Quarterly Newsletter

Fall 2019

Forward

Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD (ReSolve FSHD)

ReSolve FSHD has 8 actively recruiting sites in the United States and 3 actively recruiting sites in Europe. Contact a local site today for additional information!

Recruitment Update:
So close!

as of October 1, 2019:

The yearly ReSolve Investigator Meeting at the University of Rochester was a great success. We are ahead of schedule in our recruitment of study subjects both in the US and European sites, thanks to the great support from the FSHD community. The meeting was also attended by representatives from pharmaceutical companies and patient advocacy groups who have been instrumental in supporting this study.

-Dr. Rabi Tawil

Our goal was to recruit 160 US subjects by June 1, 2020.
We hope to meet that goal by the end of 2019!

ReSolve Investigator Meeting Year 3

The Investigators, Clinical Evaluators, Coordinators, Executive Advisory Committee, and Laboratory and Data staff all met on October 26, 2019 to discuss the status of the study and analyze the data. We found the data to be very reliable, which can be attributed to the cross-site training to allow for consistent data collection at all study sites.

Thank you to Friends of FSH Research for sponsoring the Clinical Evaluator training at our meeting! These in-person trainings are key to providing consistent data collection at each site, and perfecting techniques.

The ReSolve FSHD Team

The Patient Perspective
(A note from one of our subjects)

Living with FSHD can provide us with opportunities to be helpful, make a difference, and contribute in a meaningful way to the future. At this point, there is no "cure," but by participating in this important trial you are helping to bring effective treatment ever closer. ReSolve brings significant information to the scientific and medical community, which will further the progress of establishing a treatment plan. This is one way to manage this disease we have no control over at this point.

I have finished 3 of 4 visits spread out over 18 months. The staff deeply appreciates the volunteer effort being made by participants, and I always feel welcome and well taken care of. I believe it is of utmost importance for us to step up to the plate and become part of the network of clinical trials These trials will benefit everyone with FSHD and their families. As my husband and I drive the 5-1/2 hours to participate, I think about being able to help hundreds of thousands of people affected with this disease. Knowing I have been a part of this march for the cure, I will continue to participate in as many trials as possible. By saying yes to the ReSolve trial you make such a difference! If you are considering this trial, I encourage you to take this opportunity to make a difference.

In the News

Dyne Therapeutics recently announced its support for the ReSolve study. They will provide funding for the FSHD CTRN to open additional European sites. This will provide more FSHD patients the opportunity participate in research, and give researchers more diverse data to make more confident conclusions.

https://www.dyne-tx.com/dyne-therapeutics-announces-support-for-resolve-natural-history-study-of-patients-with-facioscapulohumeral-muscular-dystrophy-fshd/

ReSolve Publication

We published the ReSolve protocol in the BMC Neurology journal in September. As this is the largest FSHD study to date, it serves as a model for many other researchers who want to pursue similar studies in other diseases or in FSHD.

Click HERE to read the full article!

Check out our
FSHD CTRN website!

The Ohio State University PI, Dr. Samantha LoRusso, recently conducted a webinar with the FSH Society about FSHD. Click the link to listen to this very informative webinar!

https://www.youtube.com/watch?v=4fu5hH3Q5SM

Study Criteria

If anyone is asking about the ReSolve study, below is the criteria :

18 month study with a total of 5 research visits
We are actively seeking FSHD1 patients at all 8 sites in the US and all 3 sites in Europe with an enrollment goal of 220 participants.

If you have any questions about these requirements, please contact one of the site coordinators below.

Inclusion Criteria:

-FSHD1 genetically confirmed OR you have FSHD symptoms with a genetically confirmed parent, sibling or child.

-Be able to walk 30 feet without the assistance of a person or walker. (Canes, walking sticks, or orthotic braces can be used to complete assessments safely.)

-Leg weakness (i.e. difficulty going upstairs, cannot get up from a seated position without using your hands, cannot walk long distances).

Exclusion Criteria:

-No major heart, breathing, or bone issues that would make study participation unsafe.

-Medication use should not include any muscle anabolic or catabolic agents such as steroids, oral testosterone, or oral beta agonists.

-Women who are pregnant or planning to become pregnant during the course of the study cannot participate. (Those who are postpartum or breastfeeding can safely participate.)

Don't worry! If you don't qualify for the ReSolve study, there are other options available. Contact your local site to see what other studies may be available to you!

ReSolve FSHD Site Contacts

Kiley Higgs-University of Kansas Medical Center
Study Project Manager
Phone: 913.945.9922
Ksims2@kumc.edu

Leann Lewis- University of Rochester Medical Center
Lead Clinical Research Coordinator
Phone: 585.275.7680
Leann_lewis@urmc.rochester.edu

Katie Roath- University of Kansas Medical Center
Clinical Research Coordinator
Phone: 913.945.9928
kroath@kumc.edu

Mary Yep- Kennedy Krieger Institute
Clinical Research Coordinator
Phone: 443.923.7318
Yep@kennedykrieger.org

Dianne DeGuzman- University of California Los Angeles
Clinical Research Coordinator
Phone: 310.825.3264
ddeguzman@mednet.ucla.edu

Marco Tellez - The Ohio State University
Clinical Research Coordinator
Phone: 614.688.7837
Marco.Tellez@osumc.edu

Laura Sissons-Ross- University of Washington
Clinical Research Coordinator
Phone: 206.543.0081
lsissons@uw.edu

Sarah Moldt- University of Utah
Clinical Research Coordinator
Phone: 801.585.9399
Sarah.moldt@hsc.utah.edu

Raven Pillow-Virginia Commonwealth University
Clinical Research Coordinator
Phone: 804.404.9336
raven.pillow@vcuhealth.org