Collecting motor and functional outcomes in patients with FSHD
Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
This study is tracking how Facioscapulohumeral Muscular Dystrophy affects movement and daily activities in about 450 people over three years to learn more about the condition.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 450 (estimated) |
| Sex | All |
| Sponsor | University of Kansas Medical Center Academic / other |
| Locations | 21 sites (Los Angeles, California and 20 other locations) |
| Trial ID | NCT04635891 on ClinicalTrials.gov |
What this trial studies
This observational study aims to gather motor and functional outcomes specific to Facioscapulohumeral Muscular Dystrophy (FSHD) over a minimum of three years. Approximately 450 participants with genetically confirmed or clinically diagnosed FSHD will be followed to better understand how the condition affects motor function and other health outcomes, such as breathing and wheelchair use. A subset of 200 participants will also engage in additional assessments, including whole body MRI and optional muscle biopsy, to enhance the understanding of FSHD's impact on health.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals with genetically confirmed FSHD or a clinical diagnosis of FSHD with characteristic findings.
Not a fit: Patients who are unwilling or unable to provide informed consent or have other medical conditions that interfere with participation may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved clinical care and accelerate drug development for patients with FSHD.
How similar studies have performed: Other studies focusing on motor outcomes in neuromuscular diseases have shown promise, indicating that this approach may yield valuable insights.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Genetically confirmed FSHD (types 1 or 2) or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring. Exclusion Criteria: * Unwilling or unable to provide informed consent. * Any other medical condition which in the opinion of the investigator would interfere with study participation.
Where this trial is running
Los Angeles, California and 20 other locations
- David Geffen School of Medicine at UCLA — Los Angeles, California, United States (Recruiting)
- Neuromuscular Disorders Program at Stanford University School of Medicine — Stanford, California, United States (Recruiting)
- University of Colorado Anschutz Medical Campus — Aurora, Colorado, United States (Recruiting)
- Univeristy of Florida Gainesville — Gainesville, Florida, United States (Recruiting)
- University of Iowa — Iowa City, Iowa, United States (Recruiting)
- Univeristy of Kansas Medical Center — Kansas City, Kansas, United States (Recruiting)
- Kennedy Krieger Institute — Baltimore, Maryland, United States (Recruiting)
- University of Rochester Medical Center — Rochester, New York, United States (Recruiting)
- Duke University - Lenox Baker Children's Hospital — Durham, North Carolina, United States (Recruiting)
- The Ohio State University Medical Center — Columbus, Ohio, United States (Recruiting)
- Austin Neuromuscular Center — Austin, Texas, United States (Recruiting)
- Univeristy of Texas Southwestern Medical Center — Dallas, Texas, United States (Recruiting)
- University of Texas Health San Antonio — San Antonio, Texas, United States (Not_yet_recruiting)
- University of Utah — Salt Lake City, Utah, United States (Recruiting)
- Virginia Commonwealth University — Richmond, Virginia, United States (Recruiting)
- University of Washington Medical Center — Seattle, Washington, United States (Recruiting)
- University of Sao Paulo — São Paulo, Brazil (Recruiting)
- University of Calgary — Calgary, Alberta, Canada (Not_yet_recruiting)
- Ottawa Hospital Research Institute — Ottawa, Ontario, Canada (Recruiting)
- University of McGill — Montreal, Quebec, Canada (Not_yet_recruiting)
- Sheffield Teaching Hospital — Sheffield, South Yorkshire, United Kingdom (Recruiting)
Study contacts
- Principal investigator: Jeffrey Statland, MD — University of Kansas Medical Center
- Study coordinator: Michaela Walker, MPH
- Email: mwalker20@kumc.edu
- Phone: 913-945-9920
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.