What’s so great about the MOVE study? A lot, if you are research geeks like us or care about getting treatments to patients sooner. MOVE is what’s called an “observational” or “natural history” study of people with facioscapulohumeral muscular dystrophy (FSHD) that evaluates how symptoms and abilities change over a period of time. It is currently recruiting volunteers through the 12 centers of the FSHD Clinical Trial Research Network. Excitingly, MOVE accepts volunteers of all ages, including children.
There have been other natural history studies in FSHD, so why do we need another one? In this webinar, neurologist Johanna Hamel, MD, and research physical therapist Katy Eichinger, PhD, explain. One problem with traditional research studies is that they have focused on major milestones such as needing a brace, or walker, or wheelchair, which may occur only once in many years. Some individuals will never reach one of these milestones. A three-year study might not capture any.
MOVE asks the question, is there a way to measure smaller changes in muscle strength during a doctor’s appointment? The study eliminates the need for patients to make a separate visit to a research lab. And by having doctors or other clinicians (such as a physical therapist) do the tests, it creates “an infrastructure for better care.”
Engaging more people in research
MOVE solves a second problem, engaging people in research. Right now, only a small subset of people living with FSHD participate in research. “They are usually financially better off and more mobile, compared to the whole group of people living with FSHD,” Hamel observes. Someday, “if there is a drug for FSHD, we need to know that it works for everyone with FSHD, not just for the small group that volunteers.” Future treatments also need to be monitored for safety and efficacy, Hamel adds, and so “it’s important to prepare the infrastructure”–experienced doctors and physical therapists–“to collect this information.”
People who go to the doctor are a larger and more diverse group than those who volunteer for research. “MOVE will expand our bubble to include clinic patients. It overcomes the artificial barrier between clinic and research,” Hamel explains. The study aims to enroll 300 volunteers by the end of this year. Because MOVE is clinic-based, it can easily be extended to collect data beyond its initial 3 years.
MOVE also will reach out to patients who aren’t able to go to one of the clinical trial research centers where the study is being conducted. To engage people who are seeing community doctors, MOVE has added a sub-study that uses remote assessments. The remote assessment sub-study is being piloted at the University of Kansas and University of Rochester and aims to enroll 20 volunteers. “Instead of you traveling to us, we will travel to you by sending a toolkit to your home and we do a remote assessment,” Hamel said. “It’s a great idea but is it feasible? Will it work? That’s what we’re assessing.”
Why is this important?
Studies like MOVE are essential for developing drugs that significantly improve the lives of people with FSHD. It also aims to discover which factors predict whether and when an individual will reach on of the milestones of needing an AFO, mobility aid, or wheelchair. While one may not want to hear this information, it may be better to know and be able to plan for it than to live in a constant state of existential dread.
How to enroll
- Contact Michaela Walker and let her know you’re interested
- The MOVE study can be done during a CTRN clinic visit
- If you don’t live close to a CTRN, ask if you can enroll through Telehealth visits.
What happens during the clinic visit
- Your clinical history is noted
- Blood and saliva are collected
- You will be asked about symptoms, function, pain and exercise
- Your strength will be assessed through manual muscle testing. This is a hands-on, with the clinician pushing and pulling different parts of the body to assess strength and see how various muscle groups are working.
- You will also have pulmonary function tests to better understand the muscles involved with breathing.
- Details available at clinicaltrials.gov.
My daughter and 2 granddaughters have FSHD. I do not but am the source of the disease. Do you ever collect information from people like me?
You are what researchers call a “nonsymptomatic” individual with FSHD, and yes, there is great interest in studying families like yours. It’s possible that you have some genetic variant that protects you from developing symptoms. It might be possible to determine what that is by comparing your DNA with that of your symptomatic daughter and granddaughters. This in turn could lead to ideas for new therapies that might mimic whatever is protecting you. We recommend that you contact a clinical trial research network site close to you (if there is one) and make inquiries.
https://www.fshdsociety.org/therapeutic-accelerator/clinical-trials-copy/
Is anything like this or any other trial available to people living with FSHMD in Australia
Not yet, but it’s something we’re looking into. We strongly recommend that you join the Australian FSHD registry. That is a natural history study that is intended to provide the backbone for additional studies like MOVE. To sign up, contact Robin Forbes robin.forbes@mcri.edu.au.
Hello, my mother, brother and myself suffer from FSHD, we are all located in Sydney, Australia. Is there any way I could volunteer and give data for the study from here?
Thanks so much for your interest. We apologize that our MOVE story implied “anyone in the world.” Currently the study is open only in the U.S., but there is constant discussion about FSHD studies elsewhere. For you and your family, we recommend you enroll in the Australian FSHD registry. That is a natural history study, which is the backbone for all clinical trial readiness studies. To sign up, contact Robin Forbes robin.forbes@mcri.edu.au.
Can people in the UK take part? I live in Scotland.
Thanks for your interest. The MOVE study is currently only open to the U.S. (we should have been specific about that), but there may be opportunities to participate in research coming up in the UK. University College London is in the process of joining the international FSHD Clinical Trial Research Network. Also, if you have not enrolled in the UK FSHD patient registry, we recommend that you do. That’s an excellent registry and is conducting a variety of important studies.
Hello,
Whats the expected country of residency for a volunteers participating in this research? I will be glad to help.
Thank you
Our apologies for the U.S-centricity of our MOVE story. Currently the study is open only in the U.S.
Hi,
Great to hear of this interest in accessing the small changes that may not be apparent to those other than the patient and caregivers. I have had more than 30 years of annual strength measurements and volumetric capacity testing at the Hospital for Special Care in New Britain, CT under Dr, Kevin Felice. The HFSC supports over 100 FSHD patients and could provide historic baseline data to the study. I would certainly release my data and encourage others as well.
Hope the patient in China can take part