Purpose

The primary goal of this proposal is to collect motor and functional outcomes specific to FSHD over time. By collecting measures specific to FSHD, this will help ensure the best level of clinical care is being provided. Also, the hope is to speed up drug development by gaining a better understanding of how having FSHD impacts motor function and other health outcomes (i.e. breathing, wheelchair use, etc.) and how big a change in motor function would be clinically meaningful to those with FSHD.

Condition

Eligibility

Eligible Ages
All ages
Eligible Genders
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Study Type
Observational
Observational Model
Cohort
Time Perspective
Prospective

Arm Groups

ArmDescriptionAssigned Intervention
Study Visits Patients will receive standard of care as determined by their treating physician. Study visits will occur per standard of care.

Recruiting Locations

Univeristy of Kansas Medical Center
Kansas City, Kansas 66160
Contact:
Michaela Walker, MPH
913-945-9920
mwalker20@kumc.edu

More Details

Status
Recruiting
Sponsor
University of Kansas Medical Center

Study Contact

Michaela Walker, MPH
913-945-9920
mwalker20@kumc.edu

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.