Motor Outcomes Evaluation for Facioscapulohumeral Muscular Dystrophy
(MOVE FSHD Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to gather information on how facioscapulohumeral muscular dystrophy (FSHD) affects movement and everyday functions such as breathing and wheelchair use. By understanding these impacts, the researchers hope to improve care for people with FSHD and accelerate new drug development. Participants will be monitored over time, with some undergoing additional tests like body scans and muscle studies. Individuals with a confirmed diagnosis of FSHD, either genetically or through family history, may be eligible to join. As an unphased study, this trial offers participants the chance to contribute to foundational research that could lead to better treatments and care for FSHD.
Do I need to stop my current medications for the trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.
Why are researchers excited about this trial?
Researchers are excited about the MOVE FSHD trial because it aims to develop a new method for assessing motor outcomes in patients with facioscapulohumeral muscular dystrophy (FSHD). Unlike current treatments and assessments that may not fully capture the progression of motor function in FSHD, this trial seeks to validate more precise and reliable evaluation techniques. By improving how we measure and understand motor changes in FSHD, this approach could lead to more effective treatment strategies and enhance the development of future therapies.
What evidence suggests that this trial's evaluations could be effective for FSHD?
The MOVE FSHD study aims to understand how facioscapulohumeral muscular dystrophy (FSHD) affects movement. Research has shown that specific measurement methods for FSHD can improve patient care and aid in drug development. This study will track changes in movement and daily activities over time to assess FSHD's impact on breathing and mobility. By identifying significant changes in movement, the study seeks to guide better treatment plans. Although this study does not test a specific treatment, it is crucial for enhancing future care and therapies for FSHD.12345
Who Is on the Research Team?
Jeffrey Statland, MD
Principal Investigator
University of Kansas Medical Center
Rabi Tawil, MD
Principal Investigator
University of Rochester
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Baseline Assessment
Initial assessments including motor and functional outcomes specific to FSHD, spirometry, and other baseline measures
Longitudinal Follow-up
Participants are followed for a minimum of 3 years with periodic assessments to collect motor and functional outcomes
MOVE+ Sub-study
Sub-group of participants undergo additional assessments including whole body MRI, reachable workspace, and optional muscle biopsy
Follow-up
Participants are monitored for safety and effectiveness after the main study period
What Are the Treatments Tested in This Trial?
Interventions
- Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Kansas Medical Center
Lead Sponsor
University of Rochester
Collaborator
Dyne Therapeutics
Industry Sponsor
FSHD Canada
Collaborator
AMRA Medical
Collaborator
Seattle Children's Hospital
Collaborator
Avidity Biosciences, Inc.
Industry Sponsor
Hoffmann-La Roche
Industry Sponsor
Dr. Levi Garraway
Hoffmann-La Roche
Chief Medical Officer since 2019
MD from the University of Basel
Dr. Thomas Schinecker
Hoffmann-La Roche
Chief Executive Officer since 2023
PhD in Molecular Biology from New York University
University of Nevada, Reno
Collaborator
Springbok Analytics
Collaborator