PPMD was happy to recently host Dr. Krista Vandenborne, Director of ImagingNMD, and Dr. Claudia Senesac, ImagingNMD project manager, from University of Florida’s ongoing ImagingNMD program for a webinar on the development of a new study evaluating imaging biomarkers for Becker muscular dystrophy.
This is the first time that ImagingNMD has opened their imaging protocol to men with Becker, expanding greatly upon our knowledge of Becker natural history and progression over time.
Webinar Recap & Recording
Through a series of powerful MRI images and associated data points, Dr. Vandenborne eloquently illustrated the pattern of muscle loss and fat replacement in Duchenne muscular dystrophy. She also provided an explanation of fat fraction, which is a measurement of fat replacement based on water and fat imaging and provides an assessment of disease progression.
Dr. Vandenborne then shared an explanation of MRI changes compared with the 6-minute walk test, asking the question, “Can you see changes in fat fraction even when a child’s 6-minute walk test is stable?” In fact, MRI can detect changes in muscle even when function is otherwise stable. This wide swath of data has been used to advance Duchenne care and research, and has been used to inform natural history and drug development in a number of clinical trials, decreasing the need for placebo arms in some trials.
All of this information illuminated the need to expand learnings to include Becker muscular dystrophy. This is particularly true due to the slower progression of Becker muscular dystrophy, smaller population, and variability across people with Becker, making monitoring over long periods of time critical. As part of this new study, researchers will complete whole body imaging, respiratory imaging, and functional assessments that evaluate physical progression of disease.
Dr. Claudia Senesac then shared information on how to get involved in the study, including benefits of participating. The study will help to identify biomarkers beneficial for clinical trials, provide information to pharmaceutical companies to help plan for effective trials, generate natural history data, and provide data back to the participants for their own use.
- People who can participate in the study include people with a diagnosis of Becker muscular dystrophy based on their genetic report, between ages 18-62, and medically stable. Both people who are ambulatory or non-ambulatory are encouraged to participate.
- Exclusion criteria include presence of an internal defibrillator, pacemaker, use of a ventilator full-time, pain preventing lying on your back for an hour, or severe contractures that prevent getting in the magnet.
People who are interested in participating should contact Dr. Senesac by email at firstname.lastname@example.org or by phone at 352-273-6453.
Additional Becker-Specific Information
PPMD is excited to host our first ever Becker-specific breakout sessions at our 2022 Annual Conference, which will be held June 23-26 in Scottsdale, AZ. These breakouts will include a session specific to Becker clinical care, along with a session dedicated to the therapeutic pipeline for Becker muscular dystrophy. We hope to see you there! You can register to attend PPMD’s Annual Conference either in-person or virtually here.
Study Frequently Asked Questions
Will I be paid for participating?
Yes! Participants will be paid $100 for the baseline visit, $150 for the second visit, and $250 for the third visit. All travel and lodging expenses are covered.
Can I bring a loved one with me?
Yes! Participants are able to bring a loved one, parent, or caregiver with them; their costs will also be covered.
How often are the visits?
This study includes three visits, which occur yearly for three years: at baseline, year 2, and year 3. It is possible that future funding may allow the study to extend longer than three years, but current funding allows for a three-year study.
How long is each visit? How long is the scan time?
The visit takes about two days to complete, in addition to travel time. This allows people to minimize fatigue throughout the visit. The scans take about 1.5 to 2 hours to complete per scan, broken up into several blocks of time throughout the visit.
What happens during the visits?
You will have whole body and localized imaging, respiratory assessments, functional testing, muscle biopsy at the year 2 visit (awake, needle biopsy), and complete a medical history.
Is cardiac imaging done as part of this study?
No, this study does not include cardiac MRI. However, there are other protocols that do include cardiac imaging. The ImagingNMD team can share more information about these protocols with you.
What if I am uncomfortable in the MRI magnet, can I still participate?
The ImagingNMD team uses a variety of tools to help support comfort while in the scanner including the use of pillows, pads, bolsters, straps, and sand bags with the overall goal of bringing the surface to the person in the scanner to promote maximum comfort and relaxation during the scanning process. Additionally, the team offers the ability to watch a movie or listen to music during the scan, and can provide a support person in the MRI room for additional support and comfort.
What if my child is not yet 18 years old?
If you are nearing 18 years old, you can contact the ImagingNMD team now to start the planning process. If you are between the ages of 12-18, there may be other studies within the ImagingDMD program where you can participate. Contact the team to discuss what options may be available to you.
What if I have Becker clinically, but my genetics are more indicative of Duchenne?
Current recruitment is targeted at people whose genetic test results indicate Becker muscular dystrophy. If, however, your genetics are more suggestive of Duchenne despite a milder Becker-like presentation you can likely still participate, but in a different arm of the ImagingNMD study. Contact Dr. Senesac, who can discuss which studies are currently recruiting to identify where and how you can participate.
Where can I learn more?
Please contact Dr. Senesac at email@example.com to learn more.