety assessments, functional assessments, and blood tests. You will wear an activity monitor to measure routine daily activity at various times throughout the study. There will be a follow-up phone call within 4 weeks of stopping treatment.There are no muscle biopsies in this study.
Are there any inclusion criteria related to age, genetic mutations, or physical function?
Males, between 18 and 50 years old, with a diagnosis of Becker. You will need to provide a copy of the genetic report documenting your diagnosis. This may be in the form of a lab report. You will need to be able to complete different physical function activities such as walking 100 meters under a certain amount of time. Assistive devices (like orthotics or cane) may be used to complete 100 meters.
Can I continue to take other medications and supplements?
There are some medications and supplements that are not allowed to be taken during the study. Some of these include echinacea, St. John’s wort, digoxin, barbiturates, some anti-fungal medications, ciprofloxacin (an antibiotic), and diltiazem. You also cannot have taken oral steroids for management of Becker for more than 10 days in the previous 6 months. Speak to your doctor if you have questions about medications and supplements you can take during the GRAND CANYON study.
Where is the study taking place? What if I don’t live near a research site?
This study is planned to take place at many sites across the United States. A travel support company will arrange your transportation to and from site visits. This includes air travel if you live far from the nearest research site.
Is there assistance for travel expenses?
The study will pay for your travel expenses, including transportation, hotel accommodations, and meals for you and one additional traveler to and from site visits. The site staff will work with the travel support company to arrange your travel for you. Additional out-of-pocket travel expenses (like parking and tolls) may also be reimbursed.
How do I find out more about participating in the GRAND CANYON study?
If you are interested in taking part in the GRAND CANYON study and would like more information about participating, leave your contact information and a GRAND CANYON study representative will contact you.
Sources:
• Data on file, Edgewise Therapeutics, 2023
• https://clinicaltrials.gov/ct2/show/record/NCT05291091
• Bello, L. et al. Functional changes in Becker muscular dystrophy: implications for clinical trials in dystrophinopathies. Sci. Rep. 6, 32439; doi:10.1038/srep32439 (2016).
• Magot A, et al. Diagnosis and management of Becker muscular dystrophy: the French guidelines. J Neurol. 2023 Jul9. doi:10.1007/s00415-023-11837-5
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