About the ImagingNMD Study
Seeking Adults 18-62 Diagnosed With Becker Muscular Dystrophy.
Becker Muscular Dystrophy (BMD) is caused by a genetic defect and leads to muscle instability and progressive muscle weakness. The natural history of the disease progression in BMD is not well understood, and drug development and clinical trials have been slow.
Becker muscular dystrophy (BMD) has been largely neglected in therapeutic development due to its heterogeneity, small patient population, lack of outcome measures, and uncertainty surrounding the patterns of disease progression, which may be mutation-dependent.
This study will help guide future clinical trials of new drugs for Becker MD by mapping the disease progression of Becker MD.
The purpose of this study is to conduct multi-center research to validate the potential of noninvasive magnetic resonance imaging (MRI) to monitor disease progression and to serve as a sensitive outcome measure for future clinical trials in muscular dystrophies. This research provides the necessary access to sensitive measures such as MRI which are critical for testing new drugs for BMD treatment.
This is not an interventional study. No treatment will be provided during study participation.
Overview of Procedures
During the screening phase, a research coordinator and the study team will ensure that this study is a good fit for you based on the eligibility criteria. This process will include a survey to confirm eligibility and informed consent.
During the participation phase, we will use an MRI to measure the muscles of men with BMD and muscles of age-matched, healthy controls to determine whether this new method can be used to track disease progression – measuring functional skills such as walking, arm and leg function, and breathing function. We also hope to determine if MRI can be used as an outcome measure for researchers to evaluate whether a drug is effective in BMD.
Muscle biopsies will be used to understand how these new measures relate to disease processes within the muscle itself and be used to compare information collected through imaging. If you are selected for this study, you will have a needle biopsy on your second visit.
Participants will meet with experienced researchers and can choose to receive the results at the end of the study. Our researchers will walk you through the results and are happy to discuss questions you may have.
What is this data being used for?
- Evaluating the relationship between the muscle biopsy (the histology-chemical makeup) and the MRI measures to assist future clinical trials that are targeting dystrophin restoration.
- Sharing all of our data with our research and pharmaceutical community to facilitate the development of future treatments for BMD.
- Sharing all of your data with you, so you can have a better understanding of your own health and progression. We couldn’t do this without you—you’re our most important team member.
The following General Inclusion Criteria* offer the essential factors that determine if a person is eligible for this study. It is not a complete list. Each potential participant who satisfies these general guidelines will be further screened by one of our research coordinators.
To be included, each participant must be:
- Diagnosed with BMD
- 18–62 years old
- Ambulatory or non-ambulatory
The following General Exclusion Criteria* indicate the most significant factors why a person may be ineligible for the study. Once again, a research coordinator will discuss these factors further during a telephone screening.
Individuals may be excluded if they:
- Have a cardiac defibrillator
- Have a pacemaker
- Have another neuromuscular disease/diagnosis that affects your muscles
- Require non-invasive ventilation during the day or are on a ventilator
*Disclaimer: These are only general guidelines for inclusion in the study. A site coordinator will go into more detail with each individual at the time of screening for eligibility.
There are no costs for you to participate. We’re committed to ensuring that all participants can easily access this study. If this study is a good fit, you will receive all study-related procedures, including medical screenings and assessments, all at no cost to you. We will also pay directly for your lodging and travel to one of our sites (Florida or Portland).
Approximately 105 men with BMD, 18–62 years of age
Your participation in this study will last for a total duration of 24 months. You will be asked to participate in 3 visits over that time (baseline, 12 and 24 months). Each visit will take 6–8 hours. Participation is completely voluntary, and you may discontinue at any time.
This study is being conducted on behalf of the University of Florida and Oregon Health and Science University with trained and qualified specialists.
An Institutional Review Board, or IRB, is a panel of professionals with diverse experiences in research, medicine, and ethics that reviews and approves human studies before they are started. Before a study can begin, an independent IRB must review all information about the study design to decide whether it’s ethical to ask patients to volunteer for the study.
There are state, national, and international regulations and policies in place to protect the rights and safety of people while participating in a study, which ensures that strict scientific and ethical principles are followed. Studies are run by medical professionals who follow these guidelines to make sure that participants are safe, and the protocol is being followed.
All study participation is completely voluntary, and you may decide to discontinue at any time and for any reason.
How to Enroll
If you are interested in learning more about whether the Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy study—specific to Becker MD—may be a good fit for you, please submit the intake form. The answers you submit will be used for study purposes only.
Once you submit the form, a member from our team will reach out to you with Becker Muscular Dystrophy resources and connect you with a member of the research team if you may qualify for this study.
Please note that answering these questions does not obligate you to participate in the study. If you have any questions, simply email Imagingnmd@phhp.ufl.edu.